华氏巨球蛋白血症

定义 英文原文（暂无中文）

A rare indolent B-cell non-Hodgkin lymphoma, characterized by the infiltration of monoclonal lymphoplasmacytic cells in the bone marrow and the production of serum immunoglobulin M (IgM) monoclonal protein.

基本事实

遗传方式

多基因/多因素

发病年龄

老年期

患病率

1-9 / 1 000 000（Europe）

相关基因 1

临床表型 45

近两年的全球研究 843L2

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。

• 2026-09开放获取
  Chemoimmunotherapy-associated CD20 (<i>MS4A1</i>) resistance mutations in Waldenström macroglobulinemia: Epitope disruption independent of expression with implications for CD20-directed cellular therapies
  HemaSphere · DOI · Europe PMC
• 2026-09病例报告
  Uveitis as a class effect of Bruton tyrosine kinase inhibitors: ibrutinib and zanubrutinib ocular toxicity
  BMJ case reports · DOI · Europe PMC
• 2026-09病例报告开放获取
  Gastrointestinal Manifestation of Light Chain Deposition Disease: Beware of the Snakeskin Duodenum
  ACG case reports journal · DOI · Europe PMC
• 2026-09综述
  Current and new approaches to the treatment landscape for adults with Waldenström macroglobulinemia
  Expert opinion on pharmacotherapy · DOI · Europe PMC
• 2026-09
  Comorbidity burden is a major independent prognostic factor in Waldenström Macroglobulinemia: A national cancer database retrospective study
  Leukemia research · DOI · Europe PMC
• 2026-09
  Real-World Treatment Patterns, Healthcare Resource Use, and Costs in Patients with Follicular Lymphoma, Waldenström Macroglobulinemia, and Marginal Zone Lymphoma: A Claims-Based Study in the USA
  Clinical drug investigation · DOI · Europe PMC
• 2026-09综述开放获取
  PU.1 as a Master Regulator of Immune Cell Development and Function: Structural Basis, Gene Regulatory Networks, Disease Roles, and Therapeutic Targeting
  Biology · DOI · Europe PMC
• 2026-09
  Prognostic significance of monotypic plasma cell infiltration of the bone marrow in patients with Waldenström macroglobulinemia
  Haematologica · DOI · Europe PMC
• 2026-09开放获取
  Clonotypic characterization defines B-cell drivers of clonal expansion and intratumor heterogeneity in IgM monoclonal gammopathies
  HemaSphere · DOI · Europe PMC
• 2026-09
  Latin American Consensus for the Diagnosis, Staging, and Treatment of Peripheral T-Cell Lymphoma Not Otherwise Specified
  JCO global oncology · DOI · Europe PMC
• 2026-09
  Cost-effectiveness of zanubrutinib versus ibrutinib as first-line treatment for patients with Waldenström macroglobulinemia in China
  Expert review of pharmacoeconomics & outcomes research · 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
  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病例报告开放获取
  Pseudo-negative serum protein electrophoresis due to IgM aggregation in a patient with Waldenström macroglobulinemia: A case report
  Medicine · DOI · Europe PMC
• 2026-09开放获取
  DNA Replication Stress-Induced Transcriptome of Human Burkitt's Lymphoma Identifies Reciprocal Regulation Between MBD1 and BCL6 During Germinal Center-Derived B-Lymphomagenesis
  Hematological oncology · DOI · Europe PMC
• 2026-09开放获取
  Anti-CD19 CAR-T in transformed indolent lymphoma vs de novo large B-cell lymphoma: a meta-analysis of 5006 patients
  Blood advances · DOI · Europe PMC
• 2026-09开放获取
  KIT mutation and AHN-associated oncogenic profiles in systemic mastocytosis with an associated hematological neoplasm
  Blood advances · DOI · Europe PMC
• 2026-09综述开放获取
  Analysis of cell-free DNA in lymphomas: from sample collection to genotyping and minimal residual disease monitoring
  Blood advances · DOI · Europe PMC
• 2026-09
  Genomic Features Do Not Account for Differences in Multiple Myeloma Risk by Ancestry
  Blood cancer discovery · DOI · Europe PMC
• 2026-09
  Unmasking Hidden Monoclonal Proteins: Leveraging the Lipemia Index
  The journal of applied laboratory medicine · DOI · Europe PMC

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。

• 伊布替尼胶囊乙类西药
  限：1.既往至少接受过一种治疗的套细胞淋巴瘤(MCL)患者的治疗；2.慢性淋巴细胞白血病/小淋巴细胞淋巴瘤(CLL/SLL)患者的治疗；3.华氏巨球蛋白血症患者的治疗，按说明书用药。
• 泽布替尼胶囊乙类谈判药品
  限：1.既往至少接受过一种治疗的成人套细胞淋巴瘤(MCL)患者；2.成人慢性淋巴细胞白血病(CLL)/小淋巴细胞淋巴瘤(SLL)患者；3.成人华氏巨球蛋白血症(WM)患者；4.联合奥妥珠单抗用于既往接受过至少二线系统性治疗的复发或难治性滤泡性淋巴瘤(FL)成人患者。

境外已获批用于本病的药物 0L2

欧盟与美国均未检索到已获批用于本病的药物。

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。

中国境外的在招试验 1L2

共 1 项。

• 招募中NCT05724121
  Observational Study of Cardiac Arrhythmias During Treatment With BTK Inhibitors or Venetoclax
  观察性 · 2023/03/01National Heart, Lung, and Blood Institute (NHLBI)
  美国

外部标识与链接