Department II of Internal Medicine and Center for Molecular Medicine Cologne, University of Cologne, Faculty of Medicine and University Hospital Cologne, Cologne, Germany.
Cologne Cluster of Excellence on Cellular Stress Responses in Ageing-Associated Diseases, Cologne, Germany.
Blood Adv. 2020 Jul 14;4(13):3085-3092. doi: 10.1182/bloodadvances.2020001973.
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.
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 被批准用于治疗成人急性发作的 aTTP，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。我们对德国 29 家医疗中心的 60 名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分析。caplacizumab 可迅速使血小板计数正常化（中位数 3 天；平均 3.78 天）。1 名患者因 aTTP 相关并发症而在晚期治疗开始后死亡。在最初疾病表现的 2 名患者和在初始治疗后出现实验室恶化或复发迹象的另外 4 名患者中，可以建立无 PEX 的治疗方案，总体结果良好。caplacizumab 独立于治疗时机和辅助治疗方式对 aTTP 的治疗有效。基于这一真实世界的经验和已发表的文献，我们建议对所有急性发作的 aTTP 患者立即使用 caplacizumab。使用 PEX 的治疗决策应基于临床表型的严重程度和已知的危险因素。在某些患者中，PEX 可能不是必需的。
