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冷凝集素病

Cold agglutinin disease

定义 英文原文(暂无中文)

Cold agglutinin disease is a type of autoimmune hemolytic anemia defined by the presence of cold autoantibodies (autoantibodies which are active at temperatures below 30°C).

别名

慢性冷凝集素病

基本事实

遗传方式
多基因/多因素
发病年龄
成年期
患病率
1-9 / 1 000 000(Europe)

临床表型 14

极常见 99–80%6

  • 关节疼痛 HP:0002829
  • 自身免疫 HP:0002960
  • 疲乏 HP:0012378
  • 溶血性贫血 HP:0001878
  • 肌无力 HP:0001324
  • 苍白圈 HP:0000980

偶见 29–5%8

  • 尿液颜色异常 HP:0012086
  • 背部疼痛 HP:0003418
  • 腹泻 HP:0002014
  • 头痛 HP:0002315
  • 肝脏肿大 HP:0002240
  • 淋巴结肿大 HP:0002716
  • 恶心和呕吐 HP:0002017
  • 脾肿大 HP:0001744

近两年的全球研究 329L2

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。

  • 2026-10
    Evolution of Therapeutic Plasma Exchange Use and Outcomes in Children Across American Society for Apheresis Categories: A Single-Center Cohort Study
    Journal of clinical apheresis · DOI · Europe PMC
  • 2026-09病例报告开放获取
    Case Report: Severe mixed autoimmune hemolytic anemia associated with acute cytomegalovirus infection in an immunocompetent adult
    Frontiers in immunology · DOI · Europe PMC
  • 2026-09病例报告开放获取
    Complement inhibition by sutimlimab in cold agglutinin disease promotes red blood cell agglutination and may lead to circulatory complications, eryptosis and hemolysis
    Frontiers in immunology · DOI · Europe PMC
  • 2026-09综述开放获取
    Cold agglutinin disease: pathophysiology, diagnosis, and the evolving therapeutic landscape
    Blood research · DOI · Europe PMC
  • 2026-09开放获取
    The impact of chemoimmunotherapy on primary cold agglutinin disease and Waldenström macroglobulinemia-associated cold agglutinin syndrome
    Frontiers in medicine · DOI · Europe PMC
  • 2026-09
    Guidelines on the Use of Therapeutic Apheresis in Clinical Practice-Evidence-Based Approach From the Writing Committee of the American Society for Apheresis: The Tenth Special Issue
    Journal of clinical apheresis · DOI · Europe PMC
  • 2026-09综述开放获取
    Complement Activation by Post-Translationally Modified Proteins: Links to Chronic Inflammation and Autoimmunity
    Immunological reviews · DOI · Europe PMC
  • 2026-09综述开放获取
    Anti-CD38 monoclonal antibodies in multiple myeloma and beyond: immunotherapeutic mechanisms, evidence maturity, and clinical positioning
    Frontiers in immunology · DOI · Europe PMC
  • 2026-08开放获取
    Therapeutic Plasma Exchange Using Membrane Plasma Separation Across a Broad Spectrum of Indications: An Eight-Year Single-Center Retrospective Cohort Study
    International journal of general medicine · DOI · Europe PMC
  • 2026-08开放获取
    Complement-Dominant Relapse of Mixed Autoimmune Hemolytic Anemia After Rituximab Response
    EJHaem · DOI · Europe PMC
  • 2026-08
    Interpretive Challenges in Electrophoretic and Mass Spectrometric Methods for Monoclonal Gammopathy Detection in Patients with Cold Agglutinin Disease
    Clinical chemistry · DOI · Europe PMC
  • 2026-08综述开放获取
    Targeting CD38 Across Autoimmune and Plasma Cell-Driven Immune-Mediated Diseases: Rationale, Clinical Evidence, and Emerging Therapeutic Landscape
    BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene th · DOI · Europe PMC
  • 2026-08病例报告开放获取
    Combination therapy of sutimlimab and rituximab can lead to stable remission in autoimmune hemolytic anemia: a case series
    Annals of hematology · DOI · Europe PMC
  • 2026-08综述开放获取
    Innate immunity: current understandings and future perspectives
    Signal transduction and targeted therapy · DOI · Europe PMC
  • 2026-08开放获取
    Effectiveness of the DigniCap® Scalp Cooling System for the Prevention of Chemotherapy-Induced Alopecia: An Observational Study in a Uruguayan Cohort
    Cureus · DOI · Europe PMC
  • 2026-08病例报告开放获取
    Probable Cold Agglutinin Disease Presenting With Profound Anemia and Cardiopulmonary Decompensation in a 93-Year-Old Woman: A Case Report
    Cureus · DOI · Europe PMC
  • 2026-08病例报告开放获取
    A blended phenotype of primary immunodeficiency and Temtamy syndrome: Dual homozygosity for <i>STK4</i> and <i>C12orf57</i> gene variants in a Tunisian infant
    Molecular genetics and metabolism reports · DOI · Europe PMC
  • 2026-08病例报告开放获取
    DAT-negative autoimmune hemolytic anemia following acute hepatitis a in a pediatric patient with elevated IgA: a rare case report and literature review
    Annals of medicine and surgery (2012) · DOI · Europe PMC
  • 2026-08综述开放获取
    Lack of Differences in the Pharmacokinetics of Therapeutic Monoclonal Antibodies Between Japanese and Non-Japanese Individuals
    Journal of clinical pharmacology · DOI · Europe PMC
  • 2026-08综述病例报告开放获取
    Temperature Safeguards and Peri-Procedural Strategies for Stem Cell Collection in Cold Agglutinin Disease: A Case Report and Literature Review
    Journal of clinical apheresis · DOI · Europe PMC

境外已获批用于本病的药物 1L2

欧盟 1 项、美国 0 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。

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 2L2

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 2

  • 招募中NCT07019038
    National Longitudinal Cohort of Hematological Diseases- Autoimmune Hemolytic Anemia
    观察性 · 2001/01/01Institute of Hematology & Blood Diseases Hospital, China
    中国研究中心 1 个:Tianjin
  • 尚未开始招募NCT06847607
    A Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)
    不适用 · 干预性 · 2025/03/01Bing Han
    中国研究中心 1 个:Beijing

中国境外的在招试验 6L2

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。

澳大利亚1美国1英国1意大利1

CT.gov 报告命中 6 项,此处取回并展示最近的 4 项。

  • 尚未开始招募NCT07091370
    Inaticabtagene Autoleucel Injection in the Treatment of Autoimmune Hemolytic Anemia After Three or More Lines of Therapy
    I 期 · 干预性 · 2025/12/27Juventas Cell Therapy Ltd.
  • 招募中NCT07083960
    OM336 in Autoimmune Cytopenias
    I 期 · 干预性 · 2025/08/01Lakefront Biotherapeutics West LLC
    澳大利亚、美国
  • 招募中NCT05318534
    Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study in Healthy Male and Female Subjects and Safety, Tolerability, Pharmacokinetics, and Pilot Efficacy Biomarkers in Subjects With Cold Agglutinin Disease
    I 期 · 干预性 · 2022/04/08Gliknik Inc.
    英国
  • 招募中NCT05931718
    Prospective Evaluation of Diagnosis and Treatment of Patients With Autoimmune Cytopenias Including Autoimmune Hemolytic Anemia, Immune Thrombocytopenia, and Chronic Idiopathic/Autoimmune Neutropenia
    观察性 · 2019/06/01Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
    意大利

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)