Caplacizumab prevents refractoriness and mortality in acquired thrombotic thrombocytopenic purpura: integrated analysis
| Autor: | Paul Knoebl, Javier de la Rubia, Filip Callewaert, Hilde De Winter, Johanna A. Kremer Hovinga, Spero R. Cataland, Marie Scully, Katerina Pavenski, Jessica Minkue Mi Edou, Flora Peyvandi, Ara Metjian, Paul Coppo |
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| Rok vydání: | 2021 |
| Předmět: |
medicine.medical_specialty
Exacerbation Thrombotic thrombocytopenic purpura 030204 cardiovascular system & hematology Placebo Gastroenterology 03 medical and health sciences 0302 clinical medicine Fibrinolytic Agents Refractory Internal medicine medicine Humans 610 Medicine & health Acquired Thrombotic Thrombocytopenic Purpura Plasma Exchange Purpura Thrombotic Thrombocytopenic business.industry Hazard ratio Hematology Single-Domain Antibodies medicine.disease Stimulus Report Tolerability 030220 oncology & carcinogenesis Caplacizumab business |
| Zdroj: | Blood Adv |
| ISSN: | 2473-9537 2473-9529 |
| DOI: | 10.1182/bloodadvances.2020001834 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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