Once-weekly prophylaxis with glycoPEGylated recombinant factor VIII (N8-GP) in severe haemophilia A: Safety and efficacy results from pathfinder 2 (randomized phase III trial)
| Autor: | Pål Andre Holme, Elena Santagostino, Steven R. Lentz, Claude Negrier, Andrea Landorph, Mudi Misgav, Canan Albayrak, Allison P. Wheeler, Miguel A. Escobar, Robert Klamroth, Nicola Curry, Susan Kearney, Sidsel Marie Tønder, Midori Shima |
|---|---|
| Přispěvatelé: | OMÜ |
| Jazyk: | angličtina |
| Rok vydání: | 2019 |
| Předmět: |
Adult
Male FVIII safety medicine.medical_specialty Randomization Haemophilia A efficacy Hemorrhage haemophilia A 030204 cardiovascular system & hematology Hemophilia A Haemophilia Drug Administration Schedule Polyethylene Glycols Recombinant factor VIII N8 N8-GP 03 medical and health sciences 0302 clinical medicine Internal medicine medicine Humans once‐weekly prophylaxis Clinical Haemophilia Genetics (clinical) Factor VIII business.industry Incidence (epidemiology) Hematology General Medicine Turoctocog alfa medicine.disease N8‐GP once-weekly prophylaxis Cohort Original Article Female Severe haemophilia A ORIGINAL ARTICLES business 030215 immunology |
| Zdroj: | Haemophilia |
| ISSN: | 1351-8216 |
| Popis: | Curry, Nicola/0000-0002-3849-0688; Escobar, Miguel/0000-0002-2944-0240 WOS: 000470929100022 PubMed: 30817066 Introduction Turoctocog alfa pegol (N8-GP) is a site-specific, 40 kDa glycoPEGylated recombinant factor VIII (FVIII) product with an extended half-life. The comprehensive main phase of the pivotal pathfinder 2 trial showed N8-GP dosed every 4 days (Q4D) provided favourable safety and efficacy for preventing bleeds in 175 patients with haemophilia A. Aim and methods We investigated the safety and efficacy of N8-GP prophylaxis when administered weekly (Q7D) for 24 weeks to patients with low bleeding rates in the pathfinder 2 extension trial. Patients (>= 12 years) with 50% of patients experienced no bleeds. Median ABR for overall, joint, spontaneous, traumatic and muscle was 0.00 for both cohorts. Overall estimated success rate for treating bleeding episodes was 87.5%; 94.7% of bleeds were controlled with |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|
Plný text