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Caplacizumab prevents refractoriness and mortality in acquired thrombotic thrombocytopenic purpura: integrated analysis

Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy. Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy. Division of Hematology, Department of Internal Medicine, The Ohio State University, Columbus, OH. Department of Haematology, University College London Hospital National Health Service Trust, London, United Kingdom. Department of Hematology, Reference Center for Thrombotic Microangiopathies, Saint-Antoine University Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France. Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna, Austria. Department of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland. Department of Medicine, Duke University School of Medicine, Durham, NC. Hematology Department, Internal Medicine, School of Medicine and Dentistry, Catholic University of Valencia and Hospital Doctor Peset, Valencia, Spain. Departments of Laboratory Medicine and Medicine, St. Michael's Hospital, Toronto, Canada. Departments of Laboratory Medicine and Pathobiology and Medicine, University of Toronto, Toronto, Canada. Clinical Development, Ablynx, a Sanofi company, Ghent, Belgium; and. Medical Affairs, Sanofi, Diegem, Belgium.

Blood advances. 2021;5(8):2137-2141
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Abstract
The efficacy and safety of caplacizumab in individuals with acquired thrombotic thrombocytopenic purpura (aTTP) have been established in the phase 2 TITAN and phase 3 HERCULES trials. Integrated analysis of data from both trials was conducted to increase statistical power for assessing treatment differences in efficacy and safety outcomes. Caplacizumab was associated with a significant reduction in the number of deaths (0 vs 4; P < .05) and a significantly lower incidence of refractory TTP (0 vs 8; P < .05) vs placebo during the treatment period. Consistent with the individual trials, treatment with caplacizumab resulted in a faster time to platelet count response (hazard ratio, 1.65; P < .001), a 72.6% reduction in the proportion of patients with the composite end point of TTP-related death, TTP exacerbation, or occurrence of at least 1 treatment-emergent major thromboembolic event during the treatment period (13.0% vs 47.3%; P < .001), and a 33.3% reduction in the median number of therapeutic plasma exchange days (5.0 vs 7.5 days) vs placebo. No new safety signals were identified; mild mucocutaneous bleeding was the main safety finding. This integrated analysis provided new evidence that caplacizumab prevents mortality and refractory disease in acquired TTP and strengthened individual trial findings, with a confirmed favorable safety and tolerability profile. These trials were registered at www.clinicaltrials.gov as #NCT01151423 and #NCT02553317.
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Language : eng
Credits : Bibliographic data from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine