Abou-Ismail Mouhamed Yazan, Kapoor Sargam, Citla Sridhar Divyaswathi, Nayak Lalitha, Ahuja Sanjay
Division of Hematology and Hematologic Malignancies Department of Internal Medicine University of Utah Health Sciences Center Salt Lake City Utah USA.
Department of Hematology & Oncology Alaska Native Medical Center Anchorage Alaska USA.
Res Pract Thromb Haemost. 2022 Apr 22;6(3):e12708. doi: 10.1002/rth2.12708. eCollection 2022 Mar.
The thrombotic microangiopathies (TMAs) are a heterogenous group of disorders with distinct pathophysiologies that cause occlusive microvascular or macrovascular thrombosis, and are characterized by microangiopathic hemolytic anemia, thrombocytopenia, and/or end-organ ischemia. TMAs are associated with significant morbidity and mortality, and data on the management of certain TMAs are often lacking. The nomenclature, classification, and management of various TMAs is constantly evolving as we learn more about these rare syndromes. Thorough clinical and laboratory evaluation is essential to distinguish various TMAs and arrive at an accurate diagnosis, which is key for appropriate management. In this illustrated review, we focus on thrombotic thrombocytopenic purpura (TTP), Shiga toxin-associated hemolytic uremic syndrome, complement-mediated hemolytic uremic syndrome, hematopoietic cell transplant-associated TMA, and drug-induced TMA, and describe their incidence, pathophysiology, diagnosis, and management. We also highlight emerging complement-directed therapies under investigation for the management of complement-mediated TMAs.
血栓性微血管病（TMA）是一组病理生理学各异的异质性疾病，可导致微血管或大血管闭塞性血栓形成，其特征为微血管病性溶血性贫血、血小板减少和/或终末器官缺血。TMA与显著的发病率和死亡率相关，且关于某些TMA治疗的数据往往缺乏。随着我们对这些罕见综合征的了解不断深入，各种TMA的命名、分类和治疗也在不断演变。全面的临床和实验室评估对于区分各种TMA并做出准确诊断至关重要，这是恰当治疗的关键。在本图文综述中，我们重点关注血栓性血小板减少性紫癜（TTP）、志贺毒素相关溶血性尿毒症综合征、补体介导的溶血性尿毒症综合征、造血细胞移植相关TMA和药物性TMA，并描述它们的发病率、病理生理学、诊断和治疗。我们还强调了正在研究的用于治疗补体介导的TMA的新型补体靶向疗法。
