镰状细胞病及相关疾病
Sickle cell disease
近两年的全球研究 7,963L2
2024/10 起在 Europe PMC 检索所得，按发表时间倒序显示最近 20 篇。标题未译成中文——自动翻译需要接入 LLM 服务，尚未引入。
- 2026-10Exploring the Patient Perspective of Gene Therapy for Sickle Cell Disease
- 2026-10Brief Cognitive Assessment in Sickle Cell Care: Psychometric Properties of the RUDAS Across Two Languages
- 2026-10Reframing sickle cell disease care: improving care with an embedded primary care model for chronic comorbidity management
- 2026-10Abstracts
- 2026-10综述开放获取Ovarian Reserve in Women of Reproductive Age With Sickle Cell Anaemia: A Scoping Review
- 2026-10Maternal and perinatal outcomes associated with prophylactic erythrocytapheresis in pregnant women with sickle cell disease
- 2026-10Integrating sexual health into sickle cell disease care
- 2026-10From approval to practice: are clinicians and health systems ready for gene therapy in sickle cell disease?
- 2026-10Flipping the fetal switch across β-hemoglobinopathies
- 2026-10Bimacular Avascular Zone in a Woman Aged 39 Years With Sickle Cell Disease
- 2026-10开放获取Small populations, big impact: leveraging rare disease gene therapies to benefit millions
- 2026-10综述Red blood cell transfusion in sickle cell disease: From clinical necessity to immunological complexity
- 2026-10开放获取Real-World Claims Data on Prescribed Drugs and the Associated Drug-Drug Interaction Risk in Sickle Cell Disease in the United States
- 2026-10开放获取Teaching Cell and Gene Therapy in Genetic Counseling Programs: Survey Findings and Suggested Practical Resources for Integration
- 2026-10开放获取The Role of Ferroptosis Induced by Iron Overload in Osteoblast and Osteoclast Function
- 2026-10开放获取Kidney Structure and Blood Flow Predict Function and Enable Image-Based eGFR With Non-Contrast MRI
- 2026-10Decision-Making in IVF: Restrictions on PGT-M Positive Embryo Transfers-A Qualitative Study
- 2026-10综述Paediatric Drug Optimization for Sickle Cell Disease: priorities for research and development in children
- 2026-10Predicting Non-Target Neutrophil Collection During HPC(A): Implications for Research Protocols in Apheresis
- 2026-09开放获取Assessing Quality of Life and Associated Factors Among Caregivers of Children With Chronic Conditions at Tamale Teaching Hospital: A Cross-Sectional Study
境外已获批用于本病的药物 8L2
欧盟 2 项、美国 6 项。同一药物在两地各批一次的，会分别列出。
「境外已获批」不等于「在中国能用」。中间隔着进口注册、临床急需境外新药通道、海南博鳌乐城国际医疗旅游先行区等几条路径，各有各的条件与费用。这一节能确定地告诉你的只有一件事：这个病在世界范围内已经有获得批准的药物，它叫什么名字。拿这个名字去问主治医生，是下一步最省力的做法。
药名一律保留英文原文，不作翻译——中国的药品通用名与英文名的音译经常不一致，译错会让人去找一个不存在的药。
- Xromi欧盟2019-07-01hydroxycarbamide官方记录
- Casgevy欧盟2024-02-09exagamglogene autotemcel官方记录
- Endari美国2017-07-07L-glutamine官方记录
- Siklos美国2017-12-21hydroxycarbamide (hydroxyurea)官方记录
- ADAKVEO美国2019-11-15crizanlizumab官方记录
- Oxbryta美国2019-11-25Voxelotor官方记录
- Casgevy美国2023-12-08exagamglogene autotemcel官方记录
- Lyfgenia美国2023-12-08lovotibeglogene autotemcel官方记录
尚未获批的在研药物（73 项）
这些药已被欧盟或美国的监管机构认定为罕见病用药（英文 orphan drug designation，中文行业里通称「孤儿药资格」——「孤儿」说的是这类药市场太小、没有厂商愿意认领，不是在说病人）。但这只是一种监管身份：意味着监管机构给予研发上的激励，不代表这个药已被证明有效，也不代表将来一定能上市，绝大多数最终不会成药。列在这里是为了看清有哪些方向正在被尝试。
- Extract of Sorghum bicolour leaf, Pterocarpus osun stem, Piper guineen欧盟2005-08-26Treatment of sickle cell disease官方记录
- 2,2-Dimethylbutyric acid, sodium salt欧盟2009-03-18Treatment of sickle cell disease官方记录
- pegylated carboxyhaemoglobin欧盟2009-11-26Treatment of sickle cell disease官方记录
- Deferiprone欧盟2011-02-23Treatment of sickle cell disease官方记录
- human haptoglobin欧盟2011-12-09Treatment of sickle cell disease官方记录
- human erythrocytes encapsulating inositol hexaphosphate欧盟2012-07-04Treatment of sickle cell disease官方记录
- Xyndari欧盟2012-07-04LevoglutamideTreatment of sickle cell disease官方记录
- sevuparin sodium欧盟2015-02-12Treatment of sickle cell disease官方记录
- Oxbryta欧盟2016-11-182-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde (voxelotor)Treatment of sickle cell disease官方记录
- decitabine;tetrahydrouridine欧盟2017-06-20Treatment of sickle cell disease官方记录
- sirolimus欧盟2018-01-17Treatment of sickle cell disease官方记录
- Docosahexaenoic acid ethyl ester欧盟2018-03-21Treatment of sickle cell disease官方记录
- benserazide hydrochloride欧盟2019-01-11The treatment of sickle cell disease官方记录
- Hemopexin欧盟2020-07-27Treatment of sickle cell disease官方记录
- (S)-1-(5-((2,3-dihydro-[1,4]dioxino[2,3-b]pyridin-7-yl)sulfonyl)-3,4,5欧盟2020-10-19Treatment of sickle cell disease官方记录
- 2-(2-{[2-(1h-benzimidazol-2-yl)ethyl]amino}ethyl)-n-[(3-fluoropyridin-欧盟2021-02-19Treatment of sickle cell disease官方记录
- autologous CD34+ hematopoietic stem and progenitor cells transfected w欧盟2021-02-19Treatment of sickle cell disease官方记录
- Epeleuton欧盟2022-10-11Treatment of sickle cell disease官方记录
- nicotinamide mononucleotide欧盟2023-06-20Treatment of sickle cell disease官方记录
- tarperprumig欧盟2023-07-25Treatment of sickle cell disease官方记录
- mitapivat sulfate欧盟2024-12-13Treatment of sickle cell disease官方记录
- nangibotide欧盟2025-11-21Treatment of sickle cell disease官方记录
- poloxamer 188 (purified)美国1989-06-27Treatment of sickle cell disease (this includes the treatment and prevention of complications of sickle cell disease)官方记录
- Synthetic derivative of 16-hydroxy-9Z, 12Z, 14E-octadecatrienoic acid美国1991-10-24Prophylactic treatment of sickle cell disease.官方记录
- Isobutyramide美国1994-05-25Treatment of sickle cell disease and beta thalassemia.官方记录
- Clotrimazole美国1995-04-24Treatment of sickle cell disease.官方记录
- Bis(4-fluorophenyl)phenylacetamide美国2000-03-02Treatment of sickle cell disease.官方记录
- niprisan美国2003-08-15Treatment of sickle cell disease官方记录
- oral unfractionated heparin美国2004-01-29Treatment of sickle cell disease官方记录
- 5-hydroxymethyl-2-furfuraldehyde美国2006-05-26Treatment of sickle cell disease官方记录
- sodium 2, 2 dimethylbutyrate美国2008-07-25Treatment of sickle cell disease.官方记录
- pentosan polysulfate sodium美国2008-11-21Treatment of sickle cell disease.官方记录
- (1R,3R,4R,5S)-3-O-[2-O-benzoyl-3-O-(sodium(2S)-3-cyclohexyl-propanoate美国2009-02-17Treatment of vaso-occlusive crisis in patients with sickle cell disease.官方记录
- pegylated carboxyhemoglobin美国2010-10-21Treatment of acute painful sickling crises in patients with sickle cell disease官方记录
- carbon monoxide美国2012-09-28Treatment of sickle cell disease官方记录
- extract of sorghum bicolor extract美国2012-11-19Treatment of sickle cell disease官方记录
- recombinant humanized IgG1k monoclonal antibody to human invariant T c美国2013-04-12Treatment of sickle cell disease官方记录
- human haptoglobin美国2013-11-19Treatment of sickle cell disease官方记录
- pegylated carboxyhemoglobin bovine美国2015-01-15Treatment of sickle cell disease官方记录
- hydroxyurea美国2015-03-16Treatment of symptomatic sickle cell disease in pediatric patients, less than 17 years of age.官方记录
另有 33 项未列出。
数据来自欧洲药品管理局（EMA）的药品与罕见病用药资格公开导出表，以及美国 FDA 的罕见病用药资格数据库（Orphan Drug Product Designation Database）。两边口径不同：欧盟一侧取的是当前状态仍为「已授权」的药品；美国一侧记录的是「曾获批准」这一事实，FDA 的公开表不追踪药物此后是否退市（例如 Relyvrio 于 2024 年撤市，表中仍记为已获批）。请以官方记录页为准。
在中国开展的临床试验 7L2
按病名在 ClinicalTrials.gov 检索、并校验研究中心含中国大陆而来。登记状态不等于现在真的能入组——务必按 NCT 号到原站核实，并与主治医生商量。
登记为可入组 4
- 招募中NCT06399107Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease中国研究中心 1 个：Beijing
- 招募中NCT06565026CS-206 in Patients With Sickle Cell Disease中国研究中心 1 个：Nanning
- 尚未开始招募NCT07190001YOLT-204 in Patients With Hemoglobinopathies中国研究中心 1 个：Guangzhou
- 尚未开始招募NCT07000318CS-206 in Patients With Sickle Cell Disease中国研究中心 1 个：Shanghai
其他状态的试验（3 项）
- 已完成NCT00171821A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload中国研究中心 3 个：Guangzhou、Nanjing、Shanghai
- 状态未知NCT06300723Clinical Study of BRL-101 in Severe SCD中国研究中心 1 个：Nanning
- 已完成NCT06581627A Research Study Looking at a Single Dose of Etavopivat in Healthy Chinese Participants中国研究中心 1 个：Beijing
中国境外的在招试验 217L2
这些试验在中国没有研究中心，通常无法直接报名——入组一般要求在当地居住并接受随访。列在这里是因为它另有用处：看清楚全世界正在试哪些药、做到了哪一期、由谁在做。把药名和 NCT 编号记下来去问主治医生，或据此进一步查该药是否已在境外获批、是否有拓展性用药（expanded access）通道。
美国119法国31英国10加拿大9Nigeria8意大利8西班牙8德国7土耳其7Uganda6比利时6埃及5巴西5Kenya5另有 29 个国家/地区
CT.gov 报告命中 217 项，此处取回并展示最近的 15 项。
- 尚未开始招募NCT07540767Development of a Provider-Focused Intervention to Improve Health Outcomes in Pediatric Sickle Cell Disease美国
- 尚未开始招募NCT07797582MBI for Pain and Sleep in Adolescents and Young Adults With Sickle Cell Disease
- 尚未开始招募NCT05605366Minocycline In Neurocognitive Outcomes - Sickle Cell Disease
- 尚未开始招募NCT07530965Omega-3-Fatty Acids in Children With Sickle Cell Disease
- 尚未开始招募NCT07648043Feasibility and Acceptability of Amani美国
- 尚未开始招募NCT07826988Adaptive Platform Trial for Pain Management in Sickle Cell Vaso-Occlusive Crisis
- 尚未开始招募NCT07799571Buprenorphine for the Management of Pain in Children With Sickle Cell Disease美国
- 尚未开始招募NCT07774351A Clinical Exploratory Study of YOLT-204 in the Treatment of Hemoglobinopathies
- 招募中NCT07369024Sub-dissociative Dose Ketamine in Treatment of Vaso-occlusive Pain Event in Children and Young Adults美国
- 尚未开始招募NCT07703566CPAP for Hypoxemic Acute Chest Syndrome in Sickle Cell Disease
- 尚未开始招募NCT07782489Pleuropulmonary Ultrasound Imaging in Patients With Sickle Cell Disease in a Vaso-occlusive Crisis. "ECHODREP"
- 尚未开始招募NCT07217548Solving Stigma Through POV Simulation: Enhancing Pharmacist Empathy-based Practices With Sickle Cell Disease Patients美国
- 尚未开始招募NCT07802210Effectiveness of Innovative Educational Methods in Empowering Children With Sickle Cell DiseaseIraq
- 尚未开始招募NCT07752043Standard of Care Comparative Arm of Phase 1/2 Gene Therapy Trial DREPAMIR" in Severe Sickle Cell Disease Patients法国
- 招募中NCT07616154Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease美国
外部标识与链接
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本页数据来源
- 疾病定义、同义词、基因、表型、流行病学：Orphanet（CC BY 4.0）
- 中文病名：Orphanet 中文包，冻结于 2020-06-01
- 表型中文标签：HPO 简体中文翻译（CHPO 上游成果）