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

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Item Type:	Article
Title:	Impact of first-line use of caplacizumab on treatment outcomes in immune thrombotic thrombocytopenic purpura
Creators Name:	Völker, L.A., Kaufeld, J., Balduin, G., Merkel, L., Kühne, L., Eichenauer, D.A., Osterholt, T., Hägele, H., Kann, M., Grundmann, F., Kolbrink, B., Schulte, K., Gäckler, A., Kribben, A., Boss, K., Potthoff, S.A., Rump, L.C., Schmidt, T., Mühlfeld, A.S., Schulmann, K., Hermann, M., Gaedeke, J., Sauerland, K., Bramstedt, J., Hinkel, U.P., Miesbach, W., Bauer, F., Westhoff, T.H., Bruck, H., Buxhofer-Ausch, V., Müller, T.J., Wendt, R., Harth, A., Schreiber, A., Seelow, E., Tölle, M., Gohlisch, C., Bieringer, M., Geuther, G., Jabs, W.J., Fischereder, M., von Bergwelt-Baildon, A., Schönermarck, U., Knoebl, P., Menne, J. and Brinkkoetter, P.T.
Abstract:	BACKGROUND: 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. OBJECTIVES: 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. METHODS: 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). RESULTS: 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%. CONCLUSION: 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.clinicaltrials.gov as #NCT04985318.
Keywords:	Purpura, Thrombotic Thrombocytopenic, Thrombotic Microangiopathies, Observational Study, Von Willebrand Factor, ADAMTS13 Protein
Source:	Journal of Thrombosis and Haemostasis
ISSN:	1538-7933
Publisher:	Elsevier
Volume:	21
Number:	3
Page Range:	559-572
Date:	March 2023
Official Publication:	https://doi.org/10.1016/j.jtha.2022.11.010
PubMed:	View item in PubMed

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