Journal article
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)
Haemophilia : the official journal of the World Federation of Hemophilia, Vol.25(3), pp.373-381
05/2019
DOI: 10.1111/hae.13712
PMCID: PMC6850405
PMID: 30817066
Abstract
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.
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 ≤2 bleeds during the preceding 6 months of the pathfinder 2 main phase were eligible for randomization to receive N8-GP 50 IU/kg Q4D or 75 IU/kg Q7D. Safety and efficacy endpoints were incidence of FVIII inhibitors and annualized bleeding rate (ABR), respectively.
Fifty-five of 143 (38.5%) patients on prophylaxis who continued into the extension phase were randomized to receive 50 IU/kg Q4D (n = 17) or 75 IU/kg Q7D (n = 38). Nine patients in the Q7D cohort reverted to 50 IU/kg Q4D. No inhibitors were detected. In both cohorts, >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 ≤2 injections.
Weekly N8-GP was well tolerated and efficacious and may benefit selected "low bleeder" patients with haemophilia A.
Details
- Title: Subtitle
- 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)
- Creators
- Nicola Curry - Churchill HospitalCanan Albayrak - Ondokuz Mayıs UniversityMiguel Escobar - The University of Texas Health Science CenterPål Andre Holme - Oslo University HospitalSusan Kearney - Children’s Minnesota - St. Paul HospitalRobert Klamroth - Klinikum im FriedrichshainMudi Misgav - Sheba Medical CenterClaude Négrier - Hôpital Louis PradelAllison Wheeler - Vanderbilt UniversityElena Santagostino - Fondazione IRCCS Ca' Granda Ospedale Maggiore PoliclinicoMidori Shima - Nara Medical UniversityAndrea Landorph - Novo NordiskSidsel Marie Tønder - Novo NordiskSteven R Lentz - Roy J. and Lucille A. Carver College of Medicine
- Resource Type
- Journal article
- Publication Details
- Haemophilia : the official journal of the World Federation of Hemophilia, Vol.25(3), pp.373-381
- DOI
- 10.1111/hae.13712
- PMID
- 30817066
- PMCID
- PMC6850405
- ISSN
- 1351-8216
- eISSN
- 1365-2516
- Grant note
- Novo Nordisk A/S (Bagsvaerd, Denmark).
- Language
- English
- Date published
- 05/2019
- Academic Unit
- Hematology, Oncology, and Blood & Marrow Transplantation; Internal Medicine
- Record Identifier
- 9984359563202771
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