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선택지 정리됨

결절성 경화증(TSC): 국가별 치료 선택지

이 페이지는 한국어 임상 치료 설명을 새로 쓰지 않습니다. 권위 출처, 진료 전 정리 질문, 임상시험 검색어, 구축 상태를 보여주는 안전한 시작점입니다.

선택지 정리됨희귀·유전 질환최종 확인 2026.06

국가별 선택지

국가별 치료 선택지

공식 규제·평가 기관 출처를 바탕으로 한 국가별 승인·접근 상태입니다. 무엇이 어디에 존재하는지를 보여줄 뿐, 추천이 아닙니다.

European Union

  • everolimus (Votubia); cannabidiol (Epidyolex)[1]EMA authorisedTSC1/TSC2 pathway condition; option selection is manifestation-defined in the fetched sources; EMA Votubia settings are manifestation-specific: renal angiomyolipoma, SEGA, and add-on treatment for refractory TSC-related partial-onset seizures. Wales cannabidiol source is a commissioning policy for seizures caused by TSC in people aged 2 years and above. · EMA authorization does not itself establish access or reimbursement in every EU member state. The Wales policy is jurisdiction-specific within the UK and does not automatically establish England, Scotland, or Northern Ireland access. The Wales document also describes other seizure-related options such as added anti-seizure medicines, ketogenic diet, vagus nerve stimulation, and surgical resection as context rather than universal recommendations. Confidence/conflicts: High for EU Votubia authorization and Wales cannabidiol commissioning; medium for broader UK because England/Scotland/Northern Ireland source cells need separate verification.

Australia

  • everolimus (Afinitor)[2]TGA-registered (Australia)TSC clinical/genetic disorder context; manifestation-defined treatment; Australian patient-advocacy access summary for everolimus across SEGA, renal angiomyolipoma, and refractory seizures. · This is a patient-advocacy source, not the live PBS Schedule. Exact subsidy criteria, formulations, prescriber rules, and authority requirements need direct PBS/TGA confirmation before use as a final access claim. Confidence/conflicts: Medium. Source is disease-advocacy and consistent with earlier fetched TGA label context, but live PBS authority criteria remain source-pending. No conflict identified. Availability/reimbursement outside the approving regulator not established.
  • everolimus (Afinitor; Afinitor dispersible tablets)[3]TGA-registered (Australia)TSC clinical/genetic disorder context; no TSC1/TSC2-specific restriction stated in the fetched Australian product information; TSC-associated SEGA requiring intervention, TSC renal angiomyolipoma not requiring immediate surgery, and adjunctive treatment for TSC-associated refractory seizures, as stated by the Australian sources. · The Australian product information distinguishes tablet and dispersible-tablet use; it states tablets are not recommended for TSC refractory seizures while dispersible tablets may be used for SEGA and refractory seizures with therapeutic drug monitoring. This entry records label status, not PBS subsidy or individual eligibility. Confidence/conflicts: High for Australian everolimus TSC label contexts; no conflict identified. confirm current TGA ARTG registration status Availability/reimbursement outside the approving regulator not established.
  • multidisciplinary pregnancy planning; everolimus medication review; renal and neurologic monitoring[4]Standard option (per Tuberous Sclerosis Australia)TSC clinical/genetic disorder context; preconception/pregnancy supportive-care planning in Australian TSC education context. · This is an educational resource, not individual pregnancy advice. Medication changes, fetal medicine referral, nephrology, neurology, and obstetric planning require specialist review. Confidence/conflicts: Medium-high for supportive-care planning context; not a drug-label or payer source. No conflict identified.

Canada

  • cannabidiol oral solution (Epidiolex)[5]Health Canada approvedTSC clinical/genetic disorder context; adjunctive therapy for TSC-associated seizures in patients aged 2 years and older, as stated by the Canadian product monograph. · This is antiseizure treatment for TSC-associated seizures, not a disease-modifying therapy for the underlying TSC process. This entry records label status, not public-drug-plan coverage or individual suitability. Confidence/conflicts: High for Canadian label status; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • cannabidiol oral solution (Epidiolex), plus contextual antiseizure and nonpharmacologic options described by CADTH[6]Health Canada approvedTSC clinical/genetic disorder context; Canadian reimbursement-review and supportive-care context for TSC-associated seizures. · CADTH is a reimbursement/evidence body and does not replace the product monograph. CADTH clinical experts noted cannabidiol does not address the underlying disease process more than conventional antiseizure medicines; it is framed here as an option to discuss for seizure control, not a cure or superior option. Confidence/conflicts: Medium-high for CADTH reimbursement and supportive-care context; no conflict with the Canadian product monograph identified.
  • everolimus (Afinitor or generic everolimus protocol context)[7]ApprovedTSC clinical/genetic disorder context; British Columbia protocol context for everolimus in TSC-associated high-risk renal angiomyolipoma. · BC Cancer protocol is a professional consensus/protocol document and states clinicians must use independent medical judgment. It is provincial and should not be generalized to all Canadian jurisdictions without local verification. Confidence/conflicts: High for BC protocol existence and eligibility summary; other provinces remain source-pending. No conflict identified. Availability/reimbursement outside the approving regulator not established.
  • everolimus (Afinitor); sirolimus comparator context[8]Health Canada approvedTSC clinical/genetic disorder context; Canadian Health Canada authorization and national HTA/evidence review context for TSC-associated renal angiomyolipoma. · CDA-AMC review is not a final universal provincial coverage decision. Pediatric renal angiomyolipoma coverage/indication, sirolimus off-label comparator use, imaging access, and provincial implementation remain uncertain. Confidence/conflicts: Medium-high for Canadian regulatory/HTA context; provincial reimbursement remains variable and source-pending. No conflict identified.
  • everolimus (Afinitor; Afinitor Disperz)[9]ApprovedTSC clinical/genetic disorder context; no TSC1/TSC2-specific restriction stated in the fetched Canadian product monograph; Canada label contexts for TSC renal angiomyolipoma, TSC-associated SEGA, and adjunctive treatment of TSC-associated seizures not satisfactorily controlled with current therapies. · The Canadian monograph states surgical resection can be curative for SEGA while everolimus has been shown only to reduce SEGA volume. It also specifies formulation limits for seizure treatment and notes that pediatric renal angiomyolipoma use is not authorized. Confidence/conflicts: High for Canadian monograph status; no conflict identified. Availability/reimbursement outside the approving regulator not established.

Multiple

  • vigabatrin; ACTH/prednisolone fallback; mTOR inhibitor; embolization; kidney-sparing therapy[10]Standard option (per TSC Alliance)TSC clinical/genetic disorder context; International consensus supportive/disease-management context for TSC infantile spasms and renal angiomyolipoma. · This is international guidance, not proof of approval or availability in any one country. Vigabatrin, ACTH/steroid, mTOR inhibitor, embolization, and kidney-sparing options must be checked against local approvals, access, and specialist pathways. Confidence/conflicts: High for consensus-guideline statements; country-specific access remains source-pending. No conflict identified.

출처

  1. European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
  2. Tuberous Sclerosis Australia — patient-advocacy treatment/access education · patient-advocacy treatment/access education
  3. Therapeutic Goods Administration (TGA) — Australian Public Assessment Report · Australian Public Assessment Report
  4. Tuberous Sclerosis Australia — GP educational resource · GP educational resource
  5. Health Canada Drug and Health Product Portal — regulator product portal · regulator product portal
  6. CADTH via NCBI Bookshelf — reimbursement recommendation / health technology review · reimbursement recommendation / health technology review
  7. BC Cancer — provincial treatment protocol · provincial treatment protocol
  8. Canada's Drug Agency / CDA-AMC — reimbursement review · reimbursement review
  9. Novartis Canada / Health Canada product monograph format — official product monograph · official product monograph
  10. TSC Alliance — international consensus surveillance and management guideline summary · international consensus surveillance and management guideline summary

위 내용은 공식 규제·접근 상태일 뿐, 의학적 조언이나 추천이 아니고, 적격성을 판단하지도 않습니다. 어떤 선택지가 적합한지는 환자의 상황과 종양내과 팀에 달려 있습니다. 규제 상태는 바뀔 수 있으니 표시된 출처에서 확인하세요. 임상 세부 내용은 영문이 정본입니다. 최종 확인 2026.06.

승인된 치료 너머

임상시험 및 신흥 선택지

환자가 거주하는 국가에서 아직 승인된 표준 치료가 아닌 선택지입니다 — 연구, 임상시험, 허가 외 사용, 담당 종양내과 의사가 먼저 언급하지 않을 수 있는 초기 근거입니다. 각 항목은 근거의 강도에 따라 구분됩니다. 여기에 실린 항목은 조사하고 의료진과 상의할 정보일 뿐, 효과가 입증되었거나 안전하거나 현재 이용 가능하다는 의미가 아닙니다. 임상 세부 내용은 영문이 정본입니다.

임상시험 진행 중

  • sirolimus (rapamycin); rapamycin topical cream; placebo comparator where applicable임상시험 · NCT05104983임상시험Trial only (NCT05104983)United States · TSC1/TSC2 pathway condition; option selection is manifestation-defined in the fetched sources; Early sirolimus seizure-prevention/progression study in infants/children as defined by the registry; topical rapamycin facial-angiofibroma dose-ranging study. · Recruiting or completed trial status does not establish routine national approval or availability. Some countries in NCT03826628 are not primary MatchMedi countries but are useful secondary geography for future coverage. Confidence/conflicts: High for registry status/geography; no routine approval inferred from trial records. ClinicalTrials.gov — clinical-trial registry
  • sirolimus gel (Rapalimus Gel / NPC-12G gel); everolimus (RAD001 / Afinitor / Votubia)임상시험 · NCT02635789임상시험Trial only (NCT02635789)Japan · TSC1/TSC2 pathway condition; option selection is manifestation-defined in the fetched sources; Japan skin-lesion sirolimus review/trial context; multinational adjunctive everolimus for TSC refractory partial-onset seizures and long-term rollover for patients completing EXIST-3 and benefitting from continued treatment. · The PMDA review and ClinicalTrials.gov records do not by themselves establish current reimbursement or routine access outside the recorded jurisdiction and trial context. Thailand and Russia are recorded here as verified trial geographies, not as verified routine-treatment access countries. Direct current PMDA package inserts and direct MFDS/Thai FDA/Russian labels remain source-pending. Confidence/conflicts: Medium-high for Japan sirolimus review/trial context and multinational trial geography; low for routine access in Thailand, Russia, and South Korea until direct regulator or reimbursement sources are fetched. Pharmaceuticals and Medical Devices Agency (PMDA) — regulator review report
  • everolimus (Afinitor / RAD001); cannabidiol (GWP42003-P / Epidiolex-Epidyolex)임상시험 · NCT03525834임상시험Trial only (NCT03525834)China · TSC1/TSC2 pathway condition; option selection is manifestation-defined in the fetched sources; Adult Chinese and Taiwanese TSC-associated renal angiomyolipoma everolimus phase 4 contexts; add-on cannabidiol phase 3 seizure context in TSC. · Trial geography does not establish NMPA, Taiwan regulator, NHS, EU member-state reimbursement, or routine access. China and Taiwan are separated because the fetched records identify different study geographies. Direct NMPA and Taiwan label/reimbursement sources remain source-pending. Confidence/conflicts: High for trial geography and status; low for routine China/Taiwan approval or reimbursement without direct regulator sources. ClinicalTrials.gov — clinical-trial registry

임상시험이나 초기 보고에 실렸다는 것은 해당 선택지가 연구되고 있다는 의미일 뿐, 효과가 있거나 특정 환자에게 안전하거나 현재 등록 가능하다는 뜻은 아닙니다. 어떤 선택지가 적합한지는 담당 종양내과 팀과 임상시험 팀이 함께 상의할 문제입니다. 최종 확인 2026.06.

현재 제공

국가별 선택지를 정리합니다

국가별로 출처가 연결된 선택지와 함께, 진단명·단계·검사 결과·진료과·임상시험 검색어를 정리합니다.

아직 하지 않음

치료 선택을 지시하지 않습니다

이 페이지는 치료 순위, 권장, 적합성 판단을 하지 않습니다.

출처

공신력 있는 출처 기반

모든 선택지는 규제·평가·지침 등 공신력 있는 출처에 연결되며, 새 출처가 검증되면 계속 확장됩니다.

진료 전 정리할 정보

  • Is the access question EU authorization, Wales commissioning, England NICE/NHS access, or another UK nation?
  • Which manifestation is being treated, and does the fetched authorization or policy cover that manifestation?
  • Are non-drug seizure options such as ketogenic diet, VNS, or epilepsy surgery assessment relevant to discuss with the specialist team?
  • Is the local question skin-lesion topical sirolimus, seizure-directed everolimus, or another TSC manifestation?

임상시험 검색어

Tuberous sclerosis complex (TSC) clinical trial