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

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Item Type:Article
Title:Real-world data confirm the effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura
Creators Name:Völker, L.A. and Kaufeld, J. and Miesbach, W. and Brähler, S. and Reinhardt, M. and Kühne, L. and Mühlfeld, A. and Schreiber, A. and Gaedeke, J. and Tölle, M. and Jabs, W.J. and Özcan, F. and Markau, S. and Girndt, M.. and Bauer, F. and Westhoff, T.H. and Felten, H. and Hausberg, M. and Brand, M. and Gerth, J. and Bieringer, M. and Bommer, M. and Zschiedrich, S. and Schneider, J. and Elitok, S. and Gawlik, A. and Gäckler, A. and Kribben, A. and Schwenger, V. and Schoenermarck, U. and Roeder, M. and Radermacher, J. and Bramstedt, J. and Morgner, A. and Herbst, R. and Harth, A. and Potthoff, S.A. and von Auer, C. and Wendt, R. and Christ, H. and Brinkkoetter, P.T. and Menne, J.
Abstract: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.
Keywords:Caplacizumab, Thrombotic Thrombocytopenic Purpura, Platelet Count Measurement
Source:Blood Advances
ISSN:2473-9529
Publisher:American Society of Hematology
Volume:4
Number:13
Page Range:3085-3092
Date:14 July 2020
Additional Information:Copyright © 2020 by The American Society of Hematology
Official Publication:https://doi.org/10.1182/bloodadvances.2020001973
PubMed:View item in PubMed

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