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Abstract：
Background: The therapeutic management of immune-mediated thrombotic thrombocytopenic purpura (iTTP) has recently benefited from the introduction of caplacizumab, an agent directed at the inhibition of platelet aggregation. This real-world analysis investigated the epidemiology and the demographic and clinical characteristics of iTTP patients in Italy before and after caplacizumab introduction in 2020. Methods: Hospitalized adults with iTTP were included using the administrative databases of healthcare entities covering 17 million residents. Epidemiological estimates of iTTP considered the 3-year period before and after caplacizumab introduction. After stratification by treatment with or without caplacizumab, iTTP patients were characterized for their baseline features. Results: The annual incidence before and after 2020 was estimated in the range of 4.3-5.8 cases/million and 3.6-4.6 cases/million, respectively. From 2018 to 2022, 393 patients with iTTP were included, and 42 of them were treated with caplacizumab. Caplacizumab-treated patients showed better clinical outcomes, with tendentially shorter hospital stays and lower mortality rates (no treated patients died at either 1 month or 3 months after caplacizumab treatment initiation, compared to 10.5% and 11.1% mortality rates at 1 and 3 months, respectively, of the untreated ones). Conclusions: These findings may suggest that caplacizumab advent provided clinical and survival benefits for patients with iTTP.
摘要：
背景：免疫介导的血栓性血小板减少性紫癜（iTTP）的治疗管理最近受益于caplacizumab的引入，一种抑制血小板聚集的药物。这项现实世界的分析调查了2020年caplacizumab引入前后意大利iTTP患者的流行病学以及人口统计学和临床特征。方法：使用覆盖1700万居民的医疗保健实体的管理数据库纳入患有iTTP的住院成年人。iTTP的流行病学估计考虑了caplacizumab引入前后的3年时间。在通过使用或不使用卡普拉珠单抗治疗进行分层后，对iTTP患者的基线特征进行表征。结果：2020年前后的年发病率估计在4.3-5.8例/百万和3.6-4.6例/百万之间，分别。从2018年到2022年，纳入了393例iTTP患者，其中42例接受了卡普拉斯单抗治疗.卡普拉斯单抗治疗的患者表现出更好的临床结果，住院时间缩短,死亡率降低(卡普拉斯单抗治疗开始后1个月或3个月无治疗患者死亡,与1个月和3个月的10.5%和11.1%的死亡率相比，分别,未经处理的)。结论：这些发现可能表明caplacizumab的出现为iTTP患者提供了临床和生存益处。
