TY - JOUR
T1 - Caplacizumab prevents refractoriness and mortality in acquired thrombotic thrombocytopenic purpura
T2 - Integrated analysis
AU - Peyvandi, Flora
AU - Cataland, Spero
AU - Scully, Marie
AU - Coppo, Paul
AU - Knoebl, Paul
AU - Kremer Hovinga, Johanna A.
AU - Metjian, Ara
AU - de la Rubia, Javier
AU - Pavenski, Katerina
AU - Mi Edou, Jessica Minkue
AU - de Winter, Hilde
AU - Callewaert, Filip
N1 - Funding Information:
Medical writing and editorial support were provided by Namiko Abe (Fishawack Communications, part of Fishawack Health, Conshohocken, PA) and were funded by Sanofi. This work was supported by Ablynx, a Sanofi company, as were the HERCULES and TITAN studies.
Publisher Copyright:
© 2021 by The American Society of Hematology
PY - 2021/4/27
Y1 - 2021/4/27
N2 - 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.
AB - 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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U2 - 10.1182/BLOODADVANCES.2020001834
DO - 10.1182/BLOODADVANCES.2020001834
M3 - Article
C2 - 33881463
AN - SCOPUS:85106281530
VL - 5
SP - 2137
EP - 2141
JO - Blood advances
JF - Blood advances
SN - 2473-9529
IS - 8
ER -