PERSEPT 1: a phase 3 trial of activated eptacog beta for on-demand treatment of haemophilia inhibitor-related bleeding
| Autor: | Jonathan M. Ducore, W. A. Alexander, Mindy L. Simpson, Lisa N. Boggio, J.-F. Schved, Ian S. Mitchell, G. Yuan, Michael Wang, Doris Quon, J. B. Lawrence |
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| Rok vydání: | 2017 |
| Předmět: |
Adult
Male medicine.medical_specialty Adolescent Haemophilia A Population Hemorrhage Factor VIIa 030204 cardiovascular system & hematology Haemophilia Hemophilia A Gastroenterology Hemophilia B 03 medical and health sciences Young Adult 0302 clinical medicine Internal medicine Hemarthrosis Clinical endpoint medicine Humans Dosing Prospective Studies education Beta (finance) Genetics (clinical) education.field_of_study Cross-Over Studies Dose-Response Relationship Drug business.industry Immunogenicity Headache Hematology General Medicine Middle Aged medicine.disease Recombinant Proteins Clinical trial business 030215 immunology |
| Zdroj: | Haemophilia : the official journal of the World Federation of Hemophilia. 23(6) |
| ISSN: | 1365-2516 |
| Popis: | Introduction Haemophilia A or B patients with inhibitors have been treated with FVIIa-containing bypassing agents for over 20 years. However, due to uncertainty regarding dose response and thrombotic risk, the use of a gradual, titrated, minimal dosing strategy remains prevalent, potentially hampering early haemostasis. Aim Evaluate the dose-dependent efficacy, safety and immunogenicity of activated eptacog beta (rhFVIIa), a new recombinant inhibitor bypassing agent for the treatment of bleeding episodes (BEs). Methods A Phase 3, randomized, cross-over study of initial dose regimens (IDRs) in 27 bleeding congenital haemophilia A or B subjects with inhibitors was conducted to evaluate on-demand treatment of mild/moderate BEs. Intravenous 75 μg/kg or 225 μg/kg initial doses with 75 μg/kg subsequent doses by schedule were administered until clinical response. Results The primary endpoint was sustained clinical response within 12 hours, determined by a composite of objective and pain measures. In the 75 μg/kg IDR, 84.9% (95% CI; 74.0%, 95.7%) of mild/moderate BEs at 12 hours were successfully treated compared to 93.2% (95% CI; 88.1%, 98.3%) treated in the 225 μg/kg IDR. Efficacy between the IDRs was statistically different (P |
| Databáze: | OpenAIRE |
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