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Real-world data confirm the effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura

2020; Elsevier BV; Volume: 4; Issue: 13 Linguagem: Inglês

10.1182/bloodadvances.2020001973

2473-9537

Linus A. Völker, Jessica Kaufeld, Wolfgang Miesbach, Sebastian Brähler, Martin Reinhardt, Lucas Kühne, Anja Mühlfeld, Adrian Schreiber, Jens Gaedeke, Markus Tölle, Wolfram J. Jabs, Fedai Özcan, Silke Markau, Matthias Girndt, Frederic Bauer, Timm H. Westhoff, H Felten, Martin Hausberg, Marcus Brand, Jens Gerth, Markus Bieringer, Martin Bommer, Stefan Zschiedrich, Johanna Schneider, Saban Elitok, Alexander Gawlik, Anja Gäckler, Andreas Kribben, Vedat Schwenger, Ulf Schoenermarck, Maximilian von Roeder, Jörg Radermacher, Jörn Bramstedt, Anke Morgner, Regina Herbst, Ana Harth, Sebastian A. Potthoff, Charis von Auer, Ralph Wendt, Hildegard Christ, Paul T. Brinkkoetter, Jan Menne,

Coagulation, Bradykinin, Polyphosphates, and Angioedema

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.