视神经脊髓炎
Neuromyelitis optica spectrum disorder
定义 英文原文（暂无中文）
A rare inflammatory disease of the central nervous system characterized mainly by attacks of uni- or bilateral optic neuritis (ON) and acute myelitis.
别名
Devic病
基本事实
- 遗传方式
- 多基因/多因素
- 发病年龄
- 青少年期、成年期、儿童期、老年期
- 患病率
- 1-9 / 1 000 000（Martinique）
临床表型 17
极常见 99–80%10
- 自身免疫性抗体阳性 HP:0030057
- 膀胱功能异常 HP:0000009
- 脊髓炎 HP:0012486
- 中枢神经系统神经元损失 HP:0002529
- 眼痛 HP:0200026
- 视神经炎 HP:0100653
- 截瘫 HP:0010550
- 周围性脱髓鞘 HP:0011096
- 躯体感觉异常 HP:0003474
- 视力丧失 HP:0000572
常见 79–30%2
- 脑形态学异常 HP:0012443
- 抗水通道蛋白 4 抗体阳性 HP:0033342
偶见 29–5%5
- 抗髓磷脂少突胶质细胞糖蛋白抗体阳性 HP:0032492
- 脑脊液细胞增多 HP:0012229
- 恶心 HP:0002018
- 反复性嗝逆 HP:0100247
- 呼吸衰竭 HP:0002878
近两年的全球研究 1,977L2
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。
- 2026-08Complement C3 inhibitory peptide AMY-101 ameliorates brain injury in a mouse model of NMOSD
- 2026-08综述病例报告Wernicke's encephalopathy in mixed connective tissue disease triggered by persistent vomiting: a case report and literature review
- 2026-08Roles of cerebrospinal fluid metabolites in neuromyelitis optica spectrum disorder: Insights from Mendelian randomization
- 2026-08Update on the Incidence of Paediatric Multiple Sclerosis, Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease, Aquaporin-4 Antibody-Positive Neuromyelitis Optica Spectrum Disorder, and Other Acquired Demyelinating Syndromes in the Netherlands, 2017-2025, Including the COVID-19 Pandemic
- 2026-07Current landscape of pediatric optic neuritis management in India: A clinical practice survey
- 2026-07综述Consensus statement for the diagnosis and management of optic neuritis
- 2026-07病例报告Area postrema syndrome and longitudinally extensive transverse myelitis masquerading as leptomeningeal carcinomatosis
- 2026-07Terminal complement inhibition is associated with renal, hematologic, and thrombotic complications during septic shock: A global propensity score-matched cohort study
- 2026-07Evaluation of Dried Plasma Spot-Based Quantification of Glial Fibrillary Acidic Protein as a Disease-Associated Biomarker in Neuromyelitis Optica Spectrum Disorder
- 2026-07Aquaporin-4's dynamic expression and biological variations within the tumor obscure the paraneoplastic phenomenon in aquaporin-4-IgG-positive neuromyelitis optica spectrum disorder
- 2026-07病例报告Neuromyelitis optica presenting with mammillary body and cervicomedullary lesions after severe hyponatremia mimicking osmotic demyelination syndrome
- 2026-07High early mortality and contributing factors in seropositive neuromyelitis optica spectrum disorder at a tertiary hospital in Zambia
- 2026-07综述Impact of smoking on clinical outcomes in NMOSD and MOGAD: A meta-analysis
- 2026-07Focal astrocyte loss reveals nuclear translocation during lesion repopulation
- 2026-07Patient-reported outcomes in Chinese patients with AQP4-IgG-positive neuromyelitis optica spectrum disorder on inebilizumab therapy: a cross-sectional study
- 2026-07French guidelines for the diagnosis and management of neuromyelitis optica spectrum disorder
- 2026-07Acute transverse myelitis excluding MS, NMOSD and MOGAD: A 15-year real-world cohort from Iran
- 2026-07荟萃分析系统综述Double-Negative Neuromyelitis Optica Spectrum Disorder: A Systematic Review and Meta-Analysis
- 2026-07综述Brain network alterations in autoimmune diseases: Current status and future prospects
- 2026-07综述Comparative Evaluation of Rituximab Versus Approved Therapies in Aquaporin-4-IgG-Positive Neuromyelitis Optica Spectrum Disorder: A Systematic Review and Network Meta-analysis
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。
- 伊奈利珠单抗注射液乙类谈判药品限抗水通道蛋白4(AQP4)抗体阳性的视神经脊髓炎谱系疾病(NMOSD)成人患者。
- 萨特利珠单抗注射液乙类谈判药品限≥12岁青少年及成人患者水通道蛋白4(AQP4)抗体阳性的视神经脊髓炎谱系疾病(NMOSD)。
境外已获批用于本病的药物 6L2
欧盟 2 项、美国 4 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。
- Enspryng欧盟2021-06-24satralizumab官方记录
- Uplizna欧盟2022-04-25inebilizumab官方记录
- Soliris美国2019-06-27eculizumab官方记录
- Uplizna美国2020-06-11inebilizumab官方记录
- Enspryng美国2020-08-14satralizumab-mwge官方记录
- Ultomiris美国2024-03-22ravulizumab-cwvz官方记录
已获孤儿药资格、尚未获批的在研药物（4 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。
- cladribine美国2015-03-19Treatment of neuromyelitis optica and neuromyelitis optica spectrum disorder官方记录
- ublituximab美国2016-08-25Treatment of neuromyelitis optica and neuromyelitis optica spectrum disorder官方记录
- Edralbrutinib美国2023-04-24Treatment of neuromyelitis optica spectrum disorder官方记录
- rituximab美国2026-02-02treatment of neuromyelitis optica spectrum disorder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 74L2
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 34
- 招募中NCT04106830Clinical and Imaging Cohort of Neuroinflammation Diseases in China (CLUE)中国研究中心 1 个：Beijing
- 招募中NCT04561557Safety and Efficacy of CT103A Cells for Relapsed/Refractory Antibody-associated Inflammatory Diseases of the Nervous System中国研究中心 1 个：Wuhan
- 招募中NCT05154370China National Registry of Neuro-Inflammatory Diseases中国研究中心 1 个：Beijing
- 招募中NCT05145361Clinical Study of B001 Injection in Subjects With Neuromyelitis Optic Spectrum Disorder (NMOSD)中国研究中心 4 个：Beijing、Taiyuan、Tianjin、Xi’an
- 招募中NCT05504694Ofatumumab in AQP4-IgG Seropositive NMOSD中国研究中心 1 个：Xi'an
- 招募中NCT06697535A Study to Evaluate the Efficacy and Safety of JYP0061 in Patients With Acute Neuromyelitis Spectrum Disease (NMOSD)中国研究中心 1 个：Changsha
- 仅邀请入组NCT06643858Normal Chinese Lifespan Brain Charts Initiative (NCLBCI)中国研究中心 1 个：Beijing
- 招募中NCT06541626Sun Yat-Sen Cohort of CNS Idiopathic Inflammatory Demyelinating Diseases中国研究中心 1 个：Guangzhou
- 招募中NCT05966467Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor Therapies中国研究中心 6 个：Beijing、Guangzhou、Henan、Shanghai、Sichuan、Wuhan
- 招募中NCT06249438A Study of C-CAR168 in the Treatment of Autoimmune Diseases Refractory to Standard Therapy中国研究中心 1 个：Shanghai
- 招募中NCT07653984Cohort Study on Neuroimmune Diseases in the Reproductive Age中国研究中心 1 个：Guangzhou
- 招募中NCT06413654A Clinical Study of B001 Injection in the Treatment of Neuromyelitis Optica Spectrum Disorders(NMOSD)中国研究中心 51 个：Beijing、Cangzhou、Changsha、Chengdu、Fuzhou、Guangzhou 等 33 地
- 招募中NCT06502015Biomarkers in Autoimmune Disease of Nervous System中国研究中心 1 个：Wuhan
- 招募中NCT06474520Efficacy and Safety of Calculus Bovis Sativus (CBS) for Idiopathic Inflammatory Demyelinating Disease (CBSinIIDD)中国研究中心 1 个：Wuhan
- 尚未开始招募NCT06485232Universal CAR-T Cells in Patients with Refractory Autoimmune Diseases of the Nervous System.中国研究中心 1 个：Beijing
- 招募中NCT06620809The Safety and Efficacy of NouvSoma001 in Neuromyelitis Optica Spectrum Disorders中国研究中心 1 个：Wuhan
- 尚未开始招募NCT06829524HANDLE-a Real World Study on Satralizumab in NMOSD中国研究中心 1 个：Shanghai
- 招募中NCT07022197Safety and Efficacy of BAFF-R CART for Refractory Neuroimmune Diseases中国研究中心 1 个：Tianjin
- 招募中NCT06763848Protein A immuNoaDsorption for the Treatment of Acute Episodes of Neuromyelitis Optica Spectrum Disorder中国研究中心 1 个：Guangzhou
- 招募中NCT06939166Universal Chimeric Antigen Receptor T-Cell （UCAR T-cell） Therapy Targeting CD19/B Cell Maturation Antigen （CD19/BCMA） in Patients With r/r Neurological Autoimmune Diseases中国研究中心 1 个：Tianjin
- 招募中NCT06885957Monoclonal Antibody-Based Therapies for AQP4-Positive NMOSD中国研究中心 1 个：Wuhan
- 尚未开始招募NCT07266051Prospective Cohort Study of Neuromyelitis Optica Spectrum Disorders (NMOSD)中国研究中心 5 个：Xiamen
- 招募中NCT07377305Efficacy and Safety of Transcranial Temporal Interference Stimulation (tTIS) for Neuropathic Pain in Neuromyelitis Optica Spectrum Disorder (NMOSD)中国研究中心 1 个：Xi'an
- 招募中NCT07420296Modified Zipper Therapy for AQP4-IgG Positive Neuromyelitis Optica Spectrum Disorder中国研究中心 1 个：Tianjin
- 招募中NCT07410039Therapy in the Acute Phase of NMOSD: A Multicenter Prospective Real-World Study中国研究中心 1 个：Beijing
另有 9 项未列出。
其他状态的试验（40 项）
- 状态未知NCT02021825Efficacy and Safety of Mitoxantrone in Patients With Refractory Neuromyelitis Optica and Spectrum Disorders中国研究中心 1 个：Beijing
- 状态未知NCT01364246Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Progressive Multiple Sclerosis and Neuromyelitis Optica中国研究中心 1 个：Nanjing
- 已完成NCT02249676Autologous Mesenchymal Stem Cells for the Treatment of Neuromyelitis Optica Spectrum Disorders中国研究中心 1 个：Tianjin
- 状态未知NCT02836327Multimodel Magnetic Resonance Imaging (MRI)of Multiple Sclerosis and Neuromyelitis Optica Spectrum Disorders中国研究中心 1 个：Beijing
- 已完成NCT02893111Efficacy and Safety of Bortezomib as add-on Treatment in Relapsing Neuromyelitis Optica Spectrum Disorder中国研究中心 1 个：Tianjin
- 已完成NCT03062579A Longitudinal Study of ACTEMRA® (Tocilizumab) as Monotherapy in Highly Active NMOSD中国研究中心 1 个：Tianjin
- 已完成NCT03350633Tocilizumab vs Azathioprine in Neuromyelitis Optica Spectrum Disorders中国研究中心 1 个：Tianjin
- 已终止NCT03330418A Phase III Study of TACI-antibody Fusion Protein Injection (RC18) in Subjects With Neuromyelitis Optica Spectrum Disorders中国研究中心 1 个：Beijing
- 状态未知NCT03514030Clinic Registry Study of Optic Neuromyelitis Spectrum Disease in China中国研究中心 1 个：Beijing
- 已撤回NCT03605238Treatment of Relapsed and/or Refractory AQP4-IgG Seropositive NMOSD by Tandem CAR T Cells Targeting CD19 and CD20中国研究中心 1 个：Beijing
- 状态未知NCT04101058Treatment Response Among Chinese Neuromyelitis Optica Spectrum Disorders中国研究中心 1 个：Guangzhou
- 已完成NCT05896605Monitoring of Azathioprine Metabolite Concentrations and Cytokine Levels in Neuromyelitis Optica Spectrum Disorder中国研究中心 1 个：Nanning
- 已完成NCT04227470A Study of HBM9161 in NMOSD Patients中国研究中心 1 个：Guangzhou
- 已完成NCT04388072An Diagnostic Cohort on AQP4-Immunoglobulin G Detection Kit中国研究中心 2 个：Fuzhou、Guangzhou
- 已完成NCT04146285A Phase I Clinical Trial of BAT4406F Injection in Patients With Neuromyelitis Optica Spectrum Disorders中国研究中心 1 个：Shanghai
- 状态未知NCT04601142Association Between the Effect of Glucocorticoid Pulse Therapy on Neuromyelitis Optica (NMO) and Gene Polymorphism中国研究中心 1 个：Beijing
- 状态未知NCT06443333National, Multicentric Registry Study on Neuroimmunological Diseases in China中国研究中心 1 个：Beijing
- 已完成NCT04670770An Open Label Study of the Effects of SHR1459 in NMOSDs Patients中国研究中心 8 个：Changsha、Chengdu、Lanzhou、Rizhao、Shanghai、Taiyuan 等 8 地
- 已完成NCT05154734Efficacy and Safety of Belimumab in Neuromyelitis Optica Spectrum Disorders中国研究中心 1 个：Tianjin
- 已完成NCT05871658Efficacy of Electroacupuncture in NMOSD Patients With Pain: Study Protocol中国研究中心 1 个：Guangzhou
- 状态未知NCT05284175A Study of Orelabrutinib in Patients With AQP4-IgG Positive Neuromyelitis Optica Spectrum Disorder中国研究中心 1 个：Beijing
- 已终止NCT05356858An Open Label Study of the Effects and Safety of Zanubrutinib in NMOSDs Adult Patients中国研究中心 1 个：Beijing
- 已完成NCT05432713A Study of LP-168 in Healthy Volunteers中国研究中心 1 个：Hangzhou
- 状态未知NCT05414890Sensitivity and Specificity of TSA-CBA for Autoantibodies Against Neural Antigen Determination中国研究中心 1 个：Beijing
- 进行中·不再招募NCT05314010A Study of MIL62 in Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD)中国研究中心 1 个：Beijing
- 状态未知NCT05414487Effects of Ofatumumab Treatment on Immune Cells and Meningeal Lymphatic Drainage in Patients With Demyelinating Diseases中国研究中心 1 个：Tianjin
- 进行中·不再招募NCT05403138Safety and Efficacy of Daratumumab in Patients With Anti-Aquaporin 4 Antibody Positive Neuromyelitis Optica Spectrum Disorders中国研究中心 1 个：Tianjin
- 已完成NCT05551598Efficacy and Safety of Mitoxantrone Hydrochloride Liposome Injection in the Treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD)中国研究中心 1 个：Beijing
- 进行中·不再招募NCT05792462Efficacy and Safety of Baricitinib in Neuromyelitis Optica Spectrum Disorders中国研究中心 1 个：Tianjin
- 已完成NCT05828212Evaluate the Safety and Efficacy of CAR-T Cells in the Treatment of R/R Neuromyelitis Optica中国研究中心 1 个：Hangzhou
- 进行中·不再招募NCT06044350A Clinical Trial of BAT4406F Injection in Patients With Neuromyelitis Optica Spectrum Disorders中国研究中心 45 个：Baotou、Beijing、Changsha、Chongqing、Dalian、Fuzhou 等 31 地
- 已完成NCT06888622BLAZE-Limiting Approach in NMOSD中国研究中心 1 个：Shanghai
- 状态未知NCT06212245A Clinical Research Study of Inebilizumab in Neuromyelitis Optica Spectrum Disorders中国研究中心 1 个：Beijing
- 进行中·不再招募NCT06180278Long-term, Open-label, Safety Study of Inebilizumab in Neuromyelitis Optica Spectrum Disorder (NMOSD)中国研究中心 1 个：Hong Kong
- 已撤回NCT05909943Efficacy and Safety of Ruxolitinib in Neuromyelitis Optica Spectrum Disorders中国研究中心 1 个：Tianjin
- 状态未知NCT05891379Inebilizumab in Acute Neuromyelitis Optica Spectrum Disorders中国研究中心 1 个：Beijing
- 已撤回NCT06548620A Study of RD06-04 in Patients With Active Autoimmune Diseases中国研究中心 1 个：Zhengzhou
- 进行中·不再招募NCT06724809Efficacy, Safety, PK, PD, and ADA of Eculizumab in Chinese Adults With NMOSD中国研究中心 7 个：Beijing、Dongguan、Jinan、Shanghai、Taiyuan、Wenzhou 等 7 地
- 已撤回NCT06869278A Study of LCAR-AIO CAR-T Cells for Treating Relapsed/Refractory Neurological Autoimmune Diseases中国研究中心 3 个：Fuzhou、Suzhou、Wuhan
- 进行中·不再招募NCT07184840Eculizumab For the Acute Attack of Neuromyelitis Optica Spectrum Disorder中国研究中心 1 个：Tianjin
中国境外的在招试验 71L2
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14法国6德国4意大利4英国3希腊1澳大利亚1韩国1以色列1捷克1阿根廷1巴西1加拿大1荷兰1另有 8 个国家/地区
CT.gov 报告命中 71 项，此处取回并展示最近的 15 项。
- 尚未开始招募NCT07595965Evaluate the Safety and Tolerability of CE211NS21 in Patients With AQP4-IgG-positive NMOSD Relapse
- 尚未开始招募NCT07573332A Safety and Tolerability Study of CC- 97540 (BMS-086353) in Anti-Aquaporin 4 Antibody Positive Neuromyelitis Optica Patients美国
- 尚未开始招募NCT07704294Early Prediction of Outcomes Following Optic Neuritis: Development and Acceptability of a Prognostic Tool英国
- 尚未开始招募NCT07738952Safety and Efficacy Study of Sivelestat in Neuromyelitis Optica Spectrum Disorder
- 尚未开始招募NCT07010302Rituximab Versus Ravulizumab, Inebilizumab, Satralizumab, and Eculizumab in NMOSD德国、美国
- 尚未开始招募NCT07685678Riptylimab for the Treatment of Optic Neuritis and Myelitis Spectrum Diseases
- 招募中NCT05909761Observational Safety Study in Women With Neuromyelitis Optica Spectrum Disorder (NMOSD) Exposed to UPLIZNA® During Pregnancy美国
- 尚未开始招募NCT07526298Anticipating Irreversible Disability in Neuromyelitis Optica Spectrum Disorder: a Study to Assess Disease Activity in Apparently Stable Patients意大利
- 尚未开始招募NCT07341828A Study of C-CAR168 in the Treatment of Central Nervous System Autoimmune Diseases Refractory to Standard Therapy
- 尚未开始招募NCT07273292Autonomic Dysfunction in Neuromyelitis Optica Spectrum Disorder and Multiple Sclerosis : A Comparative Study
- 尚未开始招募NCT07304960Multiple Sclerosis Versus Neuromyelitis Optica Spectrum Disorder
- 尚未开始招募NCT06561009Safety and Efficacy of BAFFR CART for Relapsed/ Refractory Neuromyelitis Optica Spectrum Disorder
- 尚未开始招募NCT07182409Efficacy and Safety of Monoclonal Antibody in Acute Phase of Neuromyelitis Optica Spectrum Disorder
- 尚未开始招募NCT06903130Clinical Study to Evaluate the Effects of the Complement C5 Inhibitor Ravulizumab on Serum Neurofilament Light Chain (sNfL) and Glial Fibrillary Acidic Protein (sGFAP) Levels in Patients With Aquaporin-4-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD)希腊
- 尚未开始招募NCT07132398Slow vs. Rapid Glucocorticoids Tapering With Inebilizumab in NMOSD
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）