Real-world data confirm the effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura
Real-world data confirm the effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura
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DOI：
10.1182/bloodadvances.2020001973
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发表时间：
2020-07-14
期刊：
影响因子：
7.5
通讯作者：
Menne, Jan
中科院分区：
文献类型：
--
作者：
Voelker, Linus A.;Kaufeld, Jessica;Menne, Jan
获得性血栓性血小板减少性紫癜（aTTP）是一种罕见但危及生命的疾病。2018年，纳米抗体caplacizumab被批准用于治疗aTTP急性发作的成人，与血浆置换（PEX）和免疫抑制联合使用，在停止每日PEX后至少30天。我们对来自德国29家医疗中心的60例患者在急性疾病管理期间使用caplacizumab的情况进行了回顾性、观察性分析。Caplacizumab使血小板计数迅速恢复正常（中位数：3天;平均值：3.78天）。1例患者在延迟治疗开始后死于aTTP相关并发症。在2例有初始疾病表现的患者和另外4例在初始治疗后有急性发作或复发实验室体征的患者中，可以建立无PEX治疗方案，总体结局良好。Caplacizumab治疗aTTP有效，与时间和辅助治疗方式无关。基于该真实世界经验和已发表文献，我们建议对所有aTTP急性发作患者立即给予caplacizumab。关于使用PEX的治疗决定应基于临床表现的严重程度和已知的风险因素。对于某些患者来说，PEX可能是可有可无的。
Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare but life-threatening condition. In 2018, the nanobody caplacizumab was approved for the treatment of adults experiencing an acute episode of aTTP, in conjunction with plasma exchange (PEX) and immunosuppression for a minimum of 30 days after stopping daily PEX. We performed a retrospective, observational analysis on the use of caplacizumab in 60 patients from 29 medical centers in Germany during acute disease management. Caplacizumab led to a rapid normalization of the platelet count (median, 3 days; mean 3.78 days). One patient died after late treatment initiation due to aTTP-associated complications. In 2 patients with initial disease presentation and in 4 additional patients with laboratory signs of an exacerbation or relapse after the initial therapy, PEX-free treatment regimens could be established with overall favorable outcome. Caplacizumab is efficacious in the treatment of aTTP independent of timing and ancillary treatment modalities. Based on this real-world experience and published literature, we propose to administer caplacizumab immediately to all patients with an acute episode of aTTP. Treatment decisions regarding the use of PEX should be based on the severity of the clinical presentation and known risk factors. PEX might be dispensable in some patients.