Options mapped

Pediatric solid tumors: options by country

Sourced options by country plus visit-prep questions for pediatric solid tumors, with age-specific sources and a careful, family-centered tone.

Options mappedPediatricLast checked May 2026

Options by country

Treatments by country

Regulatory and access status by country, from official sources. It shows what exists and where — not a recommendation.

Neuroblastoma

United States

Risk-adapted standard therapy (surgery, chemotherapy, radiation, myeloablative therapy with stem-cell rescue, dinutuximab/GM-CSF/isotretinoin per risk group)[1]NCI PDQ: listed among standard treatment optionschildhood neuroblastoma, low- / intermediate- / high-risk and stage 4S/MS, treatment assigned by risk group · PDQ is an information summary, not a patient-specific eligibility rule; the regimen is selected by risk group and clinical factors.
dinutuximab (Unituxin)[2]FDA-approvedhigh-risk neuroblastoma in pediatric patients who achieved at least a partial response to prior first-line multiagent, multimodality therapy; given with GM-CSF, IL-2 and isotretinoin · Anti-GD2 antibody; the prescribing information carries boxed warnings (serious infusion reactions, neuropathic pain). Refresh against current Drugs@FDA labeling before patient-facing reuse.

European Union

dinutuximab beta (Qarziba)[3]EMA-authorised (central marketing authorisation)high-risk neuroblastoma in patients aged 12 months and over, after induction chemotherapy and stem-cell transplant, and relapsed/refractory disease · Central EU authorisation only; member-state reimbursement (e.g. Germany/France) not verified.

United Kingdom

dinutuximab beta (Qarziba)[4]NICE-recommended on the NHS (England and Wales)high-risk neuroblastoma in people aged 12 months and over · Recommended only under the agreed commercial arrangement; NHS England/Wales context, not a universal UK availability statement. Appraisal published 2018.

NTRK fusion-positive sarcoma

United States

larotrectinib (Vitrakvi)[5]FDA-approvedadult and pediatric solid tumours with an NTRK gene fusion (no known resistance mutation) that are metastatic or where surgery is likely to cause severe morbidity, with no satisfactory alternative or progression after treatment · Tissue-agnostic approval; applies to pediatric sarcoma only when an NTRK fusion is confirmed by an adequate test.
entrectinib (Rozlytrek)[6]FDA-approvedNTRK fusion-positive solid tumours in patients (including children as young as 1 month) that are metastatic or unresectable, after standard treatment with no effective alternatives · Tissue-agnostic approval; pediatric expansion to age 1 month noted. Applies to pediatric sarcoma only when an NTRK fusion is confirmed.
repotrectinib (Augtyro)[7]FDA-approved (accelerated approval)adult and pediatric patients aged 12 years and older with NTRK fusion-positive solid tumours that are locally advanced or metastatic or where surgery is likely to cause severe morbidity, after progression or with no satisfactory alternative therapy · Accelerated approval (age 12+); applies to pediatric sarcoma only when an NTRK fusion is confirmed.

European Union

larotrectinib (Vitrakvi)[8]EMA-authorised (central marketing authorisation)adult and paediatric solid tumours with an NTRK gene fusion, locally advanced or metastatic or where surgical removal is likely to cause serious complications, with no satisfactory treatment options · Central EU authorisation only; tissue-agnostic — applies to pediatric sarcoma only when an NTRK fusion is confirmed. Member-state reimbursement not verified.

Medulloblastoma

United States

Standard multimodality therapy (maximal safe surgery, craniospinal radiation, chemotherapy; selected stem-cell-rescue contexts)[9]NCI PDQ: listed among standard treatment optionschildhood medulloblastoma, average-risk / high-risk / younger-child contexts · PDQ is an information summary; radiation approach is age-dependent and craniospinal irradiation is generally deferred in very young children.

European Union

Standard treatment framework (surgery followed by craniospinal irradiation and chemotherapy; modern radiation techniques)[10]Standard treatment framework (SIOP Europe)childhood medulloblastoma, broad European standard-of-care plan · European professional-society clinical-plan document, not a reimbursement or country-access statement.

United Kingdom

Standard treatment (surgery, radiotherapy, chemotherapy)[11]Standard treatment framework (Cancer Research UK)children's medulloblastoma, broad treatment categories · Patient-information framework; page review was due January 2026, so confirm against current guidance. Treatment for children under 3 differs.

Ewing sarcoma

United States

Multimodality therapy (multiagent chemotherapy, surgery, radiation; high-dose chemotherapy with autologous stem-cell rescue in selected contexts)[12]NCI PDQ: listed among standard treatment optionslocalized, metastatic, and recurrent Ewing sarcoma · PDQ is an information summary; systemic chemotherapy is required for all patients, with local control by surgery and/or radiation per tumour site.

Rhabdomyosarcoma

United States

Multimodality therapy (chemotherapy, surgery, radiation therapy; selected brachytherapy)[13]NCI PDQ: listed among standard treatment optionschildhood rhabdomyosarcoma, localized / metastatic / relapsed contexts · PDQ is an information summary; all patients receive chemotherapy, with surgery and/or radiation for local control by risk group.

Wilms tumor

United States

Risk-adapted therapy (nephrectomy or kidney-sparing surgery where appropriate, vincristine/dactinomycin/doxorubicin-based chemotherapy, radiation by stage and histology)[14]NCI PDQ: listed among standard treatment optionsnewly diagnosed unilateral or bilateral and recurrent Wilms tumor (nephroblastoma) · PDQ is an information summary; North American practice generally begins with nephrectomy, whereas European (SIOP) practice begins with preoperative chemotherapy.

Pediatric solid tumors

United States

Chemotherapy including vincristine, dactinomycin, cyclophosphamide, irinotecan, temozolomide, ifosfamide, etoposide contexts; surgery; external-beam radiation; brachytherapy in selected sites; allogeneic bone marrow transplant context in rare reports [13]Standard option (per NCI PDQ)Fusion status and histology influence risk group in modern RMS care, but no biomarker-gated approved therapy was recorded from fetched sources; Localized, metastatic, and recurrent/refractory childhood RMS as described in NCI PDQ. · NCI PDQ is an evidence summary, not personalized advice. Risk group, primary site, histology, fusion status, age, and clinical trial availability affect treatment planning. Confidence/conflicts: High for NCI multimodal framework; no drug-specific approval claim made.
Enucleation, local focal therapy including cryotherapy and thermotherapy, plaque radiation therapy, external-beam radiation therapy, systemic chemotherapy, intra-arterial chemotherapy, intravitreal chemotherapy, CNS-directed therapy, myeloablative chemotherapy with autologous hematopoietic stem cell rescue, surveillance and genetic counseling [15]Standard option (per NCI PDQ)RB1 germline or somatic pathogenic variant context; MYCN amplification noted in a rare RB1-negative subset; treatment grouping is driven by intraocular/extraocular extent, unilateral/bilateral disease, eye-salvage potential, seeds, optic nerve/orbital/metastatic involvement, and heritable-risk surveillance; Newly diagnosed intraocular and extraocular retinoblastoma; progressive/recurrent intraocular and extraocular retinoblastoma; heritable retinoblastoma surveillance context. · NCI emphasizes balancing life-saving treatment with preservation of useful vision. The source distinguishes intraocular from extraocular disease and notes poor prognosis for intracranial disease; do not generalize ocular-salvage options to extraocular or metastatic disease. Confidence/conflicts: High for treatment-category framework; no approval claim for individual chemotherapy routes is made.
External-beam radiation therapy; chemotherapy for infants in selected contexts; palliative care/supportive care [16]NCI PDQ: standard optionH3 K27M/H3 K27-altered status is relevant to diffuse midline glioma classification, but the NCI patient DIPG page records broad treatment categories; Newly diagnosed DIPG for external-beam radiation; infant chemotherapy context; supportive/palliative care throughout; pediatric diffuse high-grade glioma categories from NCI PDQ. · NCI summaries are evidence information, not individualized advice or a payer rule. Surgery is often limited by location for DIPG/DMG; clinical trials and molecular testing are especially important to discuss with specialists. Confidence/conflicts: Medium-high for broad NCI modality categories; DMG/DIPG-specific sequencing and trial options require specialist protocol review.
Maximal safe surgery; second-look surgery for residual disease; conformal radiation therapy including proton/charged-particle approaches; selected preirradiation chemotherapy in residual or very-young-child contexts [17]Standard option (per NCI PDQ)NCI PDQ discusses molecular-risk features including PF-EPN-A, 1q gain, ZFTA fusion, and posterior fossa subgrouping in outcome context; Newly diagnosed childhood ependymoma after surgery, residual nondisseminated disease, CNS disseminated disease, and children younger than 1 year/very young children. · NCI PDQ is an evidence summary, not a payer or personalized treatment rule. Radiation dose, reoperation, chemotherapy, proton therapy, and late-effect decisions depend on age, tumor site, resection extent, dissemination, and molecular subgroup. Confidence/conflicts: High for NCI modality categories and chemotherapy caveat; no conflict found in fetched source.
Multiagent chemotherapy including vincristine, doxorubicin, cyclophosphamide, ifosfamide, and etoposide contexts; surgery; radiation therapy; high-dose chemotherapy with autologous stem cell rescue in selected contexts [12]Standard option (per NCI PDQ)Ewing sarcoma family translocation/fusion biology relevant to diagnosis; no targeted approved therapy biomarker recorded from fetched sources; Localized, metastatic, and recurrent Ewing sarcoma contexts as described in NCI PDQ. · NCI PDQ is an evidence summary, not personalized advice. Recurrent Ewing sarcoma chemotherapy is noted as not standard treatment in the table, so trial/specialist discussion is important. Confidence/conflicts: High for NCI PDQ multimodal treatment framework; no targeted approval claim made.
Nephrectomy or kidney-sparing surgery where appropriate, chemotherapy including vincristine, dactinomycin, doxorubicin and other risk-adapted agents, radiation therapy, observation in selected very-low-risk contexts, relapse therapy including chemotherapy-surgery-radiation and possible hematopoietic stem cell transplant in selected high-risk relapse contexts [14]Standard option (per NCI PDQ)Risk, histology, stage, loss of heterozygosity, 1q gain, bilateral predisposition, and syndrome context are discussed by NCI; no single targetable biomarker is presented as a routine approved-treatment selector in this finding.; Newly diagnosed unilateral, bilateral/stage V, and recurrent Wilms tumor settings as described in the PDQ. · NCI distinguishes COG immediate-nephrectomy and SIOP preoperative-chemotherapy approaches and emphasizes risk stratification. Do not infer that any listed component applies to every child or adult with Wilms tumor. Confidence/conflicts: High for U.S. national evidence-summary framework; no conflict recorded. The source is a broad PDQ and does not substitute for protocol-specific eligibility.
Observation; surgery; chemotherapy; radiation therapy; targeted therapy; dabrafenib (Tafinlar) plus trametinib (Mekinist); tovorafenib (Ojemda)[18]FDA accelerated approvalBRAF V600E for dabrafenib plus trametinib; BRAF fusion/rearrangement or BRAF V600 mutation for tovorafenib; broader MAPK pathway alterations noted in NCI recurrent/progressive discussion; Newly diagnosed low-grade glioma requiring systemic therapy for dabrafenib plus trametinib; relapsed or refractory BRAF-altered pediatric low-grade glioma after at least one prior systemic therapy for tovorafenib; NCI framework includes newly diagnosed and progressive/recurrent settings. · FDA approvals are biomarker- and setting-specific. NCI states management should be guided by a multidisciplinary pediatric brain tumor team and notes no single standard treatment option for progressive/recurrent low-grade glioma categories. Confidence/conflicts: High for U.S. framework and FDA approval statuses. No conflict identified; FDA tovorafenib approval is accelerated and setting-specific.
Sodium thiosulfate (Pedmark in the United States; Pedmarqsi in the European Union/United Kingdom)[19]FDA-approvedNot biomarker-selected; linked to cisplatin exposure and localized/non-metastatic solid tumor setting; Supportive otoprotection after cisplatin for localized, non-metastatic pediatric solid tumors; FDA and EMA note SIOPEL 6 hepatoblastoma evidence among the supporting studies. · This is supportive care to reduce hearing-loss risk, not anticancer therapy. FDA warns Pedmark is not substitutable with other sodium thiosulfate products. NICE access is tied to the commercial arrangement. Confidence/conflicts: High for supportive-care approval/reimbursement statements; scope is localized/non-metastatic solid tumors, not all hepatoblastoma.
Surgery; craniospinal radiation therapy; adjuvant chemotherapy including cisplatin/lomustine/vincristine or cisplatin/cyclophosphamide/vincristine regimens; selected high-dose chemotherapy with stem cell rescue contexts [9]Standard option (per NCI PDQ)Risk and molecular subgrouping are used in NCI PDQ context; WNT, SHH, group 3/group 4 and TP53-related risk caveats are discussed by NCI; Newly diagnosed medulloblastoma, stratified by age, average-risk versus high-risk features, metastatic disease, extent of resection, and molecular/histologic subgroup. · NCI PDQ is an evidence summary, not a personalized recommendation or payer rule. Treatment varies substantially by age, molecular subgroup, metastatic status, residual disease, and late-effect risk. For younger children, NCI notes efforts to omit or delay radiation because of developmental toxicity. Confidence/conflicts: High for NCI-listed modality categories; regimen details require pediatric/adult neuro-oncology protocol review.
observation/supportive care; surgery; chemotherapy; radiation therapy; myeloablative therapy with hematopoietic stem cell transplant; dinutuximab with GM-CSF and isotretinoin [1]Standard option (per NCI PDQ)MYCN amplification and tumor biology affect risk assignment per NCI PDQ context; no single biomarker required for all options in this finding; Low-risk: observation and/or surgery with emergency radiation only in selected contexts; intermediate-risk: chemotherapy with or without surgery, surgery/observation in infants, radiation for progressive disease if needed; high-risk: induction, consolidation, and postconsolidation phases; stage 4S/MS: observation/supportive care for asymptomatic favorable biology and chemotherapy for symptomatic/unfavorable contexts. · PDQ is an evidence summary, not individualized eligibility guidance; risk assignment and treatment intensity require pediatric oncology team assessment. Confidence/conflicts: High confidence for U.S. evidence-summary framework. Specific product approval/access requires separate regulator or payer sources.

European Union

CCNU/lomustine; cisplatin; carboplatin; vincristine; ifosfamide; cyclophosphamide; etoposide; high-dose methotrexate; intraventricular methotrexate; high-dose chemotherapy with autologous stem-cell transplantation [20]Standard option (per AWMF / Gesellschaft für Pädiatrische Onkologie und Hämatologie)SHH-activated, desmoplastic/nodular, MBEN, non-WNT/non-SHH, TP53, MYC/MYCN, metastasis/residual disease contexts; Postoperative maintenance chemotherapy, young-child radiation-sparing strategies, high-risk/metastatic and selected consolidation contexts. · The guideline notes most substances do not have formal pediatric marketing authorization, but their use is established in prospective studies. This is an options-to-discuss catalog entry, not proof of individual eligibility or reimbursement. Confidence/conflicts: High for guideline-listed systemic and cell-therapy contexts; regulatory authorization for individual pediatric uses remains a caveat. No conflict identified. Primary source not in English. English summary pending human review — confirm exact wording with your care team.
Chemotherapy; surgery; radiotherapy [21]Standard option (per SIOP Europe)Risk group assignment; no regulator-approved biomarker-gated therapy recorded from fetched source; Pediatric RMS multimodal treatment across risk groups. · SIOP Europe is a clinical practice source, not a regulator/reimbursement source. National protocols and trial availability vary. Confidence/conflicts: Medium-high for European RMS framework; national reimbursement not established.
Chemotherapy; surgery; radiotherapy; high-dose chemotherapy/stem cell support in selected guideline contexts [22]EMA authorisedFusion/translocation testing relevant to diagnosis; no EMA-authorised Ewing-specific targeted therapy verified in this cycle; Localised and disseminated Ewing sarcoma guideline context. · SIOP Europe is a clinical practice recommendation source, not a regulator or reimbursement source. EU member-state reimbursement and national protocols require separate verification. Confidence/conflicts: Medium-high for European multimodal framework; regulator/reimbursement status remains source-pending.
Dabrafenib (Finlee) plus trametinib (Spexotras)[23]EMA authorisedBRAF V600E mutation confirmed before treatment; Pediatric patients aged 1 year and older with low-grade glioma with BRAF V600E mutation who require systemic therapy. · EMA marketing authorization does not by itself define access in every EU member state. NICE applies to England/Wales NHS appraisal context. HAS and G-BA are national reimbursement/benefit-assessment sources and should not be generalized to all EU countries. Confidence/conflicts: High for EMA, NICE, HAS, and G-BA claims. No conflict identified; country-specific reimbursement scope is intentionally separated from EMA authorization.
Initial surgery; craniospinal irradiation; chemotherapy; modern radiation techniques including tomotherapy, VMAT, and proton therapy as toxicity-reduction approaches [10]Established standard of careMolecular subgrouping is recognized in the SIOP Europe plan but this cell records broad standard treatment; Newly diagnosed medulloblastoma standard-treatment framework. · This is a European pediatric oncology clinical research/strategy source, not an EMA medicine approval, EU-wide reimbursement decision, or country-specific Germany/France access source. Local protocols and trial participation can differ by center and country. Confidence/conflicts: Medium-high for Europe standard-treatment framework; country-specific reimbursement/access remains unverified.
Surgery; radiotherapy; selected chemotherapy schedules in residual disease or young-child radiation-delay contexts [24]Standard option (per SIOP Europe)Pediatric ependymoma standard-practice framework, especially post-surgery radiotherapy and selected chemotherapy contexts. · This is a European pediatric oncology professional standard-practice document, not an EMA approval, reimbursement decision, or country-specific Germany/France access rule. The fetched source cautions that chemotherapy's role is unproven compared with surgery and radiotherapy. Confidence/conflicts: Medium-high for Europe standard-practice framing; no country reimbursement inference.
brain and spine MRI staging; diffusion-weighted imaging; central neuroimaging review; biopsy mainly for atypical DIPG imaging or research/clinical-trial contexts; molecular classification and registry participation [25]Standard option (per SIOP Europe High-Grade Glioma Working Group)H3K27-altered DMG; H3.3/H3.1/H3.2 K27M; EZHIP overexpression with H3K27me3 loss; ACVR1, PDGFRA, MYC, EGFR contexts; MYCN-amplified pediatric HGG differential context; Initial diagnostic staging, trial/registry preparation, and molecular classification. · This is a SIOP Europe standard-practice document, not an EMA/MHRA marketing authorization or national reimbursement rule. Local country implementation may differ. Confidence/conflicts: High for SIOP Europe diagnostic/staging framework. No conflict identified.
chemotherapy with cisplatin, vincristine, carboplatin, etoposide, cyclophosphamide; high-dose chemotherapy with autologous stem-cell transplantation not supported by current evidence; selected molecular-target search or drug testing in recurrence/refractory contexts [26]Standard option (per AWMF / GPOH and German pediatric neuro-oncology contributors)Not drug-biomarker-specific; age, residual disease, metastasis, spinal location, and recurrence/progression contexts described; Study-based chemotherapy, residual-disease second-look strategy, very-young-child radiation-delay, progression/dissemination when local options are exhausted. · This is a guideline caveat cell, not an approval or routine-access claim. Pediatric drug use, study participation, and off-label status require local protocol review. Confidence/conflicts: High for guideline chemotherapy caveats; individual-drug regulatory/procurement status is not established. No conflict identified. Primary source not in English. English summary pending human review — confirm exact wording with your care team.
craniospinal irradiation; local tumor-bed/posterior-fossa boost; IMRT/VMAT/tomotherapy/proton therapy; image-guided radiotherapy [20]Standard option (per AWMF / Gesellschaft für Pädiatrische Onkologie und Hämatologie)PTCH/SUFU/Gorlin and TP53/Li-Fraumeni germline contexts influence radiation risk discussions; WNT subgroup de-escalation is under study; Postoperative radiotherapy planning for pediatric/adolescent medulloblastoma. · PTCH/SUFU/Gorlin and Li-Fraumeni contexts require individualized radiation-risk discussion. Access to proton therapy and exact protocol details should be checked locally. Confidence/conflicts: High for German guideline radiotherapy framework. No conflict identified. Primary source not in English. English summary pending human review — confirm exact wording with your care team.
focal/adjuvant radiotherapy; conformal radiotherapy; evaluation of second-look surgery; chemotherapy such as VEC or modified HIT-SKK bridging contexts; avoidance of intraventricular methotrexate in the cited standard-practice document [24]Standard option (per SIOP Europe Brain Tumour Group)WHO 2021 molecular groups; residual disease; age >=12 months versus younger; metastatic/disseminated status; European pediatric practice context where SIOP Ependymoma II enrollment/stratum criteria are not met. · This is a SIOP Europe standard clinical practice document rather than a French regulator decision. Local French center practice, trial eligibility, and reimbursement must be confirmed. Confidence/conflicts: Medium-high for EU practice guidance applicable to France through SIOP Europe context; not a France-specific legal availability claim. No conflict identified.
histopathologic and molecular classification; maximal safe resection; intraoperative neurophysiologic monitoring and imaging where available; second-look resection if residual tumor remains [26]Standard option (per AWMF / GPOH and German pediatric neuro-oncology contributors)WHO 2021 molecular classification; supratentorial, infratentorial, and spinal biologic groups; ZFTA/RELA, MYCN-amplified spinal ependymoma, NF2 association, myxopapillary ependymoma contexts; Initial diagnosis, surgery, molecular workup, and postoperative residual-disease evaluation. · German-language professional guideline; human review is needed before patient-facing reuse. Complete resection must be balanced against cranial-nerve, brainstem, and spinal-cord functional risks. Confidence/conflicts: High for German guideline classification and surgical framework. No conflict identified. Primary source not in English. English summary pending human review — confirm exact wording with your care team.
lomustine (Lomustine Medac; Belustine reference product)[27]ApprovedNot biomarker-specific; adults and children older than 3 years are specifically described for first-line post-surgery use with radiotherapy when surgery is not feasible or is insufficient; First-line post-surgery medulloblastoma in adults and children older than 3 years when radiotherapy-associated chemotherapy is described. · HAS notes Belustine shortage/replacement history and that several medulloblastoma drugs are used off-label. This entry does not establish individual eligibility, supply, dose, or reimbursement implementation at a specific hospital. Confidence/conflicts: High for HAS reimbursement-position statement and medulloblastoma place-in-therapy. No conflict identified.
multidisciplinary planning; maximal safe neurosurgical resection; CSF diversion when needed [20]Standard option (per AWMF / Gesellschaft für Pädiatrische Onkologie und Hämatologie)SHH-activated, desmoplastic/nodular, extensive nodularity, WNT, TP53 germline, PTCH/SUFU, MYC/MYCN, metastasis and residual disease contexts described; Initial therapy planning, surgery, and hydrocephalus management. · German-language professional guideline; translation/human review is needed before patient-facing reuse. Surgery must be balanced against neurologic risk. Confidence/conflicts: High for German guideline surgical/supportive framework. No conflict identified. German-language clinical content requires human review. Primary source not in English. English summary pending human review — confirm exact wording with your care team.
postoperative local radiotherapy; high-conformal radiotherapy; proton therapy; craniospinal irradiation for metastatic disease; re-irradiation in recurrence contexts [26]Standard option (per AWMF / GPOH and German pediatric neuro-oncology contributors)Metastatic versus nonmetastatic status; residual tumor; age; spinal ependymoma context; Postoperative adjuvant radiotherapy and recurrent-disease radiotherapy planning. · Exact dose/fractionation details are not reproduced here. Radiotherapy decisions depend on age, prior radiation, residual disease, metastasis, and center expertise. Confidence/conflicts: High for German radiotherapy framework. No conflict identified.
proton therapy; PBS-IMPT craniospinal irradiation at Institut Curie Orsay [28]Standard option (per Institut Curie)Pediatric tumor and medulloblastoma context; no molecular eligibility stated; Craniospinal irradiation access context for pediatric/adult medulloblastoma. · This is a provider/center source describing capability and recognized indications, not proof that an individual patient can access proton therapy or that it is preferred in every case. Confidence/conflicts: Medium-high for France proton availability and Curie craniospinal capability; access and indication review remain local. No conflict identified.
proton therapy; PBS-IMPT craniospinal irradiation capability; intensity-modulated radiotherapy and stereotactic radiotherapy as broader CNS radiotherapy techniques [28]ApprovedNot biomarker-specific; pediatric tumor and CNS tumor-location context; Radiotherapy modality availability for selected pediatric/adolescent CNS tumor cases; ependymoma-specific use requires radiation oncology review. · Curie pages document French proton-service availability and pediatric/CNS indication context but do not state that every ependymoma should receive proton therapy. Eligibility depends on tumor location, prior therapy, age, treatment plan, and center access. Confidence/conflicts: Medium-high for proton availability in France and pediatric/CNS context; ependymoma-specific use is inferred from CNS/pediatric radiotherapy context and should be verified by the treating center. No conflict identified.
radiotherapy; standard fractionated radiotherapy; hypofractionated radiotherapy; re-irradiation; craniospinal irradiation with boost for metastatic DMG; corticosteroids restricted to specific symptom contexts [25]Standard option (per SIOP Europe High-Grade Glioma Working Group)DIPG versus non-DIPG DMG; metastatic disease; recurrent/progressive disease; raised intracranial pressure/steroid context; Initial DIPG radiotherapy, metastatic DMG radiotherapy, recurrent/progressive re-irradiation, and symptom-supportive steroid use. · Dose/fraction data are recorded as source details, not medical advice. Re-irradiation and CSI require individualized radiation oncology review. Confidence/conflicts: High for SIOP Europe radiation/supportive framework. No conflict identified.
surgery; craniospinal irradiation; chemotherapy including cisplatin, cyclophosphamide, vincristine, lomustine, etoposide, carboplatin, methotrexate; high-dose chemotherapy contexts [27]HAS reimbursement opinionAge, tumor biology, molecular risk factors, standard-risk/high-risk, metastasis, MYC amplification, and beta-catenin mutation contexts described by sources; Initial and postoperative France pathway context for pediatric and adult medulloblastoma. · HAS is an HTA/reimbursement body summarizing external guideline frameworks rather than issuing a France-only clinical protocol. Gustave Roussy is a cancer-center source; exact protocol, risk group, and access decisions require local specialist review. Confidence/conflicts: Medium-high for France pathway context; HAS and Gustave Roussy are directionally consistent. No conflict identified. Primary source not in English. English summary pending human review — confirm exact wording with your care team.
temozolomide (TMZ) chemoradiation and adjuvant TMZ as contested practice; targeted/biologic agent only preferably in clinical trial when target identified; systemic chemotherapy caveat [25]Studied — did not show benefitH3K27-altered DMG; pontine versus non-pontine DMG; MGMT promoter context; biopsy-identified target context; Newly diagnosed and recurrent/progressive H3K27-altered DMG/DIPG systemic therapy discussion and trial matching. · Do not present TMZ or targeted agents as standard or proven for DIPG. This is explicitly an area of controversy and weak evidence. Confidence/conflicts: High for SIOP Europe chemotherapy controversy and trial-first targeted-agent caveat. No conflict identified.

United Kingdom

Chemotherapy; surgery; radiotherapy [29]Standard option (per British Journal of Cancer / UK sarcoma guideline authors)No biomarker required by fetched UK sources; Pediatric/AYA RMS multimodal treatment context. · CCLG is not a drug regulator or reimbursement source. UK national/devolved protocols, trial availability, and specialist MDT decisions need separate verification. Confidence/conflicts: Medium-high for broad UK multimodal framework; exact national RMS protocol remains open.
Multiagent chemotherapy; surgery; radiotherapy; MDT pathway and supportive/palliative radiotherapy contexts [30]NICE recommendedNo biomarker required by fetched UK sources; Localised and metastatic/palliative bone sarcoma/Ewing sarcoma contexts in UK guidance. · Sources are UK service/guideline context rather than medicine reimbursement decisions. Exact regimen/funding is determined by UK sarcoma MDTs and national/devolved policies. Confidence/conflicts: Medium-high for UK multimodal guideline framework; no drug-specific UK reimbursement claim made.
Surgery; radiotherapy; chemotherapy [11]Standard option (per Cancer Research UK)Broad treatment categories for children's medulloblastoma / children's brain tumours. · The Cancer Research UK page was last reviewed January 3, 2023 and listed next review due January 3, 2026, so UK medulloblastoma protocol and NHS commissioning details remain active refresh gaps. The source does not give individualized sequencing, eligibility, or molecular risk protocol details. Confidence/conflicts: Medium for broad treatment categories; source is overdue for review and does not establish NHS access details.
Surgery; radiotherapy; chemotherapy in selected contexts; follow-up MRI surveillance [31]Standard option (per Cancer Research UK)Cancer Research UK notes treatment depends on type including grade and molecular markers; Broad UK treatment categories for newly diagnosed and recurrent ependymoma. · Cancer Research UK is patient guidance, not a NICE funding decision or detailed NHS protocol. It does not establish eligibility, exact radiation fields, chemotherapy regimen, or individual access. Confidence/conflicts: Medium-high for broad UK modality categories; detailed NHS protocol/funding status remains active gap.

Japan

avoidance of tumor resection; hydrocephalus surgery when hydrocephalus occurs; ventriculoperitoneal shunt and other CSF-diversion approaches as clinically selected [32]Standard option (per Japan Society for Neuro-Oncology)Brainstem/pons diffuse tumor; hydrocephalus context; Initial surgical decision-making and hydrocephalus management. · The source distinguishes tumor resection from biopsy or hydrocephalus procedures. No individual surgical eligibility is implied. Confidence/conflicts: High for Japan guideline surgery and hydrocephalus distinction. No conflict identified. Primary source is in Japanese. English summary pending human review — confirm exact wording with your care team.
chemotherapy before radiotherapy to delay radiation in young children; chemotherapy before second-look surgery in selected residual tumor contexts [33]Standard option (per Japan Society for Neuro-Oncology)Age under 3 years, residual tumor, histology, and molecular classification inform risk/late-effect decisions; no drug-specific biomarker stated; Selected postoperative residual-disease planning and infant/young-child radiation-delay strategy. · This is not a general endorsement of chemotherapy for all ependymoma. The guideline states evidence is limited and toxicity is important; individual regimens and dosing require specialist protocol review. Confidence/conflicts: High for guideline-stated chemotherapy caveats; regimen-level access remains unresolved. No conflict identified. Primary source is in Japanese. English summary pending human review — confirm exact wording with your care team.
cisplatin; cyclophosphamide; vincristine; craniospinal irradiation and local boost [34]Standard option (per Japan Society for Neuro-Oncology)Risk-group context; no single biomarker eligibility stated in the recommendation; Postoperative standard-risk medulloblastoma in patients aged at least 3 years. · This is a guideline recommendation and does not establish individualized eligibility, exact dosing, or hospital formulary access. Toxicity and late-effects monitoring are major considerations. Confidence/conflicts: High for guideline standard-risk chemoradiotherapy statement. No conflict identified. Primary source is in Japanese. English summary pending human review — confirm exact wording with your care team.
clinical-course, clinical-finding, and imaging-based diagnosis; biopsy discussion where molecular diagnosis is needed or findings are atypical [32]Standard option (per Japan Society for Neuro-Oncology)DIPG clinical-imaging diagnosis; diffuse midline glioma correspondence where molecular confirmation/genetic analysis is required; H3 K27-altered / H3K27M context from 2025 review; Initial diagnostic workup and molecular/biopsy discussion. · Japanese-language guideline/review; human review is required before patient-facing reuse. The JSNO page reflects DIPG guideline terminology and should be reconciled with WHO 2021 H3 K27-altered terminology. Confidence/conflicts: High for Japan guideline diagnostic framework and 2025 molecular/biopsy context. No conflict identified. Primary source is in Japanese. English summary pending human review — confirm exact wording with your care team.
craniospinal irradiation, focal boost radiotherapy, proton therapy as conditional option [34]Standard option (per Japan Society for Neuro-Oncology)Risk group informed by metastasis, residual disease, histology, and molecular profile per guideline context; Postoperative radiotherapy for medulloblastoma; proton therapy consideration in radiotherapy planning. · Proton therapy is a conditional proposal, not a blanket recommendation or availability guarantee. The guideline flags limited access and low-certainty evidence for several outcomes. Confidence/conflicts: High for guideline radiation/proton statements. No conflict identified; access caveat captured. Primary source is in Japanese. English summary pending human review — confirm exact wording with your care team.
gross total resection / maximal surgical removal [34]Standard option (per Japan Society for Neuro-Oncology)Molecular subgroup/risk context includes WNT and other gene-profile subgroups in guideline discussion; histology and metastasis are also prognostic factors; Initial surgery and risk classification for pediatric/AYA medulloblastoma. · Japanese-language professional guideline; translation/human review is needed before patient-facing reuse. Extent of resection must be balanced against neurologic risk and specialist judgment. Confidence/conflicts: High for Japanese professional-guideline recommendation. No conflict identified. Japanese-language clinical content requires human review. Primary source is in Japanese. English summary pending human review — confirm exact wording with your care team.
gross total surgical resection; repeat/second-look resection context if residual disease is present [33]Standard option (per Japan Society for Neuro-Oncology)Molecular classification is described as prognostic but not yet directly determinative for treatment selection; guideline scope includes WHO grade II and III intracranial ependymoma, excluding intramedullary spinal ependymoma; Initial surgery and postoperative residual-disease assessment for pediatric/AYA intracranial ependymoma. · Japanese-language professional guideline; human review is needed before patient-facing reuse. Surgery must be balanced against neurologic function and feasibility at an experienced center. Confidence/conflicts: High for Japan guideline surgical recommendation. No conflict identified. Primary source is in Japanese. English summary pending human review — confirm exact wording with your care team.
intensified multi-agent chemotherapy; local radiotherapy or high-dose chemotherapy in selected infant/high-risk contexts; radiotherapy or palliative treatment in recurrence depending on prior therapy and response [34]Standard option (per Japan Society for Neuro-Oncology)Desmoplastic/nodular or extensive nodularity histology and metastasis context for infants; disseminated recurrence context for relapse; High-risk postoperative medulloblastoma; infants/young children; recurrent/disseminated medulloblastoma. · The guideline emphasizes uncertainty in very young children and recurrence. These are options-to-discuss, not a single standard pathway. Confidence/conflicts: High for guideline uncertainty-aware high-risk/infant/recurrent framework. No conflict identified. Primary source is in Japanese. English summary pending human review — confirm exact wording with your care team.
postoperative local radiotherapy; craniospinal irradiation when spinal dissemination is present; avoidance of CSI when spinal dissemination is absent [33]Standard option (per Japan Society for Neuro-Oncology)Postoperative status, age, histology, residual volume/site, and molecular classification are named as factors in radiotherapy decisions; Postoperative radiotherapy planning after pediatric/AYA intracranial ependymoma surgery; disseminated versus nondisseminated disease. · The guideline discusses uncertainty in some subgroups and late-effect concerns, especially in younger children. Exact field, dose, and timing must be locally planned and are not reproduced here. Confidence/conflicts: High for Japan guideline radiotherapy framework. No conflict identified. Primary source is in Japanese. English summary pending human review — confirm exact wording with your care team.
radiotherapy; conventional fractionated radiotherapy; hypofractionated radiotherapy; re-irradiation after recurrence where selected [32]Established standard of careNewly diagnosed versus post-radiotherapy recurrent DIPG; Initial DIPG radiotherapy and selected recurrent/post-radiotherapy setting. · Dose/fraction values are captured as source context, not advice. Re-irradiation evidence is weaker and requires radiation oncology review of prior dose, interval, symptoms, and risk. Confidence/conflicts: High for Japan guideline radiation framework; re-irradiation is lower-evidence per guideline. No conflict identified. Primary source is in Japanese. English summary pending human review — confirm exact wording with your care team.
repeat resection; re-irradiation; stereotactic radiosurgery in selected contexts; chemotherapy generally not recommended [33]Standard option (per Japan Society for Neuro-Oncology)Not biomarker-specific; recurrence location, prior radiation, age, and feasibility of local control are relevant; Recurrent/progressive pediatric or AYA intracranial ependymoma. · Evidence quality for recurrent ependymoma is limited, and the guideline recommendations are weak. This entry does not imply eligibility for repeat surgery, re-irradiation, or radiosurgery. Confidence/conflicts: High for guideline recurrence framework; evidence quality is limited as stated by the source. No conflict identified. Primary source is in Japanese. English summary pending human review — confirm exact wording with your care team.

Korea

KSPNO M051 regimen; KSPNO S081 protocol; alkylating-agent maintenance chemotherapy; tandem high-dose chemotherapy with autologous stem cell rescue [35]Standard option (per Korea Citation Index / Korean Cancer Association)Risk stratification context; no single biomarker eligibility stated in the fetched abstract; Korea KSPNO protocol-based therapy for newly diagnosed standard-risk and high-risk pediatric medulloblastoma. · This is peer-reviewed protocol-outcome evidence; it does not prove availability at every Korean hospital or current reimbursement. The abstract notes complications and treatment-related mortality concerns in high-risk high-dose chemotherapy contexts. Confidence/conflicts: High for abstract-level KSPNO protocol descriptions; lower for current nationwide implementation and reimbursement. No conflict identified.
craniospinal irradiation plus tumor boost; multi-agent chemotherapy [36]Standard option (per PubMed / World Journal of Pediatrics)Histologic subtype context includes classic, nodular/desmoplastic, and large cell/anaplastic in the Korean study; First-line postoperative therapy in Korean children/adolescents with average-risk medulloblastoma. · This is a retrospective/single-center Korean experience, not a national guideline or payer policy. It supports local practice context, not individualized eligibility. Confidence/conflicts: Medium for local average-risk practice because this is single-center evidence. No conflict identified.
diagnostic biopsy; immunostaining for H3K27M mutation; integrated radiologic, histopathologic, and molecular diagnosis [37]Standard option (per Brain Tumor Research and Treatment / Korean Society for Neuro-Oncology)H3K27M mutation in H3F3A or HIST1H3B/C; midline location; diffuse/infiltrating feature; EGFR hotspot mutation or EZHIP overexpression as alternative diagnostic context; Diagnostic workup for Korea adult DMG guideline population; pediatric DIPG extrapolation requires specialist review. · This is an adult KSNO DMG guideline, not a pediatric DIPG guideline. It should not override pediatric brainstem safety considerations or trial-specific requirements. Confidence/conflicts: High for adult Korea guideline diagnostic criteria; pediatric applicability is limited. No conflict identified.
multidisciplinary brain/spinal tumor clinic; neurosurgery; neuroradiology; radiation oncology; medical oncology/chemotherapy; neuroanesthesia; pain and hospice/palliative clinic involvement; development of new treatment techniques and clinical trials [38]Standard option (per Korea National Cancer Center)Not biomarker-specific; clinic-level multidisciplinary care context; Multidisciplinary evaluation and supportive-care infrastructure for CNS tumor management. · Clinic capability does not establish that a specific intervention is indicated or reimbursed for ependymoma. Clinical-trial availability is time-sensitive and requires center confirmation. Confidence/conflicts: Medium-high for clinic-level multidisciplinary and supportive-care infrastructure; not disease-specific enough to imply intervention eligibility. No conflict identified. Primary source is in Korean. English summary pending human review — confirm exact wording with your care team.
postoperative local radiotherapy; 3D conformal radiotherapy; intensity-modulated radiotherapy (IMRT); observation after complete resection in selected non-anaplastic cases; clinical-trial context for postoperative radiotherapy and chemotherapy [39]Standard option (per Journal of the Korean Medical Association / Korean Medical Association)Residual disease, anaplastic ependymoma histology, age, and local versus neuraxis dissemination context; Postoperative pediatric ependymoma radiotherapy and residual/anaplastic disease context. · This is a 2012 Korean review, not a current national guideline or regulator approval. Use as Korea-local literature context and confirm contemporary practice at the treating center. Confidence/conflicts: Medium for Korea-local ependymoma radiotherapy practice context; source is older and should be refreshed when a newer Korean guideline is found. No conflict identified. Primary source is in Korean. English summary pending human review — confirm exact wording with your care team.
proton beam therapy; proton radiotherapy for pediatric brain tumors [40]Standard option (per Journal of the Korean Medical Association / National Cancer Center Proton Therapy Center)Not biomarker-specific; pediatric brain tumor radiotherapy normal-tissue-sparing context; Radiotherapy modality consideration for selected pediatric CNS tumors, including ependymoma context requiring radiation oncology evaluation. · The proton review is from 2012 and the national cancer-information page is broad; neither states that all ependymoma patients should receive proton therapy. Access, insurance, and indication must be confirmed locally. Confidence/conflicts: Medium for Korea pediatric brain-tumor proton context; ependymoma-specific applicability must be confirmed. No conflict identified. Primary source is in Korean. English summary pending human review — confirm exact wording with your care team.
radiotherapy; 3D conformal radiation therapy; intensity-modulated radiation therapy; proton therapy as possible technique; hypofractionated radiotherapy in selected conditions; re-irradiation at progression for palliation/symptom control [37]Standard option (per Brain Tumor Research and Treatment / Korean Society for Neuro-Oncology)H3K27M-mutant DMG; gross tumor volume plus margin; T2/FLAIR abnormality inclusion; Primary/adjuvant radiotherapy where complete resection is not feasible; selected progression/re-irradiation setting. · Dose/fraction values are source context, not medical advice. Re-irradiation evidence is lower level and should be reviewed against prior dose and symptoms. Confidence/conflicts: High for adult Korea radiotherapy framework. No conflict identified.
surgery; radiotherapy; chemotherapy; hydrocephalus procedures when needed [41]Standard option (per Seoul National University Hospital)Seoul National University Hospital states medulloblastoma is now classified into SHH, WNT, Group 3, and Group 4 molecular subtypes; it also describes risk grouping by age, residual tumor size, and metastasis status; Korean tertiary-center patient-information/practice context for initial diagnosis, surgery, risk grouping, and supportive hydrocephalus management. · Korean-language hospital information source; not a national reimbursement rule. Translation/human review is needed before patient-facing reuse. Confidence/conflicts: Medium-high for Korean tertiary-center practice framing. No conflict identified. Korean-language clinical content requires human review. Primary source is in Korean. English summary pending human review — confirm exact wording with your care team.
surgery; surgery plus radiotherapy; chemotherapy by histology and extent of surgery; corticosteroids for brain edema; anticonvulsants for seizure prevention/control; cerebrospinal-fluid shunt for severe hydrocephalus [42]Standard option (per Korea National Cancer Information Center / National Cancer Center)Not ependymoma-biomarker-specific; histologic diagnosis and surgical extent guide treatment categories; General Korean pediatric brain-tumor treatment-method page applicable to the category that includes ependymoma. · This is a national cancer-information page for pediatric brain tumors, not an ependymoma-only protocol. Korean-language clinical content requires human review before patient-facing reuse. Confidence/conflicts: Medium-high for Korean national pediatric brain-tumor treatment categories and inclusion of ependymoma; lower specificity because the page is not ependymoma-only. No conflict identified. Primary source is in Korean. English summary pending human review — confirm exact wording with your care team.
temozolomide (TMZ) concurrent with radiotherapy and/or maintenance; single-agent or combination chemotherapy after multidisciplinary discussion; investigational agents such as gefitinib, ONC201, and panobinostat discussed as lacking clear benefit in the guideline [37]Studied — did not show benefitH3K27M; MGMT methylation low-incidence caveat; progression after radiotherapy; Adult Korea DMG concurrent/adjuvant systemic discussion and progression setting. · This is not a Korea regulatory approval or reimbursement claim for ONC201/dordaviprone or other agents. The guideline emphasizes lack of proven chemotherapy benefit and need for multidisciplinary decisions. Confidence/conflicts: High for Korea adult guideline systemic caveats. No conflict identified.

Australia

MRI diagnosis; biopsy to facilitate experimental therapy and clinical-trial eligibility; molecular profiling through programs such as ZERO/PRISM or ZERO2; multidisciplinary neuro-oncology review [43]Standard option (per Medical Journal of Australia / ANZCHOG CNS Tumours Group)H3K27-altered DMG; H3.3/H3.1 H3K27 alterations; EZHIP overexpression; actionable molecular target from profiling; Diagnosis, molecular profiling, trial matching, and multidisciplinary review. · The ZERO report is a research/news summary of a Nature Communications study and should not be surfaced as a guaranteed treatment pathway or outcome prediction. Biopsy and profiling require specialist review. Confidence/conflicts: High for ANZCHOG diagnostic/biopsy/trial recommendation; medium for ZERO report details pending primary-paper review. No conflict identified.
dexamethasone; bevacizumab as steroid-sparing or radiation-necrosis management option; observation after radiotherapy or clinical-trial enrollment; autopsy tumor sampling for research where feasible [43]Standard option (per Medical Journal of Australia / ANZCHOG CNS Tumours Group)Radiation necrosis/edema context; progression/relapse context; autopsy tissue donation for research context; Symptom management during/after radiotherapy, progression monitoring, and research-supportive care. · Supportive medications and research sampling are not disease-directed curative therapy. Bevacizumab use is framed for steroid sparing/radiation necrosis, not as proven antitumor therapy. Confidence/conflicts: High for ANZCHOG supportive-care framework. No conflict identified.
involved-field radiation therapy; photon or proton radiotherapy according to available center modality; re-irradiation for progressive disease; post-radiotherapy MRI baseline [43]Standard option (per Medical Journal of Australia / ANZCHOG CNS Tumours Group)Pontine tumor location; H3K27-altered DMG context; Initial radiotherapy and selected progressive disease re-irradiation. · Dose/fraction details are source context, not advice. Re-irradiation and proton/photon selection depend on local radiation oncology review. Confidence/conflicts: High for Australia/New Zealand position-statement radiotherapy guidance. No conflict identified.

China

chemotherapy regimens including vincristine, carboplatin, methotrexate, cyclophosphamide, cisplatin, etoposide; second-look surgery after chemotherapy for residual disease; supportive monitoring for toxicity [44]Standard option (per People's Medical Publishing House clinical assistant / NHC Medical Administration Bureau)Age under 18 months, WHO grade II/III, residual disease greater than 5 mm, and dissemination contexts described; no molecular drug biomarker stated; Residual disease, disseminated disease, infant radiation-delay, and pre-second-look surgery contexts. · The source includes regimen details and toxicity management; no dosing is reproduced here. Actual regimen choice and supportive care require pediatric neuro-oncology review and local formulary/access confirmation. Confidence/conflicts: High for China national-standard chemotherapy/supportive contexts; local access to individual drugs is not separately verified. No conflict identified. Primary source is in Chinese. English summary pending human review — confirm exact wording with your care team.
craniospinal radiotherapy; posterior fossa/tumor-bed boost; vincristine during radiotherapy; chemotherapy timing adjusted by age/risk [45]Standard option (per People's Medical Publishing House clinical assistant / NHC Medical Administration Bureau)Risk group and age context; molecular subtype informs risk grouping in the standard; Postoperative radiotherapy and age/risk-adapted radiotherapy timing. · The standard gives dose/range details, but this catalog intentionally omits dosing. Radiation timing and field design require pediatric radiation-oncology planning. Confidence/conflicts: High for national-standard radiotherapy timing and age/risk framework. No conflict identified. Primary source is in Chinese. English summary pending human review — confirm exact wording with your care team.
diagnosis, staging, molecular-pathology classification, brain/spine MRI, CSF cytology [44]Standard option (per People's Medical Publishing House clinical assistant / NHC Medical Administration Bureau)RELA fusion-positive, YAP1 fusion-positive, posterior fossa group A/B, NF2-mutant spinal ependymal tumors, myxopapillary ependymoma; 1q gain, H3 K27 trimethylation, and subtype prognosis contexts described; Initial diagnostic workup, staging, and treatment planning. · Chinese-language national-standard text; translation/human review is needed before patient-facing reuse. This entry does not prove availability of every molecular test at every Chinese center. Confidence/conflicts: High for China national-standard diagnostic and molecular framework. No conflict identified. Primary source is in Chinese. English summary pending human review — confirm exact wording with your care team.
dinutuximab beta (Qarziba)[46]NMPA: conditionally approvedGD2 target described in source background; Post-induction/post-myeloablative therapy/stem cell transplant with at least partial response; relapsed/refractory neuroblastoma with or without residual disease. · Fetched source is a company announcement filed with SEC, not a primary NMPA label; confidence is medium until a primary Chinese regulator or product-label source is fetched. Reimbursement and hospital availability are not established. Confidence/conflicts: Medium confidence because NMPA approval is reported in a company SEC-filed announcement rather than a primary NMPA page; no conflicting fetched source.
external-beam radiotherapy; 3D conformal radiotherapy (3D-CRT); intensity-modulated radiotherapy (IMRT); image verification such as CBCT or EPID; steroid management for radiation-related edema [47]Standard option (per People's Medical Publishing House clinical assistant / NHC Medical Administration Bureau)H3K27M-mutant DIPG; pediatric high-grade glioma; age and location affect dose adjustment; Pediatric high-grade glioma/DIPG radiation planning. · The DIPG section explicitly says mature radiotherapy/chemotherapy regimens are lacking; this record should be surfaced as a China guideline framework, not as a proven standard sequence or individual recommendation. Confidence/conflicts: High for Chinese pediatric standard radiation framework and explicit uncertainty caveat. No conflict identified. Primary source is in Chinese. English summary pending human review — confirm exact wording with your care team.
integrated histopathology and molecular diagnosis; biopsy where needed; H3K27M testing by immunohistochemistry, Sanger sequencing, or next-generation sequencing; MRI/CT/PET and molecular-pathology workup [48]Standard option (per National Health Commission of the People's Republic of China)H3K27M mutation; H3K27me3 nuclear-expression loss; EGFR hotspot mutation or EZHIP overexpression context not separately verified for China in this batch; Diagnostic classification and treatment-planning workup. · Chinese-language national/standard material; human review is required before patient-facing reuse. The pediatric standard uses 2016 WHO terminology in the fetched text, while the NHC 2022 adult glioma guideline uses the 2021 WHO framework broadly. Confidence/conflicts: High for China classification and diagnostic/molecular workup. No conflict identified beyond WHO-version terminology differences. Primary source is in Chinese. English summary pending human review — confirm exact wording with your care team.
lomustine; cisplatin; vincristine; cyclophosphamide; methotrexate; carboplatin; etoposide; high-dose chemotherapy with autologous hematopoietic stem-cell support in selected high-risk patients [45]Standard option (per People's Medical Publishing House clinical assistant / NHC Medical Administration Bureau)Risk stratification context; no single drug biomarker eligibility stated; Postoperative systemic therapy and toxicity/supportive-care monitoring by age/risk group. · The source lists regimens and monitoring contexts; actual drug availability, dosing, transplant suitability, and reimbursement require local specialist and payer review. Confidence/conflicts: High for national-standard systemic/supportive framework; local access remains unresolved. No conflict identified. Primary source is in Chinese. English summary pending human review — confirm exact wording with your care team.
maximal safe tumor resection; CSF cytology after surgery; ventriculoperitoneal shunt only when needed for unresolved hydrocephalus [44]Standard option (per People's Medical Publishing House clinical assistant / NHC Medical Administration Bureau)Not biomarker-specific; extent of resection and dissemination are key source factors; Initial surgical management and hydrocephalus/CSF dissemination assessment. · The source gives clinical-protocol details; surgical feasibility and shunt decisions depend on tumor location, neurologic risk, and center capability. Confidence/conflicts: High for China national-standard surgical/supportive framework. No conflict identified. Primary source is in Chinese. English summary pending human review — confirm exact wording with your care team.
maximal safe tumor resection; ventriculoperitoneal shunt when persistent hydrocephalus requires it [45]Standard option (per People's Medical Publishing House clinical assistant / NHC Medical Administration Bureau)Risk grouping by age, residual tumor, metastasis, histology, and molecular subtype; Initial surgery and hydrocephalus supportive management. · This is a surgical framework; individual operative risk depends on tumor location, brainstem involvement, hydrocephalus, and local pediatric neurosurgical expertise. Confidence/conflicts: High for national-standard surgery/supportive statements. No conflict identified. Primary source is in Chinese. English summary pending human review — confirm exact wording with your care team.
molecular and histologic risk stratification; staging with cranial/spinal imaging and cerebrospinal-fluid evaluation [49]Standard option (per National Health Commission of the People's Republic of China)Histologic groups; molecular groups WNT, SHH, Group 3, Group 4; CTNNB1, PTCH/SMO/SUFU, MYC/MYCN contexts described; Initial diagnostic, staging, molecular classification, and risk-stratification framework. · Chinese-language national standard mirrored by PMPH; translation/human review is needed before patient-facing reuse. The source is a standard to guide care, not individualized eligibility. Confidence/conflicts: High for China national-standard existence and risk framework. No conflict identified. Chinese-language clinical content requires human review. Primary source is in Chinese. English summary pending human review — confirm exact wording with your care team.
postoperative radiotherapy; three-dimensional conformal radiotherapy or intensity-modulated radiotherapy; spinal radiotherapy/craniospinal context only for dissemination or positive CSF cytology; avoidance of radiotherapy under age 3 where possible [44]Standard option (per People's Medical Publishing House clinical assistant / NHC Medical Administration Bureau)Disseminated lesions or positive CSF cytology influence spinal irradiation; no drug biomarker stated; Postoperative radiotherapy planning by age and dissemination status. · Exact dose and field details are intentionally not reproduced here. Pediatric late effects and dissemination status must be reviewed by radiation oncology. Confidence/conflicts: High for China national-standard radiotherapy framework. No conflict identified. Primary source is in Chinese. English summary pending human review — confirm exact wording with your care team.
temozolomide (TMZ); lomustine (CCNU); cisplatin; etoposide; vincristine; ifosfamide; PCV components procarbazine/lomustine/vincristine; drug clinical trials; individualized rehabilitation including physical, occupational, speech/swallowing, cognitive/behavioral, psychological support, and selected traditional Chinese medicine supportive approaches [47]Standard option (per People's Medical Publishing House clinical assistant / NHC Medical Administration Bureau)Molecular-pathology result may guide clinical-trial participation and potential targeted-drug selection; pediatric HGG/DIPG genetic subgroups; Pediatric high-grade glioma/DIPG systemic therapy discussion, clinical-trial consideration, and rehabilitation/supportive care. · This is not evidence that any regimen is approved, reimbursed, or beneficial for an individual DIPG case. The source explicitly says there is no mature radiotherapy/chemotherapy regimen and no standard chemotherapy regimen for pediatric high-grade glioma. Confidence/conflicts: High for China guideline caveats and listed systemic/supportive options; individual drug access and trial availability remain unverified. No conflict identified. Primary source is in Chinese. English summary pending human review — confirm exact wording with your care team.
tumor biopsy; maximal safe surgery only where anatomically feasible; hydrocephalus management with external ventricular drainage, ventriculoperitoneal shunt, Ommaya reservoir, or endoscopic third ventriculostomy; postoperative MRI/CT baseline; steroids and anticonvulsants/supportive postoperative care where indicated [47]Standard option (per People's Medical Publishing House clinical assistant / NHC Medical Administration Bureau)H3K27M mutation; brainstem/midline location; obstructive hydrocephalus and unresectable/biopsy contexts; Initial diagnosis and neurosurgical decision-making for pediatric DIPG/DMG. · This is not a recommendation for tumor-removal surgery in typical DIPG; the source explicitly cautions against routine resection and emphasizes biopsy/molecular diagnosis and supportive neurosurgical goals. Confidence/conflicts: High for China pediatric surgical/biopsy framework. No conflict identified. Primary source is in Chinese. English summary pending human review — confirm exact wording with your care team.

Russia

Molecular diagnosis, MRI/PET-CT planning, neuraxis MRI surveillance; no brand [50]Standard option (per RUSSCO / Russian Society of Clinical Oncology)H3 K27 alteration; RUSSCO also lists H3 p.K28/K27, EGFR, and EZHIP in the pediatric diffuse high-grade glioma molecular-profile table.; Initial classification, staging, and follow-up planning for diffuse midline glioma / H3 K27-altered high-grade diffuse glioma. · Russian-language guideline; human review is needed before patient-facing reuse. The fetched Russian guideline does not establish Russian approval, reimbursement, or routine access for dordaviprone/ONC201. Confidence/conflicts: High for classification/staging framework; no Russian dordaviprone availability source was verified. Primary source is in Russian. English summary pending human review — confirm exact wording with your care team.
Molecular-pathology classification and neuraxis staging; no brand [50]Standard option (per RUSSCO / Russian Society of Clinical Oncology)RUSSCO 2025 lists supratentorial ependymoma with ZFTA fusion-positive or YAP1 fusion-positive biology, posterior fossa ependymoma PFA/PFB, spinal ependymoma with MYCN amplification, subependymoma, and myxopapillary ependymoma.; Initial diagnosis, postoperative staging, and treatment planning for ependymoma. · Russian-language professional guideline; human review is needed before patient-facing reuse. This source does not establish access to every molecular test at every Russian center. Confidence/conflicts: High for guideline classification and staging framework. No conflict recorded in fetched sources. Primary source is in Russian. English summary pending human review — confirm exact wording with your care team.
Molecular/pathology workup and staging; no brand [50]Standard option (per RUSSCO / Russian Society of Clinical Oncology)RUSSCO lists WNT-activated, SHH-activated, and non-WNT/non-SHH medulloblastoma groups; the source also states TP53, PTCH, SUFU, GLI2, MYC, and MYCN testing contexts for selected groups.; Initial diagnosis, staging, and treatment planning for medulloblastoma. · Russian-language clinical guideline; human review is needed before patient-facing reuse. The source does not establish availability of every molecular test at every Russian center or payer coverage. Confidence/conflicts: High for Russian guideline classification/workup text. No conflict recorded in fetched sources. Primary source is in Russian. English summary pending human review — confirm exact wording with your care team.
Repeat surgery; repeat local radiotherapy; stereotactic radiosurgery for inoperable local recurrence or leptomeningeal metastasis context; temozolomide plus lapatinib in selected post-repeat-RT/no-repeat-surgery-and-RT context [50]Standard option (per RUSSCO / Russian Society of Clinical Oncology)Recurrent/progressive ependymoma after prior local therapy. · This is guideline-level recurrence framework, not proof that lapatinib or temozolomide is reimbursed or routinely accessible for this indication in Russia. Direct regulator/procurement sources remain gaps. Confidence/conflicts: High for guideline recurrence framework; low for product-specific access because no Russian regulator/procurement source was fetched. Primary source is in Russian. English summary pending human review — confirm exact wording with your care team.
Surgery; craniospinal radiation / radiotherapy; chemotherapy regimens including cisplatin + etoposide + cyclophosphamide, cisplatin + etoposide + ifosfamide, cisplatin/carboplatin + lomustine + vincristine, temozolomide-containing regimens, carboplatin + etoposide; vismodegib for SHH-context residual disease as guideline-mentioned option [50]Standard option (per RUSSCO / Russian Society of Clinical Oncology)SHH molecular group is specifically mentioned for possible vismodegib context after residual disease; otherwise risk/stage context is clinical and molecular.; Postoperative medulloblastoma treatment; metastatic M2-M3 pre-radiation chemotherapy context; residual-disease context after completion of chemotherapy. · The source is Russian-language and uses detailed protocol dosing that should not be surfaced directly without clinical review. Drug-by-drug Russian regulator status, procurement, pediatric access, and center capability remain separate source gaps. Confidence/conflicts: High for guideline-described modalities and regimen classes; access confidence is limited because no GRLS/procurement source was fetched for each medicine in this batch. Primary source is in Russian. English summary pending human review — confirm exact wording with your care team.
Surgical resection; repeat neurosurgical consultation for residual tumor; local radiotherapy; craniospinal irradiation if metastatic; observation after total resection for subependymoma/myxopapillary ependymoma without CSF-pathway spread [50]Standard option (per RUSSCO / Russian Society of Clinical Oncology)ZFTA/YAP1, PFA/PFB, MYCN amplification context from guideline classification; treatment primarily driven by location, metastasis, and extent of resection.; Initial/postoperative ependymoma management; metastatic versus nonmetastatic postoperative radiation planning; observation after total resection in selected low-grade entities. · The source includes specific radiation doses; those should remain clinician-facing and should not be surfaced as dosing instructions. Local surgical/radiotherapy capability and payer coverage are not established by this record. Confidence/conflicts: High for Russian guideline modality framework; no routine-access or center-capability claim is made. Primary source is in Russian. English summary pending human review — confirm exact wording with your care team.

Thailand

MRI brain and whole spine; CSF cytology; delayed lumbar puncture after surgery when obstructive hydrocephalus creates herniation risk [51]Standard option (per Chulalongkorn University / Chulalongkorn cancer education material)Not biomarker-specific; intracranial, posterior fossa/fourth-ventricle, spinal, leptomeningeal-seeding, CSF cytology, and whole-CNS imaging contexts; Diagnostic staging and treatment-planning workup before definitive treatment. · Thai-language academic material; human review is required before patient-facing use. This is not a Thai FDA or NHSO reimbursement decision. Confidence/conflicts: Medium-high for Thailand academic staging/workup statements; not a national reimbursement or regulator claim. No conflict identified. Primary source is in Thai. English summary pending human review — confirm exact wording with your care team.
MRI brain; selective spine MRI and CSF cytology when symptoms suggest spinal or leptomeningeal spread; biopsy only when clinical course or MRI is atypical [52]Standard option (per Chulalongkorn Hospital / Journal of Thai Association of Radiation Oncology)K27M-H3 / H3F3A histone mutation context; TP53 and growth-factor receptor mutation context described as research/biology; spinal/leptomeningeal spread context; Diagnostic workup and molecular/biopsy discussion for pediatric DIPG. · Thai-language professional review; human review required. The source predates WHO 2021 H3 K27-altered terminology, so terminology should be reconciled before patient-facing use. Confidence/conflicts: Medium-high for Thailand professional DIPG diagnostic context; terminology is older. No conflict identified. Primary source is in Thai. English summary pending human review — confirm exact wording with your care team.
Maximal safe resection; craniospinal irradiation with boost fields; chemotherapy including vincristine during radiotherapy and adjuvant cyclophosphamide/vincristine alternating with carboplatin/etoposide; infant neoadjuvant chemotherapy to delay radiotherapy [53]Standard option (per PLOS ONE via PubMed Central)Thai study used simplified molecular subtyping into WNT, SHH, and non-WNT/non-SHH in available tumor tissue; WHO 2021 molecular classification context is discussed.; Pediatric medulloblastoma treated at a Thai tertiary center from 2006-2018; standard-risk, high-risk, and infant contexts described. · Peer-reviewed single-center Thai cohort/protocol report; it is not a Thai FDA drug label, national payer policy, or proof that the same protocol is available at every Thai hospital. The article reports dose details, but dosing should not be reused in patient-facing content. Confidence/conflicts: Medium-high for the ThaiPOG protocol as reported by the Thai academic source; no national regulator or payer source was verified in this batch.
chemotherapy including BCNU/carmustine, CCNU/lomustine, vincristine, procarbazine, cisplatin, and other agents in pediatric brain-tumor context; chemotherapy before second-look surgery; delay/avoidance of radiotherapy in young children where possible; proton therapy as a normal-tissue-sparing modality [51]Standard option (per Chulalongkorn University / Chulalongkorn cancer education material)Young-child radiation-delay context; residual tumor after initially bulky ependymoma; tumor type and spread determine radiation volume; Pediatric CNS tumor systemic/supportive context; residual ependymoma pre-second-look context; young-child radiotherapy late-effects mitigation. · The chemotherapy statements are pediatric CNS tumor-level and not an ependymoma drug-approval claim. Specific regimen choice, drug access, and reimbursement require Thai oncology-team and payer confirmation. Confidence/conflicts: Medium for Thailand pediatric CNS chemotherapy/radiotherapy-supportive context; ependymoma-specific chemotherapy role remains limited to residual/second-look context in the source. No conflict identified. Primary source is in Thai. English summary pending human review — confirm exact wording with your care team.
chemotherapy/systemic therapies such as lomustine (CCNU), vincristine, cisplatin, cyclophosphamide, etoposide, thiotepa, temozolomide, topotecan, thalidomide, carboplatin, methotrexate studied with radiotherapy; nimotuzumab plus vinorelbine studied in small cohorts; targeted therapy research against EGFR, PDGFRA, VEGF, mTOR, farnesyl transferase, integrins, histone deacetylase; immunotherapy vaccine research; steroids and selected re-irradiation for recurrence [52]Studied — did not show benefitK27M-H3 and targeted/immunotherapy research context; recurrence after radiotherapy; local relapse context; Systemic/targeted/immunotherapy research context and recurrent DIPG symptom/re-irradiation discussion. · This is a professional review summarizing research, not Thailand approval, reimbursement, or availability for any named systemic agent. Do not present these as routine options without a trial/access source. Confidence/conflicts: High for Thai professional caveats on systemic therapy research and recurrence context; no specific Thailand access claim is made. No conflict identified. Primary source is in Thai. English summary pending human review — confirm exact wording with your care team.
no routine tumor-removal surgery; steroids for symptom relief; cerebrospinal-fluid shunt when raised intracranial pressure is present; multidisciplinary pediatric brain-tumor planning [52]Standard option (per Chulalongkorn Hospital / Journal of Thai Association of Radiation Oncology)Brainstem/pontine diffuse infiltrating tumor; raised intracranial pressure context; Initial symptom management, neurosurgical limitation, and multidisciplinary planning. · This is not a prohibition on all procedures; the source distinguishes typical DIPG from atypical cases where biopsy may be needed. Supportive measures do not imply antitumor efficacy. Confidence/conflicts: High for Thailand professional surgery/supportive caveats. No conflict identified. Primary source is in Thai. English summary pending human review — confirm exact wording with your care team.
postoperative radiotherapy; local-field radiotherapy; 3D conformal radiotherapy (3D-CRT); craniospinal irradiation (CSI) with boost for leptomeningeal seeding [51]Standard option (per Chulalongkorn University / Chulalongkorn cancer education material)Residual tumor; leptomeningeal seeding; spinal cord ependymoma with complete resection; supratentorial ependymoma with wide-margin resection contexts; Postoperative pediatric ependymoma radiotherapy and disseminated/leptomeningeal disease context. · Exact dose details are not reproduced here. Radiotherapy decisions must consider age, anatomy, prior treatment, dissemination, and center capability. Confidence/conflicts: Medium-high for Thailand academic radiation context; not a national payer/regulator claim. No conflict identified. Primary source is in Thai. English summary pending human review — confirm exact wording with your care team.
radiotherapy; CT simulation with MRI co-registration or MRI simulation; IMRT; VMAT; 2D or 3D conformal radiotherapy when faster start is needed; conventional fractionation; hypofractionated/accelerated radiotherapy as studied alternatives; proton therapy caveat [52]Standard option (per Chulalongkorn Hospital / Journal of Thai Association of Radiation Oncology)Diffuse pontine/brainstem tumor; treatment urgency and target-volume context; Initial radiotherapy for pediatric DIPG and selected alternate fractionation discussions. · Dose/fraction details are recorded as source context, not advice. The proton statement is a caution from this Thai review and should be checked against current local technology/access before patient-facing display. Confidence/conflicts: High for Thai professional radiotherapy framework. No conflict identified. Primary source is in Thai. English summary pending human review — confirm exact wording with your care team.
surgery; chemotherapy under ThaiPOG-BT-13IFB including cyclophosphamide, vincristine, high-dose methotrexate, carboplatin, and etoposide; radiotherapy delayed or avoided when possible [54]ApprovedAverage-risk and high-risk definitions include gross total removal/M0 and residual/metastatic disease categories; no molecular subgroup stated; NHSO-covered infant/under-3 brain-tumor protocol including medulloblastoma. · The chapter covers multiple infant brain tumors, not medulloblastoma only. Thai-language protocol requires human clinical review and local specialist interpretation. Confidence/conflicts: High for NHSO under-3 brain-tumor protocol coverage; medulloblastoma is one included tumor type, so disease-specific application requires team review. No conflict identified. Primary source is in Thai. English summary pending human review — confirm exact wording with your care team.
surgery; maximal resection; second-look surgery after stabilization or after chemotherapy when residual tumor remains; careful surgery near brainstem respiratory/swallowing centers [55]Standard option (per Mahidol University / Ramathibodi Department of Surgery)Residual disease after initial bulky tumor; anatomic proximity to brainstem/fourth-ventricle floor; local-invasive tumor context; Initial surgery and residual-tumor reassessment in pediatric ependymoma. · Thai-language academic/teaching sources; surgical feasibility depends on tumor anatomy and neurosurgical risk. No individual eligibility is implied. Confidence/conflicts: Medium-high for Thailand academic surgery context. No conflict identified. Primary source is in Thai. English summary pending human review — confirm exact wording with your care team.
tumor surgery; craniospinal irradiation with posterior-fossa/tumor-bed boost; vincristine with radiotherapy; post-radiotherapy chemotherapy including cyclophosphamide, vincristine, mesna, filgrastim/G-CSF, carboplatin, and etoposide [54]ApprovedAverage-risk defined in NHSO protocol by gross total removal and no metastatic disease (M0); not molecular-subgroup specific; NHSO-covered average-risk pediatric medulloblastoma treatment protocol. · Thai-language payer/protocol source; human clinical translation review is needed. The entry records NHSO protocol components, not individual eligibility, dosing, or site-level availability. Confidence/conflicts: High for NHSO average-risk protocol coverage; translation/human review needed. No conflict identified. Primary source is in Thai. English summary pending human review — confirm exact wording with your care team.

Canada

MRI-based diagnosis; biopsy when needed for mutation testing or diagnosis; supportive multidisciplinary care [56]Standard option (per Canadian Cancer Society)Histone mutation biology noted by SickKids/AboutKidsHealth; gene-mutation testing may be considered through biopsy for targeted-therapy planning; Diagnosis, mutation testing, and treatment planning. · These are Canadian cancer-information and SickKids educational sources, not Health Canada product authorization or a provincial funding rule. Biopsy is framed as conditional and specialist-led. Confidence/conflicts: High for Canadian diagnostic/biopsy framing. No conflict identified.
clinical-trial enrollment for recurrent diffuse brain stem glioma; steroids; shunt for hydrocephalus symptoms; symptom-focused palliative/supportive care [56]Established standard of careHydrocephalus context; progression/recurrence context; Initial symptom management, recurrent/progressive DIPG, hydrocephalus support, and clinical-trial discussion. · Steroids and shunting are supportive measures, not disease-directed curative treatment. Trial availability is center-, province-, age-, biomarker-, and date-specific. Confidence/conflicts: High for supportive/palliative and clinical-trial framing from Canadian sources. No conflict identified.
external beam radiation therapy; repeat radiation therapy for selected recurrence [56]Standard option (per Canadian Cancer Society)Diffuse pontine location; age and NF1 caveats for radiation noted by Canadian Cancer Society; Initial DIPG radiotherapy and selected recurrent DIPG re-irradiation. · Dose/fractionation is not specified in the Canadian sources used here. Canadian Cancer Society notes special caution in children younger than 3 years and generally avoids radiation in NF1-associated tumors where other treatments may work better. Confidence/conflicts: High for radiation as the Canadian source-backed main treatment. No conflict identified.
temozolomide (Temodal); lomustine (CeeNU, CCNU); carboplatin; vincristine; bevacizumab (Avastin and biosimilars); dabrafenib (Tafinlar); trametinib (Mekinist)[56]Standard option (per Canadian Cancer Society)Mutation or alteration identified through biopsy; source-named targeted agents include bevacizumab, dabrafenib, and trametinib; Initial/adjunct treatment discussion, very young children where radiation delay is considered, clinical-trial use, molecularly selected targeted therapy, and later/progressive disease symptom-oriented chemotherapy. · Drug names are Canadian source-described options for brain stem gliomas and not proof of DIPG-specific Health Canada approval, funding, or eligibility. SickKids explicitly cautions that chemotherapy is uncertain/not routine for DIPG. Confidence/conflicts: Medium-high. Canadian Cancer Society lists chemotherapy/targeted therapy options broadly for brain stem gliomas, while SickKids gives a narrower DIPG-specific caveat that chemotherapy is uncertain and targeted agents only fit a minority; record both with attribution.

Sources

NCI PDQ — Neuroblastoma Treatment (Health Professional) · NCI PDQ
FDA review — Unituxin (dinutuximab) BLA 125516 · FDA regulator review document
EMA EPAR — Qarziba (dinutuximab beta) · EMA EPAR
NICE TA538 · NICE health technology appraisal
FDA — approves larotrectinib for solid tumors with NTRK gene fusions · FDA regulator approval notice
NCI — FDA Approves Entrectinib for Tumors with NTRK Fusions · NCI explainer of FDA approval
FDA — accelerated approval of repotrectinib for NTRK gene fusion-positive solid tumors · FDA regulator approval notice
EMA EPAR — Vitrakvi (larotrectinib) · EMA EPAR
NCI PDQ — Childhood CNS Embryonal Tumors (incl. Medulloblastoma) Treatment · NCI PDQ
SIOP Europe — Medulloblastoma standard clinical practice (ESCP) · SIOP Europe standard clinical practice
Cancer Research UK — Medulloblastoma (children's brain tumours) · Cancer Research UK patient guidance
NCI PDQ — Ewing Sarcoma Treatment (Health Professional) · NCI PDQ
NCI PDQ — Childhood Rhabdomyosarcoma Treatment (Health Professional) · NCI PDQ
NCI PDQ — Wilms Tumor and Other Childhood Kidney Tumors Treatment · NCI PDQ
National Cancer Institute — national cancer agency evidence summary · national cancer agency evidence summary
NCI PDQ via NCBI Bookshelf — national cancer agency patient evidence summary · national cancer agency patient evidence summary
National Cancer Institute (NCI) — national cancer agency evidence summary · national cancer agency evidence summary
U.S. Food and Drug Administration — regulator approval notice · regulator approval notice
U.S. Food and Drug Administration — regulator approval notice · regulator approval notice
AWMF / Gesellschaft für Pädiatrische Onkologie und Hämatologie (GPOH) — German S1 medulloblastoma guideline PDF · German S1 medulloblastoma guideline PDF
SIOP Europe — European standard clinical practice recommendation PDF · European standard clinical practice recommendation PDF
SIOP Europe — European standard clinical practice recommendation PDF · European standard clinical practice recommendation PDF
European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
SIOP Europe — European pediatric oncology standard clinical practice document · European pediatric oncology standard clinical practice document
SIOP Europe High-Grade Glioma Working Group — European pediatric high-grade glioma standard clinical practice PDF · European pediatric high-grade glioma standard clinical practice PDF
AWMF / GPOH and German pediatric neuro-oncology contributors — German S1 pediatric/adolescent ependymoma guideline PDF · German S1 pediatric/adolescent ependymoma guideline PDF
Haute Autorite de Sante (HAS) — Transparency Committee medicine reimbursement opinion PDF · Transparency Committee medicine reimbursement opinion PDF
Institut Curie — French cancer-center proton therapy service page · French cancer-center proton therapy service page
British Journal of Cancer / UK sarcoma guideline authors — clinical guideline article · clinical guideline article
NICE — cancer service guidance PDF · cancer service guidance PDF
Cancer Research UK — cancer charity patient treatment guidance · cancer charity patient treatment guidance
Japan Society for Neuro-Oncology (JSNO) — Japanese DIPG clinical practice guideline webpage · Japanese DIPG clinical practice guideline webpage
Japan Society for Neuro-Oncology (JSNO) — Japanese pediatric/AYA ependymoma guideline webpage · Japanese pediatric/AYA ependymoma guideline webpage
Japan Society for Neuro-Oncology (JSNO) — Japanese medulloblastoma clinical guideline webpage · Japanese medulloblastoma clinical guideline webpage
Korea Citation Index / Korean Cancer Association — peer-reviewed article metadata and abstract · peer-reviewed article metadata and abstract
PubMed / World Journal of Pediatrics — peer-reviewed abstract for Korean single-center study · peer-reviewed abstract for Korean single-center study
Brain Tumor Research and Treatment / Korean Society for Neuro-Oncology — KSNO adult diffuse midline glioma guideline article · KSNO adult diffuse midline glioma guideline article
Korea National Cancer Center — National Cancer Center brain/spinal tumor clinic page · National Cancer Center brain/spinal tumor clinic page
Journal of the Korean Medical Association / Korean Medical Association — peer-reviewed Korean review PDF on pediatric brain-tumor radiotherapy · peer-reviewed Korean review PDF on pediatric brain-tumor radiotherapy
Journal of the Korean Medical Association / National Cancer Center Proton Therapy Center — peer-reviewed Korean review PDF on proton therapy in pediatric brain tumors · peer-reviewed Korean review PDF on proton therapy in pediatric brain tumors
Seoul National University Hospital — Korean academic hospital medical-information page · Korean academic hospital medical-information page
Korea National Cancer Information Center / National Cancer Center — national pediatric brain-tumor treatment information page · national pediatric brain-tumor treatment information page
Medical Journal of Australia / ANZCHOG CNS Tumours Group — Australia-New Zealand DIPG position statement · Australia-New Zealand DIPG position statement
People's Medical Publishing House clinical assistant / NHC Medical Administration Bureau — mirrored full text of China pediatric ependymal tumor diagnosis-and-treatment standard · mirrored full text of China pediatric ependymal tumor diagnosis-and-treatment standard
People's Medical Publishing House clinical assistant / NHC Medical Administration Bureau — mirrored full text of national pediatric medulloblastoma standard · mirrored full text of national pediatric medulloblastoma standard
BeiGene / EUSA Pharma via SEC filing — manufacturer regulator-approval announcement · manufacturer regulator-approval announcement
People's Medical Publishing House clinical assistant / NHC Medical Administration Bureau — mirrored full text of China pediatric glioma diagnosis-and-treatment standard · mirrored full text of China pediatric glioma diagnosis-and-treatment standard
National Health Commission of the People's Republic of China — China glioma diagnosis-and-treatment guideline PDF · China glioma diagnosis-and-treatment guideline PDF
National Health Commission of the People's Republic of China — official notice issuing 2021 pediatric tumor standards · official notice issuing 2021 pediatric tumor standards
RUSSCO / Russian Society of Clinical Oncology — professional oncology guideline PDF · professional oncology guideline PDF
Chulalongkorn University / Chulalongkorn cancer education material — Thai academic pediatric cancer/CNS tumor radiotherapy PDF · Thai academic pediatric cancer/CNS tumor radiotherapy PDF
Chulalongkorn Hospital / Journal of Thai Association of Radiation Oncology — Thai professional review PDF on pediatric DIPG · Thai professional review PDF on pediatric DIPG
PLOS ONE via PubMed Central — peer-reviewed clinical outcomes article · peer-reviewed clinical outcomes article
Thailand National Health Security Office (NHSO) — Thai pediatric-cancer public-service reimbursement guideline PDF · Thai pediatric-cancer public-service reimbursement guideline PDF
Mahidol University / Ramathibodi Department of Surgery — Thai neurosurgery educational PDF on brain tumors · Thai neurosurgery educational PDF on brain tumors
Canadian Cancer Society — Canadian childhood brain stem glioma treatment information · Canadian childhood brain stem glioma treatment information

This is official regulatory and access status only — not medical advice, not a recommendation, and not a statement about eligibility. Whether any option fits depends on your situation and your oncology team. Status changes over time; confirm the current position with the linked source. Some options shown have accelerated or conditional approval, which can be narrowed or withdrawn — reconfirm at the source. Last checked June 2026.

Beyond approved care

In clinical trials & emerging options

Options that are not — or not yet — an approved standard where you live: studies, clinical trials, off-label use, and early evidence that your own oncologist may not raise. Each is labeled by how strong the evidence is. A listing here is information to research and discuss with your team; it does not mean a treatment is proven, safe for you, or available today.

In clinical trials

GAIA-102 (natural-killer-cell therapy), alone or with dinutuximabClinical trial · NCT05608148Clinical trialIn clinical trials (NCT05608148)Japan · refractory or relapsed neuroblastoma and other pediatric solid tumours · Recruiting (Japan sites) as registered; investigational only — not an approval. PMDA authorisation not established. ClinicalTrials.gov — NCT05608148
Metronomic chemotherapy (MetroWilms-1906)Clinical trial · NCT05384821Clinical trialIn clinical trials (NCT05384821)relapsed/refractory Wilms tumor · Recruiting (France) as registered; investigational only — not an approval. ClinicalTrials.gov — NCT05384821
Complete surgical resection, delayed resection after neoadjuvant chemotherapy, orthotopic liver transplant, cisplatin-based chemotherapy, doxorubicin, vincristine, fluorouracil, irinotecan, TACE/TARE in selected unresectable contexts, radiation therapy in selected contexts, clinical trialsClinical trial · NCT00980460Clinical trialTrial only (registry)United States · PRETEXT/POSTTEXT group, AFP level, metastatic status, vascular/portal/extrahepatic/multifocal/rupture annotation factors, histology including well-differentiated fetal and small-cell undifferentiated elements, and risk group; Newly diagnosed resectable, unresectable/not resected at diagnosis, metastatic, and progressive/recurrent hepatoblastoma contexts. · NCI states surgical approach depends on PRETEXT/POSTTEXT, tumor size, multifocal disease, vascular involvement, AFP, preoperative chemotherapy response, and transplant criteria. Treatment should not be generalized without risk-group and surgical review. Confidence/conflicts: High for U.S. framework and registry-listed trial components; no FDA anticancer approval claim is made. trial-registry listing only — does not establish approval, reimbursement, or eligibility ClinicalTrials.gov — clinical-trial registry
ET140203 T cells; IL-15/IL-21 armored GPC3-CAR T cells (21.15.GPC3-CAR T cells)Clinical trial · NCT04634357Clinical trialTrial only (registry)United States · AFP-positive/HLA-A2-positive for ET140203 T cells; GPC3-positive expression for armored GPC3-CAR T cells; relapsed/refractory disease context; Relapsed/refractory pediatric hepatoblastoma for ET140203; adult GPC3-positive solid tumors including hepatoblastoma for GPC3-CAR T procurement/treatment context. · Trial eligibility is narrow and biomarker/HLA dependent. The adult GPC3-CAR T record is not pediatric hepatoblastoma-specific despite listing hepatoblastoma among eligible solid tumors. Confidence/conflicts: High for registry-listed trial and biomarker contexts; no approval or efficacy claim is made. ClinicalTrials.gov — clinical-trial registry
Intravitreal melphalan plus carboplatin, vincristine, etoposide and examinations/imaging; combination chemotherapy with carboplatin, cisplatin, cyclophosphamide, etoposide, thiotepa, vincristine, filgrastim, autologous stem cell transplant and/or radiation; GPC2 CAR T cellsClinical trial · NCT05504291Clinical trialTrial only (registry)United States · Group D vitreous seeding context for intravitreal melphalan trial; extraocular retinoblastoma context for intensive multimodality therapy; metastatic retinoblastoma context for GPC2 CAR-T; Newly diagnosed localized intraocular retinoblastoma with Group D/vitreous-seeding features; extraocular retinoblastoma; metastatic retinoblastoma. · Trial status differs by record, including completed and recruiting. GPC2 CAR-T is early-phase and metastatic/advanced context only. Confidence/conflicts: High for registry-listed settings and interventions; no approval or superiority claim is made. ClinicalTrials.gov — clinical-trial registry
Multimodal chemotherapy, surgery, radiation therapy; cisplatin, taxanes, alkylating agents; BET bromodomain inhibitors under clinical evaluation including molibresib/birabresib evidence context and ZEN003694 combinations with chemotherapy or abemaciclib trial contextsClinical trialClinical trialInvestigationalUnited States · NUTM1 rearrangement, most often BRD4::NUTM1; other partners include BRD3 or NSD3 in the fetched PDQ molecular-features section; Childhood NUT carcinoma; metastatic or unresectable NUT carcinoma in clinical-trial examples; multimodal treatment context. · PDQ states early response to chemotherapy can occur but tumor progression occurs early; BET inhibitor activity has been limited in reported trials. Treatment options under clinical evaluation should remain trial-framed. Confidence/conflicts: High for pediatric NCI treatment and trial caveats; this is a pediatric PDQ and should not be overgeneralized to all adults without additional adult sources. trial-registry listing only — does not establish approval, reimbursement, or eligibility National Cancer Institute — national cancer agency pediatric evidence summary
Primary gross-total resection and adjuvant therapy within multimodal treatmentClinical trialClinical trialInvestigationalUnited States · NUT carcinoma in the PDQ registry-survival discussion; no additional predictive biomarker beyond NUTM1-rearranged disease is specified for this treatment-outcome context; Head-and-neck primary NUT carcinoma where gross-total resection is feasible, followed by adjuvant therapy. · The source describes a small retrospective registry-survival subgroup in a highly aggressive rare cancer. It should not be generalized to all anatomic sites, unresectable disease, or all-age populations. Confidence/conflicts: High for the registry-survival statement within the PDQ; low for external generalizability because the cohort is small and anatomically selective. National Cancer Institute — national cancer agency pediatric evidence summary
Risk-adapted FHWT therapy components including nephrectomy, observation, vincristine, dactinomycin, doxorubicin, cyclophosphamide, etoposide, carboplatin, irinotecan; trastuzumab deruxtecan (DS-8201a); cabozantinib / cabozantinib S-malateClinical trial · NCT06401330Clinical trialTrial only (NCT06401330)United States · Favorable histology, tumor biology tests, response-to-therapy risk features; HER2-positive tumor-cell targeting is described for trastuzumab deruxtecan; cabozantinib is a multi-kinase inhibitor trial context without a Wilms-specific approved biomarker in the fetched record.; NCT06401330 covers stage I-IV favorable-histology Wilms tumor; NCT04616560 covers newly diagnosed, recurrent, or refractory Wilms tumor among other tumors; NCT02867592 covers recurrent, refractory, or newly diagnosed rare tumors including Wilms tumor. · Trial status differs by record: recruiting, suspended, and active-not-recruiting. Trial listings do not establish eligibility, access outside a listed site, or clinical benefit. Confidence/conflicts: High for registry-listed interventions and statuses; no approval claim is made. ClinicalTrials.gov — clinical-trial registry
Cisplatin, doxorubicin, carboplatin, fluorouracil, vincristine, etoposide, irinotecan, gemcitabine, oxaliplatin, sorafenib, surgery/resection, liver transplantationClinical trial · NCT03017326Clinical trialTrial only (registry)European Union · Risk-adapted PHITT context; PRETEXT hepatoblastoma context; no country-specific approved biomarker in the registry records; Newly diagnosed hepatoblastoma in PHITT; high-risk hepatoblastoma undergoing surgical resection in the older intensive-neoadjuvant study. · PHITT is active-not-recruiting in the fetched registry record; older neoadjuvant record is unknown status and stale. Trial protocols should not be treated as current open access without site confirmation. Confidence/conflicts: High for PHITT registry geography and interventions; medium for older high-risk study current relevance due to stale unknown status. ClinicalTrials.gov — clinical-trial registry
Cobolimab plus dostarlimabClinical trial · NCT06521567Clinical trialTrial only (NCT06521567)European Union · Pediatric and young adult cancer study context including DIPG. · Active-not-recruiting status means not currently open to new participants according to the fetched registry status. This does not establish EMA approval, French reimbursement, or routine access. Confidence/conflicts: High for France registry status; no regulator approval inferred. ClinicalTrials.gov — clinical-trial registry
Dordaviprone (ONC201); lorlatinib with radiation/chemotherapy context as trial arms describeClinical trial · NCT05580562Clinical trialTrial only (NCT05580562)European Union · H3 K27M for ACTION; ROS or ALK fusion for lorlatinib study; Post-radiotherapy H3 K27M-mutant diffuse glioma for ACTION; newly diagnosed ROS/ALK-fusion high-grade glioma including DIPG/DMG context for lorlatinib trial. · These are trial records and do not establish EMA approval, German reimbursement, or routine access. The lorlatinib Germany site was not yet recruiting in the fetched registry status. Confidence/conflicts: High for registry status; no regulator approval inferred. ClinicalTrials.gov — clinical-trial registry
Metronomic vincristine, irinotecan, temozolomide, etoposide, cis-retinoic acid; upfront vincristine-actinomycin-D-doxorubicin versus vincristine-carboplatin-etoposide in stage IV childhood renal tumor trial contextClinical trial · NCT05384821Clinical trialTrial only (registry)European Union · High-risk biology characteristics in NCT03669783 include diffuse anaplasia, 1q gain, loss of heterozygosity 1p and 16q, and blastemal residual volume; no biomarker eligibility is captured for NCT05384821 in the fetched summary.; Relapsed/refractory Wilms tumor for MetroWilms-1906; stage IV childhood renal tumor / nephroblastoma context for NCT03669783. · Registry trial participation depends on site status, eligibility, and protocol criteria. The stage IV renal tumor record is broader than Wilms tumor but explicitly states nephroblastoma/Wilms tumor context. Confidence/conflicts: High for registry-listed France trial cells; no conflict recorded. ClinicalTrials.gov — clinical-trial registry
SIOP EPENDYMOME II / SIOP Ependymoma II; centralized pre/postoperative imaging review; centralized pathology review; possible reoperation after surgery and central reviewClinical trialClinical trialTrial only (registry)European Union · Central pathology review and prospective biological marker assessment; BIOMECA biomarker ancillary study referenced; Newly diagnosed children, adolescents, and young adults with ependymoma in trial-defined strata. · The INCa entry is French-language and trial eligibility is protocol-specific. Registry presence does not imply an individual can enroll or that a listed strategy is available outside trial criteria. Confidence/conflicts: High for French registry status and central-review components. No conflict identified. Primary source not in English. English summary pending human review — confirm exact wording with your care team. Institut National du Cancer (cancer.fr) — French national clinical-trial registry entry
SIOP Ependymoma II; I-HIT-MED Registry; HIT-REZ Registry; GPOH/HIT reference panels; INFORM-style molecular target search in recurrenceClinical trialClinical trialInvestigationalEuropean Union · Molecular characterization for reference review and future targeted therapy; recurrence molecular analysis; metastasis and residual disease contexts; Newly diagnosed ependymoma study enrollment, registry-supported individualized planning, residual tumor review, and recurrent/progressive disease. · Trial and registry availability, age limits, and eligibility are time-sensitive and center-specific. Registry participation is not itself treatment. Confidence/conflicts: High for Germany study/registry infrastructure and reference-review context. No conflict identified. AWMF / GPOH and German pediatric neuro-oncology contributors — German S1 pediatric/adolescent ependymoma guideline PDF
SIOP HR-Medulloblastoma study; I-HIT-MED Registry; reference review by GPOH-mandated study groupClinical trialClinical trialInvestigationalEuropean Union · High-risk and tumor-biology contexts; SHH/TP53, Group 3 MYC amplification, metastasis context in GPOH page; High-risk medulloblastoma study access, registry-based treatment support, and relapse documentation/support context. · Study eligibility, open sites, and randomization arms must be checked with the treating center/study office. Registry participation is not itself a treatment. Confidence/conflicts: High for Germany study/register infrastructure statements. No conflict identified; trial availability is time-sensitive. Primary source not in English. English summary pending human review — confirm exact wording with your care team. AWMF / Gesellschaft für Pädiatrische Onkologie und Hämatologie (GPOH) — German S1 medulloblastoma guideline PDF
SIOPEN HR-NBL2 chemotherapy/radiotherapy/dinutuximab beta regimen components; GD2 CAR T cells; naxitamab + GM-CSFClinical trial · NCT04221035Clinical trialTrial only (registry)European Union · MYCN amplification and SIOPEN-modified INRG high-risk definitions are part of the HR-NBL2 eligibility text; GD2 target context for CAR-T and naxitamab records; Newly diagnosed high-risk neuroblastoma for HR-NBL2; relapsed/refractory neuroblastoma for GD2 CAR-T; high-risk primary refractory or incomplete response to salvage treatment in bone/bone marrow for naxitamab + GM-CSF. · Registry/trial cells only; HR-NBL2 is a complex randomized protocol, GD2 CAR-T is single-center and active-not-recruiting, and naxitamab study eligibility is restricted to bone and/or bone marrow evaluable disease. Confidence/conflicts: High confidence for regional registry cells and trial statuses; no conflicting fetched source. Routine access/reimbursement not established. ClinicalTrials.gov — trial registry
Trametinib; vinblastine; selumetinib; carboplatin plus vincristine; mirdametinib; laser interstitial thermal therapy (LITT); proton radiotherapyClinical trial · NCT05180825Clinical trialTrial only (registry)European Union · BRAF wild-type and non-NF1 for French trametinib-versus-vinblastine study; recurrent/progressive low-grade glioma with MEK-pathway rationale in U.S. mirdametinib and selumetinib records; NF1-associated low-grade glioma in U.S. selumetinib-versus-CV record; Newly diagnosed non-NF1 BRAF-wild-type pLGG in France trial; recurrent/refractory or NF1-associated low-grade glioma in U.S. selumetinib records; recurrent/progressive pLGG for mirdametinib and LITT; pediatric brain tumors requiring partial-brain irradiation for proton radiotherapy. · Trial populations differ by biomarker, age, prior therapy, and local eligibility. France record is not a general trametinib approval claim. U.S. records are registry/trial cells, including some active-not-recruiting or completed records. Confidence/conflicts: High for trial-cell content; no approval claim is made for trametinib, selumetinib, mirdametinib, LITT, or proton therapy in pLGG beyond the listed trial/procedure context. ClinicalTrials.gov — clinical-trial registry
VP16/etoposide, carboplatin, vincristine, melphalan, topotecan, thermotherapy, cryotherapy, iodine-125 plaques, intravitreal melphalan, intra-arterial chemotherapy with melphalan/topotecan, observation, orbital irradiation, cyclophosphamide, thiotepa, cytapheresis, peripheral blood stem cell transplantationClinical trial · NCT02866136Clinical trialTrial only (registry)European Union · Conservative-treatment eligibility by unilateral/bilateral group, age, vitreous seeding, macular/vision threat; postoperative histopathological risk factors after primary enucleation; Newly diagnosed eyes eligible for conservative management; extensive unilateral retinoblastoma after primary enucleation by histopathological risk factors. · Conservative ocular treatment applies to selected eyes/cases and depends on protocol criteria. Stem cell transplantation appears only in higher-risk postoperative strata in the adjuvant record. Confidence/conflicts: High for France registry cells; no national reimbursement claim is made. ClinicalTrials.gov — clinical-trial registry
clinical trials where available; SIOP Europe HGG Working Group expert advice; DIPG/DMG registries; national therapy guideline tailoring; future directions in targeted therapyClinical trialClinical trialReported in a clinical trialEuropean Union · H3K27-altered DMG; targetable molecular alteration if identified by biopsy; infant-type hemispheric glioma fusions as separate biology; Newly diagnosed or recurrent pediatric HGG/DMG/DIPG where trial/registry or expert review is available. · Registry participation is not treatment. Trial availability is country-, center-, age-, biomarker-, and date-specific. Confidence/conflicts: High for SIOP Europe clinical-trial/registry infrastructure. No conflict identified. SIOP Europe High-Grade Glioma Working Group — European pediatric high-grade glioma standard clinical practice PDF
conformal radiotherapy; VEC chemotherapy plus cisplatin (vincristine, etoposide, cyclophosphamide, cisplatin); VEC chemotherapy with or without high-dose methotrexate; radiotherapy boost where protocol-defined; dose-dense chemotherapy with or without valproateClinical trialClinical trialTrial only (registry)European Union · Trial strata based on age, tumor location, surgical result/residual disease, radiotherapy eligibility, and biological marker evaluation; Protocol-defined post-surgery newly diagnosed ependymoma strata, residual-disease strategy, and radiotherapy-ineligible/infant stratum. · This is a trial-protocol entry, not a general standard-of-care or reimbursement claim. Specific drugs, arms, schedules, and eligibility must be interpreted by the treating trial team. Confidence/conflicts: High for trial arms described in the French registry and Paris trial-site confirmation. No conflict identified. Primary source not in English. English summary pending human review — confirm exact wording with your care team. Institut National du Cancer (cancer.fr) — French national clinical-trial registry entry
sequential chemotherapy including etoposide, carboplatin, melphalan, cisplatin, thiotepa; peripheral stem-cell reinjections; possible second surgery; reduced-dose craniospinal radiotherapy by ageClinical trialClinical trialTrial only (registry)European Union · Metastatic medulloblastoma at diagnosis; age under 5 years for medulloblastoma criterion; Historical high-risk/metastatic pediatric medulloblastoma trial record; not open to enrollment. · Trial is closed to inclusion; this is a registry/history cell documenting a France protocol option and not a currently enrolling option. Confidence/conflicts: High for registry facts and closed status. No conflict identified. Primary source not in English. English summary pending human review — confirm exact wording with your care team. Institut National du Cancer (cancer.fr) — French national clinical-trial registry entry
Carboplatin, cytarabine, etoposide, vincristine, clinical observation, adjuvant therapy, radiation therapyClinical trial · NCT00360750Clinical trialTrial only (registry)United Kingdom · Histologically confirmed unilateral retinoblastoma; no metastatic spread; post-enucleation risk context; Advanced unilateral retinoblastoma after primary enucleation, with no metastatic spread. · Registry status is unknown and stale. Current UK care should be confirmed through specialist retinoblastoma services rather than inferred from this record alone. Confidence/conflicts: Medium because the registry record is old and unknown status; high that it supports the UK post-enucleation adjuvant/observation cell. ClinicalTrials.gov — clinical-trial registry
Dordaviprone (ONC201) with placebo comparator in ACTION studyClinical trial · NCT05580562Clinical trialTrial only (NCT05580562)United Kingdom · H3 K27M mutation; Following frontline radiotherapy for H3 K27M-mutant diffuse glioma. · This is trial-only UK availability and does not establish MHRA approval, NHS routine funding, current slot availability at every site, or individual eligibility. Confidence/conflicts: High for registry-listed UK recruiting trial status; no UK regulator approval inferred. ClinicalTrials.gov — clinical-trial registry
Preoperative and postoperative chemotherapy with dactinomycin, carboplatin, cyclophosphamide, doxorubicin, etoposide, vincristine; surgery; radiation therapy; relapse chemotherapy followed by surgery/radiation with or without autologous bone marrow or peripheral blood stem cell transplantation; indocyanine green surgical-imaging study for partial nephrectomy contextClinical trial · NCT00047138Clinical trialTrial only (registry)United Kingdom · Newly diagnosed Wilms tumor/nephroblastoma perioperative therapy; relapsed or refractory Wilms tumor; pediatric kidney cancer surgery/partial-nephrectomy imaging context. · Two core treatment records have unknown status and old update dates; Nephrogreen is diagnostic/surgical-imaging support rather than systemic cancer therapy. Confidence/conflicts: Medium because major treatment records are stale/unknown-status; high that fetched records name Wilms tumor/nephroblastoma contexts and countries. ClinicalTrials.gov — clinical-trial registry
SIOPEN HR-NBL2 chemotherapy/radiotherapy/dinutuximab beta regimen components; GD2 CAR T cells; naxitamab + GM-CSFClinical trial · NCT04221035Clinical trialTrial only (registry)United Kingdom · MYCN amplification and SIOPEN-modified INRG high-risk definitions are part of the HR-NBL2 eligibility text; GD2 target context for CAR-T and naxitamab records; Newly diagnosed high-risk neuroblastoma for HR-NBL2; relapsed/refractory neuroblastoma for GD2 CAR-T; high-risk primary refractory or incomplete response to salvage treatment in bone/bone marrow for naxitamab + GM-CSF. · Registry/trial cells only; HR-NBL2 is a complex randomized protocol, GD2 CAR-T is single-center and active-not-recruiting, and naxitamab study eligibility is restricted to bone and/or bone marrow evaluable disease. Confidence/conflicts: High confidence for regional registry cells and trial statuses; no conflicting fetched source. Routine access/reimbursement not established. ClinicalTrials.gov — trial registry
Abemaciclib with irinotecan and temozolomide; abemaciclib combinations with irinotecan, temozolomide, dinutuximab, and GM-CSF in pediatric/young adult solid tumor contextClinical trial · NCT05440786Clinical trialTrial only (NCT05440786)Japan · No biomarker required by fetched records; Ewing sarcoma with metastatic/neoplasm metastasis context for CAMPFIRE; relapsed/refractory pediatric/young adult solid tumors for broader abemaciclib combination record. · Registry records do not establish PMDA approval, reimbursement, routine access, active enrollment beyond stated status, or individual eligibility. Confidence/conflicts: High for registry facts; no local approval claim made. ClinicalTrials.gov — clinical-trial registry
Dabrafenib (Tafinlar/Finlee) plus trametinib (Mekinist/Spexotras); comparator carboplatin plus vincristine; dabrafenib and/or trametinib rollover follow-upClinical trial · NCT02684058Clinical trialTrial only (registry)Japan · BRAF V600 mutation; BRAF V600 mutation-positive pediatric low-grade glioma needing first systemic treatment or progressive disease after surgery/non-surgical candidates in the phase II record; rollover/follow-up for patients from Novartis-sponsored parent studies. · NCT02684058 is completed; NCT03975829 is active-not-recruiting and is a rollover/follow-up study rather than a de novo treatment-access trial. Confidence/conflicts: High for registry-listed countries, trial status, and interventions; no approval claim is made for Japan or Russia. ClinicalTrials.gov — clinical-trial registry
GAIA-102 alone; GAIA-102 with dinutuximab, filgrastim, and teceleukin; GAIA-102 with nivolumab; GAIA-102 with nivolumab and teceleukinClinical trial · NCT05608148Clinical trialTrial only (registry)Japan · Refractory/relapse neuroblastoma; inclusion text includes resistance to more than two treatment regimens and resistance to all standard guideline-based regimens for some cohorts, and residual tumor after completing a dinutuximab regimen for specified cohorts. · Early clinical-trial safety/dose context; intervention descriptions in the registry are generic "Biological" entries with regimen details in descriptions. Confidence/conflicts: High confidence for Japan recruiting registry cell and disease context; no conflicting fetched source. PMDA access not established. reimbursement not established in this jurisdiction trial-registry listing only — does not establish approval, reimbursement, or eligibility ClinicalTrials.gov — trial registry
LDE225 / sonidegibClinical trial · NCT01208831Clinical trialTrial only (registry)Japan · Hedgehog pathway inhibitor context; no mutation biomarker required by fetched registry record; Advanced solid tumor/BCC study context. · Registry record does not establish PMDA approval, reimbursement, routine access, or individual eligibility. The study is completed and includes multiple diseases. Confidence/conflicts: Medium-high for Japan registry-listed BCC study activity; PMDA/access unverified. ClinicalTrials.gov — clinical-trial registry
LDE225/sonidegib; abemaciclib with irinotecan, temozolomide, dinutuximab, and GM-CSF in pediatric/young adult solid tumor study contextClinical trial · NCT01208831Clinical trialTrial only (NCT01208831)Japan · Hedgehog-pathway context for LDE225 study; no medulloblastoma-specific biomarker listed for abemaciclib combination study in fetched fields; Advanced solid tumor/medulloblastoma context for LDE225; relapsed or refractory solid tumors in children/young adults for abemaciclib combination study. · Completed trial records do not establish PMDA approval, current enrollment, routine access, reimbursement, or individual eligibility. Confidence/conflicts: High for completed registry status; no approval inference. ClinicalTrials.gov — clinical-trial registry
Vincristine, dactinomycin, cyclophosphamide with or without radiation therapy; abemaciclib with irinotecan/temozolomide-based combinations in a pediatric/young-adult solid tumor trialClinical trial · NCT00245141Clinical trialTrial only (registry)Japan · No biomarker required by fetched registry records; Embryonal RMS treatment trial context; relapsed/refractory solid-tumor pediatric/young-adult context for abemaciclib combination record. · Registry records do not establish PMDA approval, routine access, reimbursement, current local enrollment, or individual eligibility. Two older embryonal RMS records have unknown overall status despite Japan locations shown as recruiting in the fetched registry location module. Confidence/conflicts: Medium for registry-listed Japan RMS/solid-tumor trial options; low for present-day availability because key RMS-specific Japan records are old/unknown-status. ClinicalTrials.gov — clinical-trial registry
chemotherapy not recommended as routine; steroids with radiotherapy or recurrence; temozolomide, etoposide, ifosfamide/carboplatin/etoposide, panobinostat, GD2-CAR T-cell therapy, peptide vaccines, convection-enhanced delivery, ONC201/dordaviprone research/compassionate-use references; nimustine hydrochloride via convection-enhanced delivery in Japanese phase II literatureClinical trialClinical trialInvestigationalJapan · H3 K27-altered / H3K27M; H3.3 K27M testing; recurrent disease and molecularly guided investigational therapy contexts; Non-routine systemic therapy discussion, molecular/biopsy-enabled trial or investigational treatment consideration, supportive steroid context. · Do not surface named investigational approaches as available or recommended in Japan unless a trial, PMDA, or institutional access source is separately verified. The JSNO guideline recommendation is against routine chemotherapy. Confidence/conflicts: High for Japan guideline chemotherapy caveat and investigational landscape; regulatory access remains unverified. No conflict identified. Primary source is in Japanese. English summary pending human review — confirm exact wording with your care team. Japan Society for Neuro-Oncology (JSNO) — Japanese DIPG clinical practice guideline webpage
Haploidentical stem cell transplantation and NK cell therapy; palbociclib with temozolomide/irinotecan/topotecan/cyclophosphamide; zilovertamab vedotinClinical trial · NCT01807468Clinical trialTrial only (NCT01807468)Korea · No biomarker required by fetched records; High-risk solid tumors including Ewing sarcoma; pediatric recurrent/refractory solid tumors including Ewing sarcoma; pediatric/young adult solid tumor sub-study including Ewing sarcoma. · Registry records do not establish MFDS approval, reimbursement, routine access, active enrollment beyond stated status, or individual eligibility. NCT01807468 overall status is unknown. Confidence/conflicts: Medium-high for registry facts; unknown-status caveat recorded. ClinicalTrials.gov — clinical-trial registry
Haploidentical stem cell transplantation and NK-cell therapy; palbociclib with chemotherapy; ceritinib for pediatric ALK-activated malignanciesClinical trial · NCT01807468Clinical trialTrial only (registry)Korea · ALK alteration required only for the ceritinib pediatric ALK-activated tumor study; otherwise no biomarker required by fetched registry records; High-risk solid tumors; recurrent/refractory pediatric solid tumors; pediatric ALK-activated malignancy context. · Registry listing does not establish MFDS approval, reimbursement, routine RMS access, open enrollment, or eligibility. The ceritinib record requires ALK activation and is not RMS-specific in the fetched condition field. Confidence/conflicts: Medium for South Korea registry-listed investigational options; routine availability and RMS-specific eligibility remain uncertain. ClinicalTrials.gov — clinical-trial registry
Nitroglycerin versus normal saline during intra-arterial chemotherapyClinical trial · NCT04564521Clinical trialTrial only (registry)Korea · Not biomarker-selected; intra-arterial chemotherapy procedure context; Pediatric retinoblastoma patients needing at least two more intra-arterial chemotherapy sessions under general anesthesia. · The study addresses procedural safety/support around intra-arterial chemotherapy, not a direct anticancer regimen. Registry status is unknown. Confidence/conflicts: Medium because the record is supportive/procedure-focused and unknown status; high that it names retinoblastoma and intra-arterial chemotherapy. ClinicalTrials.gov — clinical-trial registry
Palbociclib with chemotherapy options including temozolomide, irinotecan, topotecan, and cyclophosphamideClinical trial · NCT03709680Clinical trialTrial only (NCT03709680)Korea · Recurrent/refractory pediatric solid tumors including medulloblastoma. · Active-not-recruiting status is not current new enrollment. This registry cell does not establish MFDS approval, reimbursement, routine access, or individual eligibility. Confidence/conflicts: High for registry-listed Korea trial status; no regulator approval inferred. ClinicalTrials.gov — clinical-trial registry
Patritumab deruxtecan / HER3-DXd (MK-1022)Clinical trial · NCT06941272Clinical trialTrial only (registry)Korea · Advanced or metastatic hepatoblastoma after at least one prior systemic treatment; HER3-directed antibody-drug conjugate mechanism per registry summary; Pediatric relapsed or refractory advanced/metastatic hepatoblastoma after prior systemic treatment and no satisfactory alternative treatment option, per registry eligibility summary. · The condition field is broad malignant neoplasm, but the registry summary and eligibility text explicitly mention hepatoblastoma. Trial status, local site activation, and individual eligibility need confirmation. Confidence/conflicts: Medium-high because hepatoblastoma is explicit in summary/eligibility but the condition field is broad; no approval claim is made. ClinicalTrials.gov — clinical-trial registry
Tovorafenib / DAY101 / Ojemda; standard-of-care chemotherapy comparator in LOGGIC/FIREFLY-2Clinical trial · NCT04775485Clinical trialTrial only (registry)Korea · Activating BRAF alteration or activating RAF alteration; exclusions include some additional molecular alterations in the phase III record; Relapsed or progressive low-grade glioma with documented known activating BRAF alteration after at least one prior systemic therapy in FIREFLY-1; first-line systemic therapy for pediatric low-grade glioma harboring an activating RAF alteration in LOGGIC/FIREFLY-2. · FIREFLY-1 excludes known or suspected NF1 in the fetched criteria excerpt; LOGGIC/FIREFLY-2 excludes known/suspected NF1/NF2 and some additional pathogenic alterations. Trial status and site availability require local confirmation. Confidence/conflicts: High for registry-listed cells and statuses; no country approval or reimbursement claim is made outside the separate U.S. FDA tovorafenib approval. ClinicalTrials.gov — clinical-trial registry
cisplatin, doxorubicin, etoposide, cyclophosphamide, ifosfamide, carboplatin, tandem high-dose chemotherapy/autologous stem cell transplantation, radiotherapy, interleukin-2, isotretinoin, MIBGClinical trial · NCT02771743Clinical trialTrial only (registry)Korea · Newly diagnosed high-risk neuroblastoma, with treatment intensity of tandem high-dose chemotherapy tailored by response to induction chemotherapy. · Registry status unknown and last updated in 2016; historical/uncertain current-access cell only. Confidence/conflicts: Medium confidence because the fetched registry supports the protocol and country, but status is unknown and stale. No conflicting fetched source. reimbursement not established in this jurisdiction trial-registry listing only — does not establish approval, reimbursement, or eligibility ClinicalTrials.gov — trial registry
chemotherapy only in clinical-trial context; radiation-sensitising agents in clinical trials; targeted therapies through clinical trials or compassionate access when a biologic rationale exists; examples referenced in ANZCHOG statement include ACT001, DFMO plus AMXT1501, avapritinib, ONC201 plus paxalisib, everolimus, sunitinib, panobinostat, vorinostat, CBL0137, nivolumab, ipilimumab, and CAR T-cell approachesClinical trialClinical trialReported in a clinical trialAustralia · Potentially actionable target identified by molecular profiling; radiation-sensitising-agent trial context; Concurrent/adjuvant systemic therapy, investigational targeted/immunotherapy/cell therapy, and compassionate-access consideration. · Named agents are research examples, not routine access or approval claims. Compassionate access requires target rationale, clinician review, and no suitable trial. Confidence/conflicts: High for clinical-trial/compassionate-access framework; medium for current ZERO outcome summary until primary article details are separately cataloged. No conflict identified. Medical Journal of Australia / ANZCHOG CNS Tumours Group — Australia-New Zealand DIPG position statement
Apatinib with temozolomide and etoposide; craniospinal radiation with temozolomide; Tomotherapy HD versus 3DCRT; becotatug vedotinClinical trial · NCT04501718Clinical trialTrial only (NCT04501718)China · EGFR-positive required for becotatug vedotin pediatric solid tumor study; no biomarker specified in fetched fields for apatinib or radiotherapy/tomotherapy studies; Recurrent pediatric medulloblastoma for apatinib-temozolomide-etoposide; medulloblastoma radiation/chemotherapy or radiotherapy technique studies; EGFR-positive pediatric relapsed/refractory/metastatic solid tumor context including medulloblastoma. · Registry records do not establish NMPA approval, routine access, reimbursement, current enrollment for all sites, or individual eligibility. Overall-status unknown records need direct site/registry confirmation. Confidence/conflicts: Medium-high; NCT02681705 and NCT03675230 had overall-status unknown despite site query returning recruiting, so current enrollment needs confirmation. ClinicalTrials.gov — clinical-trial registry
High-dose intravitreal topotecan; topotecan or melphalan for eye-sparing chemotherapy; three cycles adjuvant chemotherapy after enucleation; intrathecal melphalan for CNS metastasisClinical trial · NCT06972602Clinical trialTrial only (registry)China · RB1 mutation/active tumor criteria appear in the high-dose topotecan vitreous-seed trial; International Retinoblastoma Staging System stage I and IVB contexts appear in other records; Recurrent/refractory vitreous seeds; eyeball-sparing treatment; stage I post-enucleation high-risk retinoblastoma; stage IVB CNS metastatic retinoblastoma. · Intrathecal melphalan is recorded for CNS metastatic disease, not routine intraocular disease. Eye-sparing studies should not be interpreted as alternatives when enucleation is medically necessary. Confidence/conflicts: High for registry-listed China studies; no NMPA approval claim is made. ClinicalTrials.gov — clinical-trial registry
Luvometinib / FCN-159; investigator-choice chemotherapy including carboplatin plus vindesine, carboplatin, temozolomide; recombinant human endostatin (Endostar) combined with carboplatin and vincristineClinical trial · NCT07004075Clinical trialTrial only (registry)China · KIAA1549-BRAF fusion or BRAF V600E mutation for FCN-159/luvometinib phase III trial; biomarker not stated as required in recombinant human endostatin plus CV record; Pediatric low-grade glioma requiring systemic therapy, including recurrent/progressive, post-surgical residual, or unresectable disease in FCN-159 trial criteria; pediatric low-grade glioma with residual/evaluable lesion and no prior radiotherapy or chemotherapy in the endostatin plus CV record. · FCN-159 trial was not yet recruiting in the fetched registry record. Endostatin plus CV record is completed; current availability and protocol adoption are not established by the registry. Confidence/conflicts: High for China trial records; no NMPA approval claim is made. ClinicalTrials.gov — clinical-trial registry
Modified PHITT strategy with sodium thiosulfate, primary resection, biopsy, resection or transplant, pulmonary nodule resection, cisplatin, fluorouracil, vincristine, doxorubicin, carboplatin, irinotecan; pucotenlimab with lenvatinib and chemotherapy; PD-1/PD-L1 inhibitor with lenvatinibClinical trial · NCT04478292Clinical trialTrial only (registry)China · Modified PHITT/risk-group context; ctDNA 5-hydroxymethylcytosine monitoring context in one China study; refractory/relapsed advanced disease in immunotherapy-targeted trials; Newly diagnosed hepatoblastoma; advanced relapsed/refractory hepatoblastoma after prior systemic therapy; refractory hepatoblastoma after chemotherapy. · Trial status varies, including unknown status for the PD-1/PD-L1 plus lenvatinib cohort. Pucotenlimab/lenvatinib/chemotherapy and PD-1/PD-L1 combinations remain trial-framed. Confidence/conflicts: High for registry-listed China trials and statuses; no NMPA approval claim is made. ClinicalTrials.gov — clinical-trial registry
Risk-stratified VAC/VAC-VII/CAV-IE chemotherapy; becotatug vedotin; albumin-paclitaxel with ifosfamide/cisplatin rare-tumor regimen; radiotherapy plus apatinib; VMAT chemoradiotherapy-surgery sequence; anlotinib-based maintenance or combination approaches; pazopanib with chemotherapy; all-trans retinoic acid with VACClinical trial · NCT06836492Clinical trialTrial only (registry)China · EGFR-positive disease required only for the becotatug vedotin pediatric solid-tumor trial; Pediatric RMS risk-stratified treatment; pediatric relapsed/refractory or metastatic solid tumors; recurrent/refractory RMS; intermediate-to-high-risk RMS; adult nasal/paranasal sinus RMS. · Registry records do not establish NMPA approval for RMS, reimbursement, routine access, or eligibility. Several records are recruiting or not-yet-recruiting; one radiotherapy/apatinib record has unknown overall status while the location module lists recruiting. Confidence/conflicts: High for existence of China registry-listed RMS trials; not an approval or access finding. ClinicalTrials.gov — clinical-trial registry
Vincristine plus temozolomide; cyclophosphamide/doxorubicin/vincristine/ifosfamide/etoposide risk-stratified chemotherapy; irinotecan liposome plus temozolomide plus fluzoparib; sarcoma-specific CAR-T cells; TK216 plus vincristineClinical trial · NCT03359005Clinical trialTrial only (NCT03359005)China · No biomarker required by fetched records; Ewing sarcoma; pediatric risk-stratified Ewing; recurrent/metastatic Ewing; relapsed/refractory Ewing. · Registry records do not establish NMPA approval, reimbursement, routine access, active enrollment beyond stated status, or individual eligibility. Some records have unknown overall status. Confidence/conflicts: Medium-high for registry facts; unknown-status caveats recorded. ClinicalTrials.gov — clinical-trial registry
Vincristine, dactinomycin/cyclophosphamide combination therapy with liposomal doxorubicin, doxorubicin, pharmorubicin, or pirarubicin comparator armsClinical trial · NCT03892330Clinical trialTrial only (registry)China · Children aged 0.5-14 years with nephroblastoma per the registry title and eligibility context. · The record is stale and not-yet-recruiting. It should be treated as a registry signal requiring local confirmation, not as an available treatment pathway. Confidence/conflicts: Medium for current local status because the registry is stale; high that the fetched registry record names nephroblastoma and the listed drug classes. trial-registry listing only — does not establish approval, reimbursement, or eligibility ClinicalTrials.gov — clinical-trial registry
dinutuximab beta with 13-cis-retinoic acid; chidamide + dinutuximab beta + irinotecan + temozolomide; dual-target GD2/B7-H3 CAR-NK cells (EB-DTNB-NK) with fludarabine/cyclophosphamide lymphodepletionClinical trial · NCT05373901Clinical trialTrial only (registry)China · GD2 and B7-H3 target-expression assessment required for the CAR-NK trial; not specified for dinutuximab beta maintenance or chidamide combination beyond disease criteria; High-risk post-induction/post-myeloablative therapy/stem cell transplant maintenance; refractory/relapsed pediatric neuroblastoma; relapsed/refractory/high-risk neuroblastoma or ganglioneuroblastoma with target-expression assessment. · Trial evidence only; one study is completed, two are recruiting; CAR-NK record is early phase and allogeneic cell therapy with eligibility restrictions. Confidence/conflicts: High confidence for China registry cells and named interventions; no conflicting fetched source. NMPA approval for combinations/cell therapy not established. ClinicalTrials.gov — trial registry
LDE225/sonidegib; temozolomideClinical trial · NCT01708174Clinical trialTrial only (NCT01708174)Russia · Hedgehog-pathway activated medulloblastoma; Relapsed Hedgehog-pathway activated medulloblastoma. · Completed registry status does not establish Roszdravnadzor approval, current availability, reimbursement, or individual eligibility. Confidence/conflicts: High for completed Russia registry status; no approval inferred. ClinicalTrials.gov — clinical-trial registry
Lipegfilgrastim (XM22) supportive-care pharmacokinetic/pharmacodynamic study; dinutuximab beta chemo-immunotherapy study in pediatric bone and soft-tissue sarcomasClinical trial · NCT01585649Clinical trialTrial only (registry)Russia · No biomarker required by fetched registry records; Pediatric Ewing family tumor/RMS supportive-care study; pediatric bone/soft-tissue sarcoma study including embryonal and alveolar RMS. · Registry listing does not establish Russian regulatory approval, reimbursement, routine access, enrollment availability outside the listed trial, or individual eligibility. A broad bevacizumab sarcoma study and a broad dendritic-cell sarcoma study were seen but not used as RMS-specific findings because the fetched condition fields were not RMS-specific. Confidence/conflicts: Medium-high for registry-listed Russia RMS-related trial/supportive options; no local approval or routine-access claim made. ClinicalTrials.gov — clinical-trial registry
modified autologous cytokine-induced killer (CIK) cells; BCD-245Clinical trial · NCT01868490Clinical trialTrial only (registry)Russia · Thailand: advanced cholangiocarcinoma or neuroblastoma failing current treatment; Russia: relapsed or refractory neuroblastoma resistant to site-adopted anti-relapse therapy. · Thailand study is invitation-only; Russia study status is unknown and last updated in 2023; both are early-phase investigational cells. Confidence/conflicts: Medium-high confidence for fetched registry support; enrollment/current access uncertain. No conflicting fetched source. ClinicalTrials.gov — trial registry
modified autologous cytokine-induced killer (CIK) cells; BCD-245Clinical trial · NCT01868490Clinical trialTrial only (registry)Thailand · Thailand: advanced cholangiocarcinoma or neuroblastoma failing current treatment; Russia: relapsed or refractory neuroblastoma resistant to site-adopted anti-relapse therapy. · Thailand study is invitation-only; Russia study status is unknown and last updated in 2023; both are early-phase investigational cells. Confidence/conflicts: Medium-high confidence for fetched registry support; enrollment/current access uncertain. No conflicting fetched source. ClinicalTrials.gov — trial registry

A clinical-trial listing or early report shows an option is being studied — not that it works, that it is safe for any one person, or that a site is enrolling today. Whether any of these fits is a conversation for your oncology team and the trial team. Last checked June 2026.

What this page does

Maps options by country

It maps sourced options by country alongside diagnosis wording, stage, test results, specialists, and trial-search terms.

What it does not do

Does not choose treatment

It does not rank treatments, recommend a choice, or decide clinical fit.

Where it comes from

Built on trusted sources

Every option links to a trusted regulator, HTA, or guideline source, and the list grows as new sources pass verification.

Why this condition is included

Pediatric cancer families need age-specific source handling and careful emotional tone.
Clinical options often depend on pediatric oncology centers and trial availability.
Pediatric coverage stays deliberately careful — it links trusted pediatric sources rather than making detailed clinical claims.

Information to gather before the next visit

What exact pediatric tumor type and pathology wording are documented?
Is care being coordinated through a pediatric oncology center?
Were molecular testing, surgery, chemotherapy, radiation, or trial referral discussed?
What records would a second-opinion pediatric center need?

Trial-search terms to discuss

pediatric solid tumor clinical trial childhood cancer molecular testing clinical trial