Portal de Periódicos da CAPES

Impact of first-line use of caplacizumab on treatment outcomes in immune thrombotic thrombocytopenic purpura

2023; Elsevier BV; Volume: 21; Issue: 3 Linguagem: Inglês

10.1016/j.jtha.2022.11.010

1538-7933

Linus A. Völker, Jessica Kaufeld, Gesa Balduin, Lena Merkel, Lucas Kühne, Dennis A. Eichenauer, Thomas Osterholt, Holger Hägele, Martin Kann, Franziska Grundmann, Benedikt Kolbrink, Kevin L. Schulte, Anja Gäckler, Andreas Kribben, Kristina Boss, Sebastian A. Potthoff, Lars Christian Rump, Tilman Schmidt, Anja Mühlfeld, Karsten Schulmann, Matthias Hermann, Jens Gaedeke, Kristin Sauerland, Jörn Bramstedt, Ulrich P. Hinkel, Wolfgang Miesbach, Frederic Bauer, Timm H. Westhoff, Heike Bruck, Veronika Buxhofer‐Ausch, Tobias Müller, Ralph Wendt, Ana Harth, Adrian Schreiber, Evelyn Seelow, Markus Tölle, Christopher Gohlisch, Markus Bieringer, Gesa Geuther, Wolfram J. Jabs, Michael Fischereder, Anke von Bergwelt‐Baildon, Ulf Schönermarck, Paul Knoebl, Jan Menne, Paul T. Brinkkoetter, Fedai Özcan, Silke Markau, Matthias Girndt, H Felten, Martin Hausberg, Marcus Brand, Jens Gerth, Martin Bommer, Stefan Zschiedrich, Johanna Schneider, Saban Elitok, Alexander Gawlik, Vedat Schwenger, Maximilian von Roeder, Jörg Radermacher, Anke Morgner, Regina Herbst, Charis von Auer,

Renal Diseases and Glomerulopathies

The von Willebrand factor-directed nanobody caplacizumab has greatly changed the treatment of immune thrombotic thrombocytopenic purpura (iTTP) in recent years. Data from randomized controlled trials established efficacy and safety.This study aims to address open questions regarding patient selection, tailoring of therapy duration, obstacles in prescribing caplacizumab in iTTP, effect on adjunct treatment, and outcomes in the real-world setting.We report retrospective, observational cohorts of 113 iTTP episodes treated with caplacizumab and 119 historical control episodes treated without caplacizumab. We aggregated data from the caplacizumab phase II/III trials and real-world data from France, the United Kingdom, Germany, and Austria (846 episodes, 396 treated with caplacizumab, and 450 historical controls).Caplacizumab was efficacious in iTTP, independent of the timing of therapy initiation, but curtailed the time of active iTTP only when used in the first-line therapy within 72 hours after diagnosis and until at least partial ADAMTS13-activity remission. Aggregated data from multiple study populations showed that caplacizumab use resulted in significant absolute risk reduction of 2.87% for iTTP-related mortality (number needed to treat 35) and a relative risk reduction of 59%.Caplacizumab should be used in first line and until ADAMTS13-remission, lowers iTTP-related mortality and refractoriness, and decreases the number of daily plasma exchange and hospital stay. This trial is registered at www.gov as #NCT04985318.