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

Flora Peyvandi, Spero Cataland, Marie Scully, Paul Coppo, Paul Knoebl, Johanna A. Kremer Hovinga, Ara Metjian, Javier de la Rubia, Katerina Pavenski, Jessica Minkue Mi Edou, Hilde de Winter, Filip Callewaert

Research output: Contribution to journalArticlepeer-review


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 as #NCT01151423 and #NCT02553317.

Original languageEnglish
Pages (from-to)2137-2141
Number of pages5
JournalBlood advances
Issue number8
Publication statusPublished - Apr 27 2021

ASJC Scopus subject areas

  • Hematology


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