Options mapped

Langerhans cell histiocytosis: options by country

Sourced options by country plus visit-prep questions for Langerhans cell histiocytosis. Each line links to its regulator, HTA, or guideline source. This page maps options; it does not recommend or rank them.

Options mappedRare diseaseLast 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.

Information to gather before the next visit

Is this single-system, multisystem risk-organ negative, or multisystem risk-organ positive LCH?
Have CD1a/CD207 pathology and BRAF/MAPK pathway testing been performed?
What response checkpoint is being used to decide whether to continue or change therapy?
Is the patient an adult with a histiocytic neoplasm covered by the label?

Trial-search terms to discuss

Langerhans cell histiocytosis clinical trial

Options by country

Treatments by country

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

United States

Cobimetinib (Cotellic)[1]FDA-approvedAny mutational status in the FDA label's histiocytic neoplasm trial; BRAF V600E patients were allowed only under specified prior-access/toxicity circumstances in the trial description; Adult histiocytic neoplasms; trial context included multisystem, recurrent/refractory, or selected single-system disease unlikely to benefit from conventional therapies. · This is an adult histiocytic-neoplasm label, not a pediatric LCH-specific approval. Label safety warnings and monitoring requirements apply; this entry does not imply eligibility or superiority over other MAPK-targeted approaches. Confidence/conflicts: High for adult FDA label; LCH subgroup was small, so disease-specific generalizability should be discussed with a histiocytosis specialist.
Observation, local therapy, surgery/curettage, radiation therapy, topical/oral/IV therapies, vinblastine/prednisone, methotrexate, cytarabine, cladribine, clofarabine, bisphosphonates, hydroxyurea with or without methotrexate, MAPK inhibitors, hematopoietic stem cell transplant, long-term follow-up [2]Standard option (per NCI PDQ)BRAF V600E, MAP2K1 and other RAS-MAPK pathway alterations; CD1a/CD207 diagnostic confirmation; risk-organ involvement of liver, spleen, and hematopoietic system; response at 6-12 weeks; Low-risk single-system or multisystem disease; high-risk multisystem disease; recurrent/refractory/progressive disease; long-term monitoring for reactivation and permanent consequences. · NCI states optimal therapy for recurrent/refractory/progressive LCH is not fully determined, and treatment depends on site, risk organs, molecular features, and early response. Do not generalize targeted therapy to all LCH without mutation and clinical context. Confidence/conflicts: High for U.S. evidence-summary framework; no conflict recorded.
Observation, local therapy, surgery/curettage, radiation therapy, topical/oral/IV therapies, vinblastine/prednisone, methotrexate, cytarabine, cladribine, clofarabine, bisphosphonates, hydroxyurea with or without methotrexate, MAPK inhibitors, hematopoietic stem cell transplant, long-term follow-up [2]Standard option (per NCI PDQ)BRAF V600E, MAP2K1 and other RAS-MAPK pathway alterations; CD1a/CD207 diagnostic confirmation; risk-organ involvement of liver, spleen, and hematopoietic system; response at 6-12 weeks; Low-risk single-system or multisystem disease; high-risk multisystem disease; recurrent/refractory/progressive disease; long-term monitoring for reactivation and permanent consequences. · NCI states optimal therapy for recurrent/refractory/progressive LCH is not fully determined, and treatment depends on site, risk organs, molecular features, and early response. Do not generalize targeted therapy to all LCH without mutation and clinical context. Confidence/conflicts: High for U.S. evidence-summary framework; no conflict recorded.
Observation, local therapy, surgery/curettage, radiation therapy, topical/oral/IV therapies, vinblastine/prednisone, methotrexate, cytarabine, cladribine, clofarabine, bisphosphonates, hydroxyurea with or without methotrexate, MAPK inhibitors, hematopoietic stem cell transplant, long-term follow-up [2]Standard option (per NCI PDQ)BRAF V600E, MAP2K1 and other RAS-MAPK pathway alterations; CD1a/CD207 diagnostic confirmation; risk-organ involvement of liver, spleen, and hematopoietic system; response at 6-12 weeks; Low-risk single-system or multisystem disease; high-risk multisystem disease; recurrent/refractory/progressive disease; long-term monitoring for reactivation and permanent consequences. · NCI states optimal therapy for recurrent/refractory/progressive LCH is not fully determined, and treatment depends on site, risk organs, molecular features, and early response. Do not generalize targeted therapy to all LCH without mutation and clinical context. Confidence/conflicts: High for U.S. evidence-summary framework; no conflict recorded.
Observation, local therapy, surgery/curettage, radiation therapy, topical/oral/IV therapies, vinblastine/prednisone, methotrexate, cytarabine, cladribine, clofarabine, bisphosphonates, hydroxyurea with or without methotrexate, MAPK inhibitors, hematopoietic stem cell transplant, long-term follow-up [2]Standard option (per NCI PDQ)BRAF V600E, MAP2K1 and other RAS-MAPK pathway alterations; CD1a/CD207 diagnostic confirmation; risk-organ involvement of liver, spleen, and hematopoietic system; response at 6-12 weeks; Low-risk single-system or multisystem disease; high-risk multisystem disease; recurrent/refractory/progressive disease; long-term monitoring for reactivation and permanent consequences. · NCI states optimal therapy for recurrent/refractory/progressive LCH is not fully determined, and treatment depends on site, risk organs, molecular features, and early response. Do not generalize targeted therapy to all LCH without mutation and clinical context. Confidence/conflicts: High for U.S. evidence-summary framework; no conflict recorded.
Observation, local therapy, surgery/curettage, radiation therapy, topical/oral/IV therapies, vinblastine/prednisone, methotrexate, cytarabine, cladribine, clofarabine, bisphosphonates, hydroxyurea with or without methotrexate, MAPK inhibitors, hematopoietic stem cell transplant, long-term follow-up [2]Standard option (per NCI PDQ)BRAF V600E, MAP2K1 and other RAS-MAPK pathway alterations; CD1a/CD207 diagnostic confirmation; risk-organ involvement of liver, spleen, and hematopoietic system; response at 6-12 weeks; Low-risk single-system or multisystem disease; high-risk multisystem disease; recurrent/refractory/progressive disease; long-term monitoring for reactivation and permanent consequences. · NCI states optimal therapy for recurrent/refractory/progressive LCH is not fully determined, and treatment depends on site, risk organs, molecular features, and early response. Do not generalize targeted therapy to all LCH without mutation and clinical context. Confidence/conflicts: High for U.S. evidence-summary framework; no conflict recorded.
Observation, local therapy, surgery/curettage, radiation therapy, topical/oral/IV therapies, vinblastine/prednisone, methotrexate, cytarabine, cladribine, clofarabine, bisphosphonates, hydroxyurea with or without methotrexate, MAPK inhibitors, hematopoietic stem cell transplant, long-term follow-up [2]Standard option (per NCI PDQ)BRAF V600E, MAP2K1 and other RAS-MAPK pathway alterations; CD1a/CD207 diagnostic confirmation; risk-organ involvement of liver, spleen, and hematopoietic system; response at 6-12 weeks; Low-risk single-system or multisystem disease; high-risk multisystem disease; recurrent/refractory/progressive disease; long-term monitoring for reactivation and permanent consequences. · NCI states optimal therapy for recurrent/refractory/progressive LCH is not fully determined, and treatment depends on site, risk organs, molecular features, and early response. Do not generalize targeted therapy to all LCH without mutation and clinical context. Confidence/conflicts: High for U.S. evidence-summary framework; no conflict recorded.
Observation, local therapy, surgery/curettage, radiation therapy, topical/oral/IV therapies, vinblastine/prednisone, methotrexate, cytarabine, cladribine, clofarabine, bisphosphonates, hydroxyurea with or without methotrexate, MAPK inhibitors, hematopoietic stem cell transplant, long-term follow-up [2]Standard option (per NCI PDQ)BRAF V600E, MAP2K1 and other RAS-MAPK pathway alterations; CD1a/CD207 diagnostic confirmation; risk-organ involvement of liver, spleen, and hematopoietic system; response at 6-12 weeks; Low-risk single-system or multisystem disease; high-risk multisystem disease; recurrent/refractory/progressive disease; long-term monitoring for reactivation and permanent consequences. · NCI states optimal therapy for recurrent/refractory/progressive LCH is not fully determined, and treatment depends on site, risk organs, molecular features, and early response. Do not generalize targeted therapy to all LCH without mutation and clinical context. Confidence/conflicts: High for U.S. evidence-summary framework; no conflict recorded.

Sources

U.S. Food and Drug Administration — official drug label · official drug label
National Cancer Institute — national cancer agency evidence summary · national cancer agency evidence summary

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. Last checked 2026-06-12.

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

Vinblastine/prednisolone, methotrexate/leucovorin, cytarabine, vinblastine/prednisone, clofarabineClinical trial · NCT00276757Clinical trialTrial only (registry)United States · Histopathologic LCH confirmation; CD1a/Langerin/CD207 confirmation in protocol contexts; recurrent/refractory disease after prior systemic therapy for clofarabine; Newly diagnosed pediatric LCH; frontline systemic therapy; recurrent/refractory LCH after prior systemic therapy. · LCH-III is completed and older; current treatment should be checked against contemporary histiocytosis-center practice. Clofarabine record requires prior systemic therapy failure for LCH stratum. Confidence/conflicts: High for registry-listed interventions and geographies; no approval claim is made for each drug in LCH. ClinicalTrials.gov — clinical-trial registry
Vinblastine/prednisolone, methotrexate/leucovorin, cytarabine, vinblastine/prednisone, clofarabineClinical trial · NCT00276757Clinical trialTrial only (registry)United States · Histopathologic LCH confirmation; CD1a/Langerin/CD207 confirmation in protocol contexts; recurrent/refractory disease after prior systemic therapy for clofarabine; Newly diagnosed pediatric LCH; frontline systemic therapy; recurrent/refractory LCH after prior systemic therapy. · LCH-III is completed and older; current treatment should be checked against contemporary histiocytosis-center practice. Clofarabine record requires prior systemic therapy failure for LCH stratum. Confidence/conflicts: High for registry-listed interventions and geographies; no approval claim is made for each drug in LCH. ClinicalTrials.gov — clinical-trial registry
Dabrafenib plus trametinibClinical trial · NCT07440290Clinical trialTrial only (registry)United Kingdom · BRAF V600 mutation-positive cancer, including LCH in the arm 07 condition and eligibility text; Rare BRAF V600 mutation-positive cancer basket setting, including LCH; patients aged at least 1 year and meeting DETERMINE master protocol criteria. · Not-yet-recruiting in the fetched registry record. This is a molecular basket trial, not an LCH-specific standard-care recommendation. Confidence/conflicts: High for UK registry cell and BRAF V600 trial framing; no approval/reimbursement claim is made. trial-registry listing only — does not establish approval, reimbursement, or eligibility ClinicalTrials.gov — clinical-trial registry
Dabrafenib plus trametinibClinical trial · NCT07440290Clinical trialTrial only (registry)United Kingdom · BRAF V600 mutation-positive cancer, including LCH in the arm 07 condition and eligibility text; Rare BRAF V600 mutation-positive cancer basket setting, including LCH; patients aged at least 1 year and meeting DETERMINE master protocol criteria. · Not-yet-recruiting in the fetched registry record. This is a molecular basket trial, not an LCH-specific standard-care recommendation. Confidence/conflicts: High for UK registry cell and BRAF V600 trial framing; no approval/reimbursement claim is made. trial-registry listing only — does not establish approval, reimbursement, or eligibility ClinicalTrials.gov — clinical-trial registry
Prednisone, cytarabine, vincristine, mercaptopurine, local therapy, dabrafenib or trametinib with clofarabine, luvometinib alone or with modified LCH-III regimen, cytarabine with thalidomide, CD207 CAR-T cellsClinical trial · NCT04773366Clinical trialTrial only (registry)China · CD1a/CD207/Langerin diagnostic confirmation; BRAF/MAPK pathway context; high-risk liver/spleen/hematopoietic involvement; special-site bone lesions; relapsed/refractory criteria; Newly diagnosed pediatric LCH; high-risk/recurrent/refractory pediatric LCH; multisystem pediatric LCH; single-system special-site/multifocal bone pediatric LCH; untreated adult multisystem or multifocal LCH; relapsed/refractory LCH. · Registry status varies, including recruiting and unknown status. Luvometinib and CD207 CAR-T remain investigational trial-framed options. Confidence/conflicts: High for China registry cells; no NMPA approval claim is made. ClinicalTrials.gov — clinical-trial registry
Prednisone, cytarabine, vincristine, mercaptopurine, local therapy, dabrafenib or trametinib with clofarabine, luvometinib alone or with modified LCH-III regimen, cytarabine with thalidomide, CD207 CAR-T cellsClinical trial · NCT04773366Clinical trialTrial only (registry)China · CD1a/CD207/Langerin diagnostic confirmation; BRAF/MAPK pathway context; high-risk liver/spleen/hematopoietic involvement; special-site bone lesions; relapsed/refractory criteria; Newly diagnosed pediatric LCH; high-risk/recurrent/refractory pediatric LCH; multisystem pediatric LCH; single-system special-site/multifocal bone pediatric LCH; untreated adult multisystem or multifocal LCH; relapsed/refractory LCH. · Registry status varies, including recruiting and unknown status. Luvometinib and CD207 CAR-T remain investigational trial-framed options. Confidence/conflicts: High for China registry cells; no NMPA approval claim is made. ClinicalTrials.gov — clinical-trial registry
Prednisone, cytarabine, vincristine, mercaptopurine, local therapy, dabrafenib or trametinib with clofarabine, luvometinib alone or with modified LCH-III regimen, cytarabine with thalidomide, CD207 CAR-T cellsClinical trial · NCT04773366Clinical trialTrial only (registry)China · CD1a/CD207/Langerin diagnostic confirmation; BRAF/MAPK pathway context; high-risk liver/spleen/hematopoietic involvement; special-site bone lesions; relapsed/refractory criteria; Newly diagnosed pediatric LCH; high-risk/recurrent/refractory pediatric LCH; multisystem pediatric LCH; single-system special-site/multifocal bone pediatric LCH; untreated adult multisystem or multifocal LCH; relapsed/refractory LCH. · Registry status varies, including recruiting and unknown status. Luvometinib and CD207 CAR-T remain investigational trial-framed options. Confidence/conflicts: High for China registry cells; no NMPA approval claim is made. ClinicalTrials.gov — clinical-trial registry
Prednisone, cytarabine, vincristine, mercaptopurine, local therapy, dabrafenib or trametinib with clofarabine, luvometinib alone or with modified LCH-III regimen, cytarabine with thalidomide, CD207 CAR-T cellsClinical trial · NCT04773366Clinical trialTrial only (registry)China · CD1a/CD207/Langerin diagnostic confirmation; BRAF/MAPK pathway context; high-risk liver/spleen/hematopoietic involvement; special-site bone lesions; relapsed/refractory criteria; Newly diagnosed pediatric LCH; high-risk/recurrent/refractory pediatric LCH; multisystem pediatric LCH; single-system special-site/multifocal bone pediatric LCH; untreated adult multisystem or multifocal LCH; relapsed/refractory LCH. · Registry status varies, including recruiting and unknown status. Luvometinib and CD207 CAR-T remain investigational trial-framed options. Confidence/conflicts: High for China registry cells; no NMPA approval claim is made. ClinicalTrials.gov — clinical-trial registry
Prednisone, cytarabine, vincristine, mercaptopurine, local therapy, dabrafenib or trametinib with clofarabine, luvometinib alone or with modified LCH-III regimen, cytarabine with thalidomide, CD207 CAR-T cellsClinical trial · NCT04773366Clinical trialTrial only (registry)China · CD1a/CD207/Langerin diagnostic confirmation; BRAF/MAPK pathway context; high-risk liver/spleen/hematopoietic involvement; special-site bone lesions; relapsed/refractory criteria; Newly diagnosed pediatric LCH; high-risk/recurrent/refractory pediatric LCH; multisystem pediatric LCH; single-system special-site/multifocal bone pediatric LCH; untreated adult multisystem or multifocal LCH; relapsed/refractory LCH. · Registry status varies, including recruiting and unknown status. Luvometinib and CD207 CAR-T remain investigational trial-framed options. Confidence/conflicts: High for China registry cells; no NMPA approval claim is made. ClinicalTrials.gov — clinical-trial registry
Prednisone, cytarabine, vincristine, mercaptopurine, local therapy, dabrafenib or trametinib with clofarabine, luvometinib alone or with modified LCH-III regimen, cytarabine with thalidomide, CD207 CAR-T cellsClinical trial · NCT04773366Clinical trialTrial only (registry)China · CD1a/CD207/Langerin diagnostic confirmation; BRAF/MAPK pathway context; high-risk liver/spleen/hematopoietic involvement; special-site bone lesions; relapsed/refractory criteria; Newly diagnosed pediatric LCH; high-risk/recurrent/refractory pediatric LCH; multisystem pediatric LCH; single-system special-site/multifocal bone pediatric LCH; untreated adult multisystem or multifocal LCH; relapsed/refractory LCH. · Registry status varies, including recruiting and unknown status. Luvometinib and CD207 CAR-T remain investigational trial-framed options. Confidence/conflicts: High for China registry cells; no NMPA approval claim is made. ClinicalTrials.gov — clinical-trial registry
Vemurafenib with cytarabine and 2-chlorodeoxyadenosine / cladribineClinical trial · NCT03585686Clinical trialTrial only (registry)Russia · BRAF V600E mutation; CD1a/CD207 histologic diagnosis; Children aged 0-18 with histologically verified LCH and BRAF V600E mutation, including life-threatening cases where urgent vemurafenib use before mutation confirmation is described in the protocol criteria. · The registry record is unknown status and should be locally confirmed. Vemurafenib is trial-framed in this pediatric LCH combination context. Confidence/conflicts: Medium because registry status is unknown; high that the fetched record supports the Russia BRAF V600E pediatric LCH trial cell. trial-registry listing only — does not establish approval, reimbursement, or eligibility ClinicalTrials.gov — clinical-trial registry
Vemurafenib with cytarabine and 2-chlorodeoxyadenosine / cladribineClinical trial · NCT03585686Clinical trialTrial only (registry)Russia · BRAF V600E mutation; CD1a/CD207 histologic diagnosis; Children aged 0-18 with histologically verified LCH and BRAF V600E mutation, including life-threatening cases where urgent vemurafenib use before mutation confirmation is described in the protocol criteria. · The registry record is unknown status and should be locally confirmed. Vemurafenib is trial-framed in this pediatric LCH combination context. Confidence/conflicts: Medium because registry status is unknown; high that the fetched record supports the Russia BRAF V600E pediatric LCH trial cell. trial-registry listing only — does not establish approval, reimbursement, or eligibility ClinicalTrials.gov — clinical-trial registry
Vemurafenib with cytarabine and 2-chlorodeoxyadenosine / cladribineClinical trial · NCT03585686Clinical trialTrial only (registry)Russia · BRAF V600E mutation; CD1a/CD207 histologic diagnosis; Children aged 0-18 with histologically verified LCH and BRAF V600E mutation, including life-threatening cases where urgent vemurafenib use before mutation confirmation is described in the protocol criteria. · The registry record is unknown status and should be locally confirmed. Vemurafenib is trial-framed in this pediatric LCH combination context. Confidence/conflicts: Medium because registry status is unknown; high that the fetched record supports the Russia BRAF V600E pediatric LCH trial cell. trial-registry listing only — does not establish approval, reimbursement, or eligibility ClinicalTrials.gov — clinical-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 2026-06-12.