Hemotherapy, Banc de Sang i Teixits, Càtedra UAB de Medicina Transfusional i Teràpia Cel·lular i Tissular, Hospital Universitary of Bellvitge, L'Hospitalet de Llobregat, Catalonia, Spain.
Internal Medicine, Hospital Universitary of Bellvitge, L'Hospitalet de Llobregat, Catalonia, Spain.
Blood Transfus. 2024 Mar;22(2):176-184. doi: 10.2450/BloodTransfus.522. Epub 2023 Sep 4.
Thrombotic thrombocytopenic purpura (TTP) is a low prevalence disease characterized by severe deficiency of the enzyme ADAMTS13, leading to the development of thrombotic microangiopathy (TMA) and often resulting in severe organ disfunction. TTP is an extremely serious condition and, therefore, timely and appropriate treatment is critical to prevent life-threatening complications.Over the past 25 years, significant advances in the understanding of the pathophysiology of immune TTP have led to the development of readily available techniques for measuring ADAMTS13 levels, as well as new drugs that are particularly effective in the acute phase and in preventing relapses. These developments have improved the course of the disease.Given the complexity of the disease and its various clinical and laboratory manifestations, early diagnosis and treatment can be challenging.To address this challenge, a group of experienced professionals from the Catalan TTP group have developed this consensus statement to standardize terminology, diagnosis, treatment and follow up for immune TTP, based on currently available scientific evidence in the field. This guidance document aims to provide healthcare professionals with a comprehensive tool to make more accurate and timely diagnosis of TTP and improve patient outcomes.
血栓性血小板减少性紫癜（TTP）是一种低发病率疾病，其特征为 ADAMTS13 酶严重缺乏，导致血栓性微血管病（TMA）的发生，并常导致严重的器官功能障碍。TTP 是一种极其严重的病症，因此，及时和适当的治疗对于预防危及生命的并发症至关重要。在过去的 25 年中，人们对免疫性 TTP 的病理生理学有了更深入的了解，这导致了易于测量 ADAMTS13 水平的技术以及新药物的发展，这些药物在急性阶段和预防复发方面特别有效。这些进展改善了疾病的进程。鉴于疾病的复杂性及其各种临床和实验室表现，早期诊断和治疗可能具有挑战性。为了应对这一挑战，一个来自加泰罗尼亚 TTP 小组的经验丰富的专业人员小组根据该领域目前可用的科学证据，制定了本共识声明，以规范免疫性 TTP 的术语、诊断、治疗和随访。本指导文件旨在为医疗保健专业人员提供一种全面的工具，以便更准确和及时地诊断 TTP 并改善患者的预后。
