Department of Medicine, Michael DeGroote School of Medicine, McMaster University Canadian Blood Services, 1200 Main Street West, Hamilton, Ontario, Canada.
Br J Haematol. 2011 Jan;152(1):52-60. doi: 10.1111/j.1365-2141.2010.08412.x. Epub 2010 Nov 18.
Immune thrombocytopenia (ITP) is a common autoimmune disease characterized by low platelet counts and an increased risk of bleeding. Antibody-mediated platelet destruction has been the prevailing hypothesis to explain ITP pathogenesis, supported by the efficacy of B-cell depletion therapy; however, the recent success of thrombopoietin receptor agonists lends support to the notion that platelet production is also insufficient. Best practice for the management of chronic ITP has not yet been established because data from comparative trials are lacking. Despite renewed interest in novel drugs capable of increasing platelet counts, ultimate treatment goals for ITP patients must be kept in mind: to improve patients' health and well-being. In this article, the pathophysiology of ITP is reviewed and key remaining questions about mechanism are explored. A rational approach to the management of ITP in adults is outlined, acknowledging evidence and evidence gaps, and highlighting the need for clinically important endpoints in future clinical trials.
免疫性血小板减少症（ITP）是一种常见的自身免疫性疾病，其特征是血小板计数降低，出血风险增加。抗体介导的血小板破坏一直是解释 ITP 发病机制的主要假说，B 细胞耗竭疗法的疗效为此提供了支持；然而，血小板生成素受体激动剂的近期成功也支持了血小板生成不足的观点。由于缺乏比较试验数据，慢性 ITP 的最佳管理实践尚未确立。尽管人们对能够增加血小板计数的新型药物重新产生了兴趣，但必须牢记 ITP 患者的最终治疗目标：改善患者的健康和福祉。本文回顾了 ITP 的病理生理学，并探讨了关于发病机制的关键遗留问题。概述了成人 ITP 的合理管理方法，承认了证据和证据差距，并强调了未来临床试验中需要有临床重要终点。
