阵发性睡眠性血红蛋白尿症
Paroxysmal nocturnal hemoglobinuria
定义 英文原文（暂无中文）
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal hematopoietic stem cell disorder characterized by corpuscular hemolytic anemia, bone marrow failure and frequent thrombotic events.
别名
Marchiafava-Micheli病
基本事实
- 遗传方式
- 不适用
- 发病年龄
- 各年龄段
- 患病率
- 1-9 / 1 000 000（United Kingdom）
相关基因 1
| 基因 | 名称 | 关联类型 |
|---|---|---|
| PIGA | phosphatidylinositol glycan anchor biosynthesis class A | Disease-causing somatic mutation(s) in |
临床表型 44
必现 100%1
- 红细胞酶水平或活性异常 HP:0030272
极常见 99–80%4
- 贫血 HP:0001903
- 虚弱 HP:0025406
- 血红蛋白尿 HP:0003641
- 溶血性贫血 HP:0001878
常见 79–30%17
- 胸痛 HP:0100749
- 慢性肾病 HP:0012622
- 结膜黄疸 HP:0032106
- 循环铁水平降低 HP:0040303
- 深静脉血栓形成 HP:0002625
- 呼吸困难 HP:0002094
- 阵发性腹痛 HP:0002574
- 红斑性肢痛症 HP:0032147
- 头痛 HP:0002315
- 含铁血黄素尿症 HP:0012543
- 血液尿素氮升高 HP:0003138
- 乳酸脱氢酶活性增高 HP:0025435
- 结合珠蛋白水平降低 HP:0020181
- 网织红细胞增多症 HP:0001923
- 血栓栓塞症 HP:0001907
- 高非结合胆红素血症 HP:0008282
- 静脉血栓形成 HP:0004936
偶见 29–5%18
- 急性肾损伤 HP:0001919
- 动脉血栓形成 HP:0004420
- Budd-Chiari综合征 HP:0002639
- 红系增生 HP:0012132
- 高血压 HP:0000822
- 阳痿 HP:0000802
- 黄疸 HP:0000952
- 昏睡 HP:0001254
- 白细胞减少症 HP:0001882
- 肠系膜静脉血栓形成 HP:0030248
- 心肌梗死 HP:0001658
- 吞咽疼痛 HP:0032043
- 全血细胞减少症 HP:0001876
- 蛋白尿 HP:0000093
- 肺栓塞 HP:0002204
- 肾功能不全 HP:0000083
- 卒中 HP:0001297
- 血小板减少症 HP:0001873
罕见 <4–1%4
- 吞咽困难 HP:0002015
- 食管痉挛 HP:0025271
- 糖尿 HP:0003076
- 肾范可尼综合征 HP:0001994
近两年的全球研究 1,050L2
2024/08 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。
- 2026-08综述Complement inhibitor therapy should be available to patients with paroxysmal nocturnal hemoglobinuria throughout the world
- 2026-07A Population-Based Study on Childhood Aplastic Anemia-Incidence, Outcomes, and Health-Related Quality of Life
- 2026-07Terminal complement inhibition is associated with renal, hematologic, and thrombotic complications during septic shock: A global propensity score-matched cohort study
- 2026-07Eltrombopag Added to Standard Immunosuppressive Treatment as Front-Line Therapy for Severe Aplastic Anemia: Long-Term Outcomes of the Phase-3 Randomized Superiority EBMT-SAAWP RACE Study
- 2026-07Prophylaxis and management of thromboembolism in pnh patients: an italian survey
- 2026-07Switching between complement inhibitors in paroxysmal nocturnal hemoglobinuria: Analysis of strategy, efficacy, and safety
- 2026-07Lifelong phylogenetic reconstruction of immune-mediated clonal trajectories in paroxysmal nocturnal hemoglobinuria
- 2026-07Efficacy and safety of HSK39297 monotherapy in patients with paroxysmal nocturnal hemoglobinuria
- 2026-07病例报告Concurrent Loss of PIGA and ZRSR2 in a Patient With Paroxysmal Nocturnal Hemoglobinuria and Myelodysplastic Neoplasm
- 2026-07AI-driven diagnostic algorithm enhances early detection of paroxysmal nocturnal hemoglobinuria in real-world settings
- 2026-07病例报告Allogeneic haematopoietic stem cell transplantation for refractory perforating intestinal Behçet disease in a patient with aplastic anaemia: A case report
- 2026-07Magnetic resonance imaging features of renal hemosiderosis in acute kidney injury caused by paroxysmal nocturnal hemoglobinuria
- 2026-06A case report of CD4<sup>-</sup> CD8<sup>+</sup> CD56<sup>-</sup> TCRγδ<sup>+</sup> T-large granular lymphocytic leukaemia with severe aplastic anemia and a PNH clone
- 2026-06Academic point-of-care anti-CD19 CAR T-cell therapy induces durable remission and transient paroxysmal nocturnal hemoglobinuria-type cell expansion in a patient with multi-refractory immune thrombocytopenia
- 2026-06Clinical characteristics and outcomes of pediatric paroxysmal nocturnal hemoglobinuria in Egypt: a real-world cohort study including ravulizumab
- 2026-06病例报告开放获取Cardiovascular Complications of Anaplasmosis: A Case of Acute Pulmonary Embolism and Literature Review
- 2026-06开放获取Time-Related Burden of Treatment With Parenterally Administered Complement Inhibitors: A Mixed Methods Observational Study Exploring Experiences among Patients With Paroxysmal Nocturnal Hemoglobinuria
- 2026-06开放获取Adult <i>TWIST2</i>-high B-ALL confirms metabolic association but reveals molecular heterogeneity
- 2026-06综述Efficacy and Safety of the C3 Inhibitor Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria: A Systematic Review and Meta-Analysis
- 2026-06Response to "Comment on 'Consensus of the hematology society of Taiwan on the management of paroxysmal nocturnal hemoglobinuria (PNH)'"
国家医保药品目录中点名本病的药品 2L2
出自《国家基本医疗保险、生育保险和工伤保险药品目录（2025年）》（医保发〔2025〕33号，2026-01-01 起执行）。下列药品在药品名称或限定支付范围里出现了本病的名称。
匹配不到 ≠ 不能报销。目录里只有约一成药品设了限定支付范围，其余按适应症正常使用同样可报销；本区块只能回答「目录有没有点名这个病」，不能回答「这个病有没有药能报销」。各省执行细则、双通道与单独支付范围另有规定，请以当地医保部门口径为准。
- 依库珠单抗注射液乙类谈判药品限：1.阵发性睡眠性血红蛋白尿症(PNH)的患者；2.非典型溶血性尿毒症综合征(aHUS)的患者；3.抗乙酰胆碱受体(AChR)抗体阳性的难治性全身型重症肌无力(gMG)成人患者。
- 盐酸伊普可泮胶囊乙类谈判药品限：1.阵发性睡眠性血红蛋白尿症(PNH)成人患者；2.C3肾小球病(C3G)成人患者。
境外已获批用于本病的药物 12L2
欧盟 6 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。
- Soliris欧盟2007-06-20eculizumab官方记录
- Ultomiris欧盟2019-07-02ravulizumab官方记录
- Aspaveli欧盟2021-12-13pegcetacoplan官方记录
- Voydeya欧盟2024-04-19danicopan官方记录
- Fabhalta欧盟2024-05-17iptacopan官方记录
- Piasky欧盟2024-08-22crovalimab官方记录
- Soliris美国2007-03-16eculizumab官方记录
- Ultomiris美国2018-12-21Ravulizumab-cwvz官方记录
- Empaveli美国2021-05-14pegcetacoplan官方记录
- Fabhalta美国2023-12-05iptacopan官方记录
- Voydeya美国2024-03-29danicopan官方记录
- Piasky美国2024-06-20crovalimab-akkz官方记录
已获孤儿药资格、尚未获批的在研药物（12 项）
孤儿药资格只是一种监管身份——它意味着监管机构认可这是罕见病用药并给予研发激励，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。
- S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-try欧盟2014-10-22Treatment of paroxysmal nocturnal haemoglobinuria官方记录
- recombinant protein derived from the saliva of the Ornithodoros moubat欧盟2016-08-29Treatment of paroxysmal nocturnal haemoglobinuria官方记录
- S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-try美国2014-10-09Treatment of paroxysmal nocturnal hemoglobinuria (PNH)官方记录
- nomacopan美国2016-09-08Treatment of paroxysmal nocturnal hemoglobinuria官方记录
- pozelimab美国2019-07-22Treatment of Paroxysmal Nocturnal Hemoglobinuria官方记录
- Pozelimab/Cemdisiran Combination美国2020-09-08Treatment of Patients with Paroxysmal Nocturnal Hemoglobinuria官方记录
- cemdisiran美国2022-02-10Treatment of paroxysmal nocturnal hemoglobinuria官方记录
- Recombinant IgG-fusion protein containing a humanized anti-C5 monoclon美国2022-07-20Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)官方记录
- Humanized immunoglobulin G4 monoclonal antibody directed against manna美国2022-07-28Treatment of Paroxysmal Nocturnal Hemoglobinuria官方记录
- Recombinant, Humanized, aglycosylated, IgG1 Kappa Anti-Properdin Monoc美国2022-10-05Treatment of Paroxysmal Nocturnal Hemoglobinuria官方记录
- Recombinant, Humanized, Aglycosylated, Immunoglobulin G1 kappa Anti-Fa美国2023-12-20Treatment of paroxysmal nocturnal hemoglobinuria官方记录
- small molecule inhibitor of complement factor B美国2024-12-11treatment of paroxysmal nocturnal hemoglobinuria官方记录
数据来自欧洲药品管理局（EMA）的药品与孤儿药资格公开导出表，以及美国 FDA 孤儿药资格数据库。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 49L2
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 18
- 招募中NCT04645199National Longitudinal Cohort of Hematological Diseases中国研究中心 1 个：Tianjin
- 招募中NCT07256301A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH)中国研究中心 2 个：Hangzhou、Tianjin
- 尚未开始招募NCT06543459To Evaluate the Effect of Single Oral Dose of MY008211A Tablets on QTc Interval in Healthy Subjects中国研究中心 1 个：Beijing
- 招募中NCT06932471Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment中国研究中心 1 个：Beijing
- 招募中NCT06932744Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy中国研究中心 2 个：Beijing、Tianjin
- 招募中NCT06933914Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria中国研究中心 1 个：Tianjin
- 招募中NCT06745622Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria中国研究中心 1 个：Nanjing
- 招募中NCT06978699A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)中国研究中心 1 个：Tianjin
- 招募中NCT07152288Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment中国研究中心 1 个：Beijing
- 招募中NCT06931691A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China中国研究中心 10 个：Changchun、Guangzhou、Hefei、Jinan、Nanchang、Nantong 等 10 地
- 仅邀请入组NCT07657052A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria中国研究中心 1 个：Tianjin
- 尚未开始招募NCT07177859A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients中国研究中心 1 个：Tianjin
- 尚未开始招募NCT07266155Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)中国研究中心 1 个：Nanjing
- 招募中NCT07470762Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria中国研究中心 1 个：Hangzhou
- 招募中NCT07462780A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy Participants中国研究中心 1 个：Beijing
- 尚未开始招募NCT07177872A Long-term Efficacy and Safety of NTQ5082 Capsules中国研究中心 1 个：Tianjin
- 尚未开始招募NCT07760467To Evaluate Safety, Tolerability, PK/PD & Food Effect of Single/Multiple Oral TUL321 Capsules in Healthy Participants.中国研究中心 1 个：Changsha
- 尚未开始招募NCT07747090A Study of the Efficacy and Safety of FXS6837 Capsules Compared to Eculizumab for 24 Weeks in Patients With PNH.中国研究中心 1 个：Tianjin
其他状态的试验（31 项）
- 状态未知NCT01642979Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria中国研究中心 1 个：Tianjin
- 状态未知NCT01760096Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013)中国研究中心 1 个：Tianjin
- 状态未知NCT03866681Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH中国研究中心 1 个：Beijing
- 进行中·不再招募NCT04434092A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors中国研究中心 7 个：Beijing、Guangzhou、Hangzhou、Nanjing、Nantong、Shanghai 等 7 地
- 进行中·不再招募NCT04654468A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition中国研究中心 5 个：Chengdu、Guangzhou、Tianjin、Wuhan
- 已完成NCT04820530Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy中国研究中心 2 个：Beijing、Tianjin
- 进行中·不再招募NCT04747613Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria中国研究中心 2 个：Beijing、Tianjin
- 已完成NCT05125341Natural History Study of Adult Patients With Paroxysmal Nocturnal Hemoglobinuria of High-risk Hemolysis in China中国研究中心 1 个：Beijing
- 已完成NCT05642585A Study of Single-dose MY008211A in Healthy Adults中国研究中心 1 个：Changsha
- 进行中·不再招募NCT05133531A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment中国研究中心 1 个：Beijing
- 已完成NCT05828485Effect of Food on Pharmacokinetics of MY008211A Tablets in Healthy Adult Subjects中国研究中心 1 个：Changsha
- 进行中·不再招募NCT05476887To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104中国研究中心 4 个：Beijing、Nanjing、Tianjin、Zhengzhou
- 已完成NCT05828472A Study of Multiple Ascending Doses MY008211A in Healthy Adults中国研究中心 1 个：Changsha
- 已完成NCT05886244Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China中国研究中心 6 个：Beijing、Hangzhou、Nantong、Shanghai、Tianjin、Wuhan
- 已完成NCT06050226A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)中国研究中心 1 个：Tianjin
- 已完成NCT06154512A Real-world, Multi-center, Prospective, Observational Study for PNH in China中国研究中心 27 个：Beijing、Changchun、Changsha、Chengdu、Guangzhou、Guiyang 等 27 地
- 已完成NCT06051357Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria中国研究中心 2 个：Beijing、Tianjin
- 已完成NCT06294301A Study of Single and Multiple Doses of LP-005 in Healthy Adult Participants中国研究中心 1 个：Shanghai
- 进行中·不再招募NCT06238544Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria中国研究中心 2 个：Beijing、Tianjin
- 已完成NCT06490991Comparison of HRS-5965 Capsules and Tablets in Healthy Subjects中国研究中心 1 个：Shanghai
- 已完成NCT06561841A Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）中国研究中心 1 个：Nanjing
- 已完成NCT06852092Study on Mass Balance of [14C]HSK39297 in Healthy Chinese Adult Male Subjects中国研究中心 1 个：Suzhou
- 已完成NCT06578949Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)中国研究中心 7 个：Beijing、Guangzhou、Hangzhou、Nantong、Shanghai、Tianjin 等 7 地
- 已完成NCT06593938A Study to Evaluate the Efficacy and Safety of Oral HRS-5965 in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy中国研究中心 2 个：Beijing、Tianjin
- 进行中·不再招募NCT07212426Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in PNH Patients.中国研究中心 1 个：Nanjing
- 已完成NCT06715943Efficacy and Safety of HRS-5965 in Patients With PNH Who Are Still Anemia After Anti-C5 Antibody Treatment中国研究中心 2 个：Tianjin
- 进行中·不再招募NCT06764303A Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients中国研究中心 2 个：Hangzhou、Tianjin
- 已完成NCT06799546A Phase III Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy中国研究中心 2 个：Hangzhou、Nanjing
- 进行中·不再招募NCT06848296Phase Ib Clinical Study to Evaluate the Safety and Tolerability of VSA012 Injection in Paroxysmal Nocturnal Hemoglobinuria中国研究中心 2 个：Beijing、Hangzhou
- 已完成NCT07052838Efficacy and Safety of HSK39297 in Anti-C5 Treated PNH Patients With Anemia中国研究中心 2 个：Hangzhou、Nanjing
- 已完成NCT07035665A Clinical Study to Evaluate the Effect of Food on the Pharmacokinetic Profiles of HRS-5965 Capsules in Healthy Subjects中国研究中心 1 个：Shanghai
中国境外的在招试验 43L2
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9意大利7英国7韩国6加拿大5西班牙4法国4波兰3德国3澳大利亚3巴西2土耳其2哥伦比亚2荷兰2另有 18 个国家/地区
CT.gov 报告命中 43 项，此处取回并展示最近的 15 项。
- 尚未开始招募NCT05646563Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris
- 招募中NCT07416162A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy韩国
- 招募中NCT07154745A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently巴西、加拿大、意大利、波兰、韩国、西班牙、土耳其
- 尚未开始招募NCT05731050Study of NM8074 in Soliris-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- 招募中NCT07457151Danicopan PMS in Korea韩国
- 尚未开始招募NCT05646524Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy
- 招募中NCT07187401A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor加拿大、韩国、英国
- 尚未开始招募NCT07387302SLN12140 in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
- 招募中NCT07229235REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria意大利
- 尚未开始招募NCT06134414Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- 招募中NCT06934967Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients巴西、哥伦比亚、德国、意大利、荷兰、美国
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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）