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Beta-thalassemia: 국가별 선택지

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선택지 정리됨희귀·유전 질환최종 확인 2026.06

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임상시험 검색어

Beta-thalassemia clinical trial

국가별 선택지

국가별 치료 선택지

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

United States

  • Luspatercept-aamt / Reblozyl; betibeglogene autotemcel / Zynteglo; exagamglogene autotemcel / Casgevy[1]FDA-approvedBeta-thalassemia requiring regular RBC transfusions; gene therapies require transplant/gene-therapy eligibility and product-specific criteria; Regular transfusion-dependent adult beta-thalassemia anemia for luspatercept; transfusion-dependent beta-thalassemia gene therapy contexts for Zynteglo/Casgevy. · Gene therapies require stem-cell collection, conditioning, infusion, and long-term follow-up. Luspatercept is not a substitute for RBC transfusions when immediate anemia treatment is needed. Confidence/conflicts: Medium-high. Reblozyl is FDA-label supported; Zynteglo source is manufacturer product page; Casgevy U.S. beta-thalassemia approval should be verified with FDA label in a future pass.
  • Luspatercept-aamt / Reblozyl; betibeglogene autotemcel / Zynteglo; exagamglogene autotemcel / Casgevy[1]FDA-approvedBeta-thalassemia requiring regular RBC transfusions; gene therapies require transplant/gene-therapy eligibility and product-specific criteria; Regular transfusion-dependent adult beta-thalassemia anemia for luspatercept; transfusion-dependent beta-thalassemia gene therapy contexts for Zynteglo/Casgevy. · Gene therapies require stem-cell collection, conditioning, infusion, and long-term follow-up. Luspatercept is not a substitute for RBC transfusions when immediate anemia treatment is needed. Confidence/conflicts: Medium-high. Reblozyl is FDA-label supported; Zynteglo source is manufacturer product page; Casgevy U.S. beta-thalassemia approval should be verified with FDA label in a future pass.
  • Luspatercept-aamt / Reblozyl; betibeglogene autotemcel / Zynteglo; exagamglogene autotemcel / Casgevy[1]FDA-approvedBeta-thalassemia requiring regular RBC transfusions; gene therapies require transplant/gene-therapy eligibility and product-specific criteria; Regular transfusion-dependent adult beta-thalassemia anemia for luspatercept; transfusion-dependent beta-thalassemia gene therapy contexts for Zynteglo/Casgevy. · Gene therapies require stem-cell collection, conditioning, infusion, and long-term follow-up. Luspatercept is not a substitute for RBC transfusions when immediate anemia treatment is needed. Confidence/conflicts: Medium-high. Reblozyl is FDA-label supported; Zynteglo source is manufacturer product page; Casgevy U.S. beta-thalassemia approval should be verified with FDA label in a future pass.

European Union

  • Casgevy / exagamglogene autotemcel[2]EMA authorisedTDT in people aged 12 years and older; no matched related HSC donor condition in EU announcement context; TDT in people aged 12 years and over for Casgevy; EU Zynteglo no-current-authorisation caveat. · Casgevy (exagamglogene autotemcel) holds an EU conditional marketing authorisation since 9 Feb 2024 for transfusion-dependent beta-thalassemia age 12+ where HSCT is appropriate and no HLA-matched related donor is available (confirmatory-data obligation ~Aug 2026). Source: Casgevy EPAR https://www.ema.europa.eu/en/medicines/human/EPAR/casgevy
  • Casgevy / exagamglogene autotemcel[2]EMA authorisedTDT in people aged 12 years and older; no matched related HSC donor condition in EU announcement context; TDT in people aged 12 years and over for Casgevy; EU Zynteglo no-current-authorisation caveat. · Casgevy (exagamglogene autotemcel) holds an EU conditional marketing authorisation since 9 Feb 2024 for transfusion-dependent beta-thalassemia age 12+ where HSCT is appropriate and no HLA-matched related donor is available (confirmatory-data obligation ~Aug 2026). Source: Casgevy EPAR https://www.ema.europa.eu/en/medicines/human/EPAR/casgevy
  • Reblozyl / luspatercept[3]EMA authorisedTDT in people aged 12 years and older; no matched related HSC donor condition in EU announcement context; TDT in people aged 12 years and over for Casgevy; EU Zynteglo no-current-authorisation caveat. · Reblozyl (luspatercept) holds full EU authorisation since 25 Jun 2020 (indication extended Feb 2023) for adult transfusion-dependent AND non-transfusion-dependent beta-thalassemia anaemia. Source: Reblozyl EPAR https://www.ema.europa.eu/en/medicines/human/EPAR/reblozyl
  • Reblozyl / luspatercept[3]EMA authorisedTDT in people aged 12 years and older; no matched related HSC donor condition in EU announcement context; TDT in people aged 12 years and over for Casgevy; EU Zynteglo no-current-authorisation caveat. · Reblozyl (luspatercept) holds full EU authorisation since 25 Jun 2020 (indication extended Feb 2023) for adult transfusion-dependent AND non-transfusion-dependent beta-thalassemia anaemia. Source: Reblozyl EPAR https://www.ema.europa.eu/en/medicines/human/EPAR/reblozyl
  • Zynteglo / betibeglogene autotemcel[4]EMA marketing authorisation withdrawn (Mar 2022)TDT in people aged 12 years and older; no matched related HSC donor condition in EU announcement context; TDT in people aged 12 years and over for Casgevy; EU Zynteglo no-current-authorisation caveat. · Only Zynteglo (betibeglogene autotemcel) is genuinely EU not-authorised — its EU marketing authorisation was withdrawn in March 2022 at bluebird bio's request for commercial reasons (withdrawal does not mean unavailable in all jurisdictions). Casgevy (conditional MA since 9 Feb 2024) and Reblozyl (full MA since 25 Jun 2020) ARE authorised and are now shown as separate available records.
  • Zynteglo / betibeglogene autotemcel[4]EMA marketing authorisation withdrawn (Mar 2022)TDT in people aged 12 years and older; no matched related HSC donor condition in EU announcement context; TDT in people aged 12 years and over for Casgevy; EU Zynteglo no-current-authorisation caveat. · Only Zynteglo (betibeglogene autotemcel) is genuinely EU not-authorised — its EU marketing authorisation was withdrawn in March 2022 at bluebird bio's request for commercial reasons (withdrawal does not mean unavailable in all jurisdictions). Casgevy (conditional MA since 9 Feb 2024) and Reblozyl (full MA since 25 Jun 2020) ARE authorised and are now shown as separate available records.

Australia

  • luspatercept (Reblozyl)[5]TGA-registered (Australia)beta-globin / beta-thalassemia context; transfusion-dependent anemia; adult patients with transfusion-dependent anemia associated with beta thalassaemia. · The AusPAR's product information attachment may have been superseded; the current TGA PI/PBS status should be checked separately. This entry does not establish payer access or suitability. Confidence/conflicts: High for Australian luspatercept registration context; PBS and current PI remain source-pending. Availability/reimbursement outside the approving regulator not established.
  • luspatercept (Reblozyl)[5]TGA-registered (Australia)beta-globin / beta-thalassemia context; transfusion-dependent anemia; adult patients with transfusion-dependent anemia associated with beta thalassaemia. · The AusPAR's product information attachment may have been superseded; the current TGA PI/PBS status should be checked separately. This entry does not establish payer access or suitability. Confidence/conflicts: High for Australian luspatercept registration context; PBS and current PI remain source-pending. Availability/reimbursement outside the approving regulator not established.
  • regular blood transfusions; iron chelation therapy; genetic counseling / reproductive options[6]Standard option (per Healthdirect Australia)thalassaemia carrier/disease context; severity-dependent transfusion need; Australian general thalassaemia supportive-care and family-planning context. · Healthdirect is patient education and does not establish a drug label, transplant eligibility, or local hospital protocol. It does not distinguish every beta-thalassemia subtype. Confidence/conflicts: Medium-high for Australian supportive-care context; beta-thalassemia subtype-specific specialist guidance remains a gap.
  • regular blood transfusions; iron chelation therapy; genetic counseling / reproductive options[6]Standard option (per Healthdirect Australia)thalassaemia carrier/disease context; severity-dependent transfusion need; Australian general thalassaemia supportive-care and family-planning context. · Healthdirect is patient education and does not establish a drug label, transplant eligibility, or local hospital protocol. It does not distinguish every beta-thalassemia subtype. Confidence/conflicts: Medium-high for Australian supportive-care context; beta-thalassemia subtype-specific specialist guidance remains a gap.

Thailand

  • Deferasirox (Deferasirox Teva)[7]ApprovedBeta-thalassemia major or non-transfusion-dependent thalassemia syndrome; chronic iron overload context.; Chronic transfusional iron overload and selected non-transfusion-dependent thalassemia iron-overload contexts as stated in product information. · Product information does not establish Thai payer approval, monitoring logistics, renal/hepatic safety suitability, or individual chelation thresholds. Confidence/conflicts: High for Thai NDI-hosted indication text; payer and monitoring implementation remain gaps. No conflict recorded. Availability/reimbursement outside the approving regulator not established.
  • Deferasirox; deferiprone; deferoxamine/desferrioxamine[8]ApprovedTransfusion-dependent thalassemia; age and prior-chelation context described in Thai NLEM conditions.; Iron-chelation access framework for transfusion-dependent thalassemia. · Thai-language NLEM text requires human review. This row describes access categories and conditions, not patient eligibility or dosing. Confidence/conflicts: Medium-high for NLEM access-condition text; human Thai-language review needed. No conflict recorded. Primary source is in Thai. English summary pending human review — confirm exact wording with your care team. Availability/reimbursement outside the approving regulator not established.
  • Luspatercept (Reblozyl)[9]ApprovedBeta-thalassemia diagnosis; transfusion-dependent anemia.; Adult transfusion-dependent anemia associated with beta-thalassemia. · Product information does not establish Thai reimbursement, routine access, transfusion program availability, iron-chelation coverage, or suitability for individual patients. Confidence/conflicts: High for Thai NDI-hosted indication text; payer and broader supportive-care access remain gaps. No conflict recorded.
  • Luspatercept (Reblozyl)[9]ApprovedBeta-thalassemia diagnosis; transfusion-dependent anemia.; Adult transfusion-dependent anemia associated with beta-thalassemia. · Product information does not establish Thai reimbursement, routine access, transfusion program availability, iron-chelation coverage, or suitability for individual patients. Confidence/conflicts: High for Thai NDI-hosted indication text; payer and broader supportive-care access remain gaps. No conflict recorded.

Canada

  • exagamglogene autotemcel (Casgevy)[10]EMA authorisedhomozygous or compound heterozygous beta-thalassemia context; autologous CRISPR/Cas9-edited CD34+ cell therapy targeting BCL11A enhancer to increase fetal hemoglobin; Canadian TDT patients aged 12 years and older; treatment-centre and autologous stem-cell-transplant suitability apply. · This is a complex cell therapy requiring conditioning and long-term follow-up, not routine transfusion support. Health Canada authorization does not guarantee provincial coverage or access to a treatment centre. Confidence/conflicts: High for Canadian TDT authorization; payer and center implementation remain gaps. Availability/reimbursement outside the approving regulator not established.
  • exagamglogene autotemcel (Casgevy)[10]EMA authorisedhomozygous or compound heterozygous beta-thalassemia context; autologous CRISPR/Cas9-edited CD34+ cell therapy targeting BCL11A enhancer to increase fetal hemoglobin; Canadian TDT patients aged 12 years and older; treatment-centre and autologous stem-cell-transplant suitability apply. · This is a complex cell therapy requiring conditioning and long-term follow-up, not routine transfusion support. Health Canada authorization does not guarantee provincial coverage or access to a treatment centre. Confidence/conflicts: High for Canadian TDT authorization; payer and center implementation remain gaps. Availability/reimbursement outside the approving regulator not established.
  • exagamglogene autotemcel (Casgevy)[11]CADTH reimbursement recommendationdocumented homozygous or compound heterozygous beta-thalassemia, transfusion-dependence criteria per reimbursement review; Canadian public-drug-plan reimbursement-with-conditions context for TDT age 12+. · CDA-AMC recommendations are nonbinding for provincial/territorial plans. The recommendation includes one-time/no-retreatment and cost-reduction conditions; access also depends on specialized-centre capacity. Confidence/conflicts: Medium-high for reimbursement recommendation; local implementation remains source-pending.
  • exagamglogene autotemcel (Casgevy)[11]CADTH reimbursement recommendationdocumented homozygous or compound heterozygous beta-thalassemia, transfusion-dependence criteria per reimbursement review; Canadian public-drug-plan reimbursement-with-conditions context for TDT age 12+. · CDA-AMC recommendations are nonbinding for provincial/territorial plans. The recommendation includes one-time/no-retreatment and cost-reduction conditions; access also depends on specialized-centre capacity. Confidence/conflicts: Medium-high for reimbursement recommendation; local implementation remains source-pending.
  • exagamglogene autotemcel (Casgevy)[11]CADTH reimbursement recommendationdocumented homozygous or compound heterozygous beta-thalassemia, transfusion-dependence criteria per reimbursement review; Canadian public-drug-plan reimbursement-with-conditions context for TDT age 12+. · CDA-AMC recommendations are nonbinding for provincial/territorial plans. The recommendation includes one-time/no-retreatment and cost-reduction conditions; access also depends on specialized-centre capacity. Confidence/conflicts: Medium-high for reimbursement recommendation; local implementation remains source-pending.
  • luspatercept (Reblozyl)[12]Health Canada approvedbeta-thalassemia-associated RBC transfusion-dependent anemia; adult Canadian patients with red blood cell transfusion-dependent anemia associated with beta-thalassemia. · Product-monograph and SBD sources establish authorization, not provincial coverage or individual suitability. Pediatric use for beta-thalassemia was not authorized in the fetched monograph. Confidence/conflicts: High for Canadian Reblozyl authorization; coverage remains source-pending. Availability/reimbursement outside the approving regulator not established.
  • luspatercept (Reblozyl)[12]Health Canada approvedbeta-thalassemia-associated RBC transfusion-dependent anemia; adult Canadian patients with red blood cell transfusion-dependent anemia associated with beta-thalassemia. · Product-monograph and SBD sources establish authorization, not provincial coverage or individual suitability. Pediatric use for beta-thalassemia was not authorized in the fetched monograph. Confidence/conflicts: High for Canadian Reblozyl authorization; coverage remains source-pending. Availability/reimbursement outside the approving regulator not established.
  • packed red blood cell transfusions; iron chelation therapy; allogeneic hematopoietic stem cell transplant; luspatercept[13]CADTH reimbursement recommendationbeta-thalassemia / transfusion-dependent phenotype; Canadian standard-of-care and comparator context for TDT. · This is health-technology-review clinical context, not a drug label or transplant referral rule. It should be paired with local hematology/transplant-center criteria and provincial coverage rules. Confidence/conflicts: Medium-high for Canadian standard-of-care context; local protocol and funding remain gaps.
  • packed red blood cell transfusions; iron chelation therapy; allogeneic hematopoietic stem cell transplant; luspatercept[13]CADTH reimbursement recommendationbeta-thalassemia / transfusion-dependent phenotype; Canadian standard-of-care and comparator context for TDT. · This is health-technology-review clinical context, not a drug label or transplant referral rule. It should be paired with local hematology/transplant-center criteria and provincial coverage rules. Confidence/conflicts: Medium-high for Canadian standard-of-care context; local protocol and funding remain gaps.

출처

  1. U.S. Food and Drug Administration — official drug label · official drug label
  2. European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
  3. European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
  4. European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
  5. Therapeutic Goods Administration (TGA) — Australian Public Assessment Report · Australian Public Assessment Report
  6. Healthdirect Australia — Australian public health information · Australian public health information
  7. Thai National Drug Information / Thai FDA-MOPH — SmPC PDF / regulator drug-information repository · SmPC PDF / regulator drug-information repository
  8. Thai National Drug Information / Thai FDA-MOPH — national essential medicines list PDF / access policy · national essential medicines list PDF / access policy
  9. Thai National Drug Information / Thai FDA-MOPH — product information PDF / regulator drug-information repository · product information PDF / regulator drug-information repository
  10. Health Canada Drug and Health Product Portal — Summary Basis of Decision · Summary Basis of Decision
  11. CDA-AMC / CADTH via NCBI Bookshelf — reimbursement recommendation / health technology review · reimbursement recommendation / health technology review
  12. Health Canada Drug and Health Product Portal — Summary Basis of Decision · Summary Basis of Decision
  13. CDA-AMC / CADTH via NCBI Bookshelf — clinical review / health technology assessment · clinical review / health technology assessment

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 2026-06-12.