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Sickle cell disease: 국가별 선택지

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선택지 정리됨희귀·유전 질환최종 확인 2026.06

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임상시험 검색어

Sickle cell disease 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

Exagamglogene autotemcel / Casgevy; lovotibeglogene autotemcel / Lyfgenia [1]FDA-approvedSCD diagnosis; gene therapy labels specify SCD and recurrent VOC/VOE history rather than a single genotype in FDA notice; Patients aged 12 years and older with recurrent VOCs for Casgevy; patients aged 12 years and older with history of VOEs for Lyfgenia; Oxbryta no longer recorded as current U.S. access. · Both gene therapies require stem-cell collection, myeloablative conditioning, infusion, and long-term follow-up. Lyfgenia has a hematologic malignancy boxed-warning caveat in the FDA notice. Oxbryta should not be represented as currently available. Confidence/conflicts: High for U.S. gene therapy approval and Oxbryta withdrawal status.
Exagamglogene autotemcel / Casgevy; lovotibeglogene autotemcel / Lyfgenia [1]FDA-approvedSCD diagnosis; gene therapy labels specify SCD and recurrent VOC/VOE history rather than a single genotype in FDA notice; Patients aged 12 years and older with recurrent VOCs for Casgevy; patients aged 12 years and older with history of VOEs for Lyfgenia; Oxbryta no longer recorded as current U.S. access. · Both gene therapies require stem-cell collection, myeloablative conditioning, infusion, and long-term follow-up. Lyfgenia has a hematologic malignancy boxed-warning caveat in the FDA notice. Oxbryta should not be represented as currently available. Confidence/conflicts: High for U.S. gene therapy approval and Oxbryta withdrawal status.
Exagamglogene autotemcel / Casgevy; lovotibeglogene autotemcel / Lyfgenia [1]FDA-approvedSCD diagnosis; gene therapy labels specify SCD and recurrent VOC/VOE history rather than a single genotype in FDA notice; Patients aged 12 years and older with recurrent VOCs for Casgevy; patients aged 12 years and older with history of VOEs for Lyfgenia; Oxbryta no longer recorded as current U.S. access. · Both gene therapies require stem-cell collection, myeloablative conditioning, infusion, and long-term follow-up. Lyfgenia has a hematologic malignancy boxed-warning caveat in the FDA notice. Oxbryta should not be represented as currently available. Confidence/conflicts: High for U.S. gene therapy approval and Oxbryta withdrawal status.
voxelotor / Oxbryta [2]Withdrawn worldwide (Sep 2024)SCD diagnosis; gene therapy labels specify SCD and recurrent VOC/VOE history rather than a single genotype in FDA notice; Patients aged 12 years and older with recurrent VOCs for Casgevy; patients aged 12 years and older with history of VOEs for Lyfgenia; Oxbryta no longer recorded as current U.S. access. · Voxelotor (Oxbryta) was voluntarily withdrawn from all worldwide markets in September 2024 for a safety signal (excess vaso-occlusive crises and deaths, including pediatric) and is NOT currently available or FDA-approved; it has not returned as of 2026. The FDA-approved/available options in this record are Casgevy and Lyfgenia ONLY.
voxelotor / Oxbryta [2]Withdrawn worldwide (Sep 2024)SCD diagnosis; gene therapy labels specify SCD and recurrent VOC/VOE history rather than a single genotype in FDA notice; Patients aged 12 years and older with recurrent VOCs for Casgevy; patients aged 12 years and older with history of VOEs for Lyfgenia; Oxbryta no longer recorded as current U.S. access. · Voxelotor (Oxbryta) was voluntarily withdrawn from all worldwide markets in September 2024 for a safety signal (excess vaso-occlusive crises and deaths, including pediatric) and is NOT currently available or FDA-approved; it has not returned as of 2026. The FDA-approved/available options in this record are Casgevy and Lyfgenia ONLY.
voxelotor / Oxbryta [2]Withdrawn worldwide (Sep 2024)SCD diagnosis; gene therapy labels specify SCD and recurrent VOC/VOE history rather than a single genotype in FDA notice; Patients aged 12 years and older with recurrent VOCs for Casgevy; patients aged 12 years and older with history of VOEs for Lyfgenia; Oxbryta no longer recorded as current U.S. access. · Voxelotor (Oxbryta) was voluntarily withdrawn from all worldwide markets in September 2024 for a safety signal (excess vaso-occlusive crises and deaths, including pediatric) and is NOT currently available or FDA-approved; it has not returned as of 2026. The FDA-approved/available options in this record are Casgevy and Lyfgenia ONLY.

European Union

Exagamglogene autotemcel / Casgevy [3]EMA authorisedSevere SCD; VOC history/managed-access criteria per NICE scope; voxelotor not a current EU option after suspension/withdrawal; Severe SCD aged 12 years and over for Casgevy in NICE managed-access context; Oxbryta safety suspension/withdrawal context for EU. · NICE managed access is not the same as permanent routine commissioning. EMA Oxbryta recommendation means new patients should not be started and alternatives should be discussed by clinicians. Confidence/conflicts: High for NICE managed-access and EMA Oxbryta suspension/withdrawal caveats. Casgevy holds an EU conditional marketing authorisation; the prior 'restricted' badge reflected the now-removed voxelotor entry.
Exagamglogene autotemcel / Casgevy [3]EMA authorisedSevere SCD; VOC history/managed-access criteria per NICE scope; voxelotor not a current EU option after suspension/withdrawal; Severe SCD aged 12 years and over for Casgevy in NICE managed-access context; Oxbryta safety suspension/withdrawal context for EU. · NICE managed access is not the same as permanent routine commissioning. EMA Oxbryta recommendation means new patients should not be started and alternatives should be discussed by clinicians. Confidence/conflicts: High for NICE managed-access and EMA Oxbryta suspension/withdrawal caveats. Casgevy holds an EU conditional marketing authorisation; the prior 'restricted' badge reflected the now-removed voxelotor entry.
voxelotor / Oxbryta [4]EMA authorisation suspended / withdrawnSevere SCD; VOC history/managed-access criteria per NICE scope; voxelotor not a current EU option after suspension/withdrawal; Severe SCD aged 12 years and over for Casgevy in NICE managed-access context; Oxbryta safety suspension/withdrawal context for EU. · Voxelotor (Oxbryta) is NOT a current option: Pfizer withdrew it worldwide on 25 Sep 2024; the EU marketing authorisation was suspended (European Commission decision made final 9 Dec 2025, following CHMP confirmation 17 Oct 2025) and it was withdrawn from the UK market 8 Oct 2024 — due to higher rates of death and vaso-occlusive crises. It should no longer be prescribed; patients previously on it should discuss alternatives with their clinician. Casgevy's genuine EU/UK authorisation status is unchanged.
voxelotor / Oxbryta [4]EMA authorisation suspended / withdrawnSevere SCD; VOC history/managed-access criteria per NICE scope; voxelotor not a current EU option after suspension/withdrawal; Severe SCD aged 12 years and over for Casgevy in NICE managed-access context; Oxbryta safety suspension/withdrawal context for EU. · Voxelotor (Oxbryta) is NOT a current option: Pfizer withdrew it worldwide on 25 Sep 2024; the EU marketing authorisation was suspended (European Commission decision made final 9 Dec 2025, following CHMP confirmation 17 Oct 2025) and it was withdrawn from the UK market 8 Oct 2024 — due to higher rates of death and vaso-occlusive crises. It should no longer be prescribed; patients previously on it should discuss alternatives with their clinician. Casgevy's genuine EU/UK authorisation status is unchanged.

Australia

acute vaso-occlusive-crisis supportive care; analgesia; hydration; oxygen where indicated; blood transfusion where indicated; chronic transfusion and/or hydroxyurea background context [5]Standard option (per Perth Children's Hospital / Child and Adolescent Health Service WA)sickle hemoglobin / HbS context; pediatric emergency department management of SCD pain/VOC and complications in Western Australia. · This is an emergency guideline from one Australian pediatric hospital and should not be generalized as a national formulary or adult-care pathway. It does not establish approval for any branded product. Confidence/conflicts: Medium-high for WA pediatric acute-care context; national and adult Australia pathways remain gaps.
hydroxyurea/hydroxycarbamide; pain management; regular blood transfusions; bone marrow or stem cell transplant where appropriate [6]Standard option (per Rare Awareness Rare Education Portal / Rare Voices Australia)inherited hemoglobin beta-globin / sickle hemoglobin context; Australian SCD management and supportive-care context. · The RARE Portal is educational and does not establish medication registration, PBS listing, transplant eligibility, or individual suitability. It advises discussion with the medical team. Confidence/conflicts: Medium-high for Australian supportive-management categories; product-specific Australian approvals remain separate gaps.
hydroxyurea/hydroxycarbamide; pain management; regular blood transfusions; bone marrow or stem cell transplant where appropriate [6]Standard option (per Rare Awareness Rare Education Portal / Rare Voices Australia)inherited hemoglobin beta-globin / sickle hemoglobin context; Australian SCD management and supportive-care context. · The RARE Portal is educational and does not establish medication registration, PBS listing, transplant eligibility, or individual suitability. It advises discussion with the medical team. Confidence/conflicts: Medium-high for Australian supportive-management categories; product-specific Australian approvals remain separate gaps.

Thailand

Deferasirox (Deferasirox Teva)[7]ApprovedSickle cell disease; chronic iron overload after transfusion support.; Chronic transfusional iron overload in other anemias, with sickle cell disease named in the clinical-evidence population; not a disease-modifying SCD therapy. · The indication is framed as chronic iron overload in other anemias, not an SCD-specific disease-modifying label. It does not establish hydroxyurea, gene therapy, transfusion-protocol, or acute VOC access in Thailand. Confidence/conflicts: Medium-high for Thailand label support for transfusional iron overload in other anemias and clinical evidence including SCD; not a direct SCD disease-modifying approval. No conflict recorded. Availability/reimbursement outside the approving regulator not established.

Canada

exagamglogene autotemcel (Casgevy)[8]EMA authorisedsevere SCD with recurrent vaso-occlusive crises; autologous CRISPR/Cas9-edited CD34+ cell therapy targeting BCL11A enhancer to increase fetal hemoglobin; Canadian SCD patients aged 12 years and older with recurrent VOCs; treatment-centre and HSCT-appropriateness requirements apply. · The monograph describes mobilization, apheresis, full myeloablative conditioning, rescue-cell backup, and treatment-centre requirements. It is not a simple outpatient medication and does not establish provincial access or individual eligibility. Confidence/conflicts: High for Canadian authorization and label logistics; payer implementation remains separately tracked. Availability/reimbursement outside the approving regulator not established.
exagamglogene autotemcel (Casgevy)[8]EMA authorisedsevere SCD with recurrent vaso-occlusive crises; autologous CRISPR/Cas9-edited CD34+ cell therapy targeting BCL11A enhancer to increase fetal hemoglobin; Canadian SCD patients aged 12 years and older with recurrent VOCs; treatment-centre and HSCT-appropriateness requirements apply. · The monograph describes mobilization, apheresis, full myeloablative conditioning, rescue-cell backup, and treatment-centre requirements. It is not a simple outpatient medication and does not establish provincial access or individual eligibility. Confidence/conflicts: High for Canadian authorization and label logistics; payer implementation remains separately tracked. Availability/reimbursement outside the approving regulator not established.
exagamglogene autotemcel (Casgevy)[9]CADTH reimbursement recommendationsevere SCD genotype and recurrent VOC history per reimbursement conditions; Canadian public-drug-plan reimbursement-with-conditions context for severe SCD with recurrent VOCs. · CDA-AMC reimbursement recommendations do not guarantee provincial/territorial implementation. The recommendation includes specialist prescribing, one-time treatment/no retreatment, and cost-reduction conditions. Confidence/conflicts: Medium-high for Canadian reimbursement recommendation; local implementation and center capacity remain gaps.
exagamglogene autotemcel (Casgevy)[9]CADTH reimbursement recommendationsevere SCD genotype and recurrent VOC history per reimbursement conditions; Canadian public-drug-plan reimbursement-with-conditions context for severe SCD with recurrent VOCs. · CDA-AMC reimbursement recommendations do not guarantee provincial/territorial implementation. The recommendation includes specialist prescribing, one-time treatment/no retreatment, and cost-reduction conditions. Confidence/conflicts: Medium-high for Canadian reimbursement recommendation; local implementation and center capacity remain gaps.
exagamglogene autotemcel (Casgevy)[9]CADTH reimbursement recommendationsevere SCD genotype and recurrent VOC history per reimbursement conditions; Canadian public-drug-plan reimbursement-with-conditions context for severe SCD with recurrent VOCs. · CDA-AMC reimbursement recommendations do not guarantee provincial/territorial implementation. The recommendation includes specialist prescribing, one-time treatment/no retreatment, and cost-reduction conditions. Confidence/conflicts: Medium-high for Canadian reimbursement recommendation; local implementation and center capacity remain gaps.
hydroxyurea/hydroxycarbamide; HLA-matched hematopoietic stem cell transplant (HSCT) background context [8]Health Canada approvedbeta-globin sickle hemoglobin context; Canadian severe SCD background-care context used in Health Canada's Casgevy review. · This is background context from a regulator review, not a product monograph for hydroxyurea in SCD and not a personalized transplant recommendation. It explicitly notes hydroxyurea is not specifically authorized in Canada for SCD. Confidence/conflicts: Medium-high for Canadian background-care context; hydroxyurea label/reimbursement details require separate source verification.
hydroxyurea/hydroxycarbamide; HLA-matched hematopoietic stem cell transplant (HSCT) background context [8]Health Canada approvedbeta-globin sickle hemoglobin context; Canadian severe SCD background-care context used in Health Canada's Casgevy review. · This is background context from a regulator review, not a product monograph for hydroxyurea in SCD and not a personalized transplant recommendation. It explicitly notes hydroxyurea is not specifically authorized in Canada for SCD. Confidence/conflicts: Medium-high for Canadian background-care context; hydroxyurea label/reimbursement details require separate source verification.

출처

U.S. Food and Drug Administration — regulator approval notice · regulator approval notice
U.S. Food and Drug Administration — regulator approval notice · regulator approval notice
European Medicines Agency (EMA) — regulator safety/suspension notice · regulator safety/suspension notice
European Medicines Agency (EMA) — regulator safety/suspension notice · regulator safety/suspension notice
Perth Children's Hospital / Child and Adolescent Health Service WA — hospital emergency department guideline · hospital emergency department guideline
Rare Awareness Rare Education (RARE) Portal / Rare Voices Australia — Australian rare-disease educational portal · Australian rare-disease educational portal
Thai National Drug Information / Thai FDA-MOPH — SmPC PDF / regulator drug-information repository · SmPC PDF / regulator drug-information repository
Health Canada Drug and Health Product Portal — Summary Basis of Decision · Summary Basis of Decision
CDA-AMC / Canada's Drug Agency — reimbursement recommendation / health technology review · reimbursement recommendation / health technology review

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.