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Vol. 43. Núm. S3.
Páginas S46-S47 (Novembro 2021)
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Vol. 43. Núm. S3.
Páginas S46-S47 (Novembro 2021)
PP 37
Open Access
INTEGRATED EFFICACY RESULTS FROM THE PHASE 2 AND PHASE 3 STUDIES WITH CAPLACIZUMAB IN PATIENTS WITH ACQUIRED THROMBOTIC THROMBOCYTOPENIC PURPURA
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...
Flora Peyvandi1, Spero Cataland2, Marie Scully3, Paul Coppo4, Paul Knoebl5, Johanna A. Kremer Hovinga6, Ara Metjian7, Javier de la Rubia8, Katerina Pavenski9, Jessica Minkue Mi Edou10, Filip Callewaert11, Hilde De Winter12
1 Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, and Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy
2 Division of Hematology, Department of Internal Medicine, The Ohio State University, Columbus, OH, USA
3 Department of Haematology, University College London Hospitals NHS Trust, London, UK
4 Department of Hematology, Reference Center for Thrombotic Microangiopathies (CNR-MAT), Saint-Antoine University Hospital, AP-HP, Paris, France
5 Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Austria
6 Department of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland
7 Division of Hematology, Department of Medicine, University of Colorado–Anschutz Medical Center, Denver, CO, USA
8 Hematology Department, Internal Medicine, School of Medicine and Dentistry, Catholic University of Valencia and Hospital Doctor Peset, Valencia, Spain
9 Departments of Medicine and Laboratory Medicine, St. Michael's Hospital and University of Toronto, Toronto, ON, Canada
10 Clinical Development, Ablynx, a Sanofi company, Ghent, Belgium
11 Medical Affairs, Sanofi, Diegem, Belgium
12 Formerly Clinical Development, Ablynx, a Sanofi company, Ghent, Belgium
Informação do artigo
Objective

An integrated analysis based on the Phase 2 TITAN (NCT01151423) and Phase 3 HERCULES (NCT02553317) studies with caplacizumab (CPLZ) in acquired thrombotic thrombocytopenic purpura (aTTP) was performed to assess treatment differences on efficacy and safety outcomes that may have been undetected in the individual trials.

Methodology

In both trials, patients with an acute episode of aTTP were randomized to receive CPLZ or placebo (PBO) in addition to therapeutic plasma exchange (TPE) and immunosuppression. All randomized patients from both studies were included in the integrated efficacy analyses (CPLZ: n=108; PBO: n=112), and those who received at least 1 dose of the study drug were included in the safety analyses (CPLZ: n=106; PBO: n=110).

Results

CPLZ significantly reduced mortality (0 vs 4 deaths; P<0.05) and refractory TTP (0 vs 8 events; P<0.05) versus PBO and improved time to platelet count response (hazard ratio, 1.65; P<0.001). CPLZ also reduced the composite endpoint of TTP-related death, exacerbation, or any treatment-emergent major thromboembolic event during the treatment period (13.0% vs 47.3%; P<0.001) and median number of TPE days (5.0 vs 7.5 days) versus PBO. Mild mucocutaneous bleeding was the main safety finding for CPLZ.

Conclusion

This integrated analysis provided new evidence that CPLZ prevents mortality and refractory disease in aTTP and reinforced the individual trial efficacy and safety findings. No new safety signals were identified for

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Idiomas
Hematology, Transfusion and Cell Therapy

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