Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura
| Autor: | Paul Knöbl, Javier de la Rubia, Johanna A. Kremer Hovinga, Spero R. Cataland, Ara Metjian, Flora Peyvandi, Paul Coppo, Marie Scully, Debjit Biswas, R.K. Zeldin, Filip Callewaert, Hercules Investigators, Hilde De Winter, Katerina Pavenski |
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| Přispěvatelé: | University of Zurich, De Winter, Hilde |
| Jazyk: | angličtina |
| Rok vydání: | 2019 |
| Předmět: |
medicine.medical_specialty
610 Medicine & health 2700 General Medicine 030204 cardiovascular system & hematology Placebo Gastroenterology law.invention 03 medical and health sciences 0302 clinical medicine Bolus (medicine) Randomized controlled trial law Internal medicine hemic and lymphatic diseases Medicine Platelet 030212 general & internal medicine caplacizumab treatment for acquired Intention-to-treat analysis Acquired Thrombotic Thrombocytopenic Purpura business.industry General Medicine ADAMTS13 Settore MED/15 - MALATTIE DEL SANGUE 10032 Clinic for Oncology and Hematology Caplacizumab business |
| ISSN: | 0255-3317 |
| Popis: | BACKGROUND In acquired thrombotic thrombocytopenic purpura (TTP), an immune-mediated deficiency of the von Willebrand factor-cleaving protease ADAMTS13 allows unrestrained adhesion of von Willebrand factor multimers to platelets and microthrombosis, which result in thrombocytopenia, hemolytic anemia, and tissue ischemia. Caplacizumab, an anti-von Willebrand factor humanized, bivalent variable-domain-only immunoglobulin fragment, inhibits interaction between von Willebrand factor multimers and platelets. METHODS In this double-blind, controlled trial, we randomly assigned 145 patients with TTP to receive caplacizumab (10-mg intravenous loading bolus, followed by 10 mg daily subcutaneously) or placebo during plasma exchange and for 30 days thereafter. The primary outcome was the time to normalization of the platelet count, with discontinuation of daily plasma exchange within 5 days thereafter. Key secondary outcomes included a composite of TTP-related death, recurrence of TTP, or a thromboembolic event during the trial treatment period; recurrence of TTP at any time during the trial; refractory TTP; and normalization of organ-damage markers. RESULTS The median time to normalization of the platelet count was shorter with caplacizumab than with placebo (2.69 days [95% confidence interval {CI}, 1.89 to 2.83] vs. 2.88 days [95% CI, 2.68 to 3.56], P=0.01), and patients who received caplacizumab were 1.55 times as likely to have a normalization of the platelet count as those who received placebo. The percentage of patients with a composite outcome event was 74% lower with caplacizumab than with placebo (12% vs. 49%, P |
| Databáze: | OpenAIRE |
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