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).
- Abstract:
- 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 ≤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. Results 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. Conclusions Weekly N8‐GP was well tolerated and efficacious and may benefit selected “low bleeder” patients with haemophilia A.
- Publication status:
- Published
- Peer review status:
- Peer reviewed
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(Preview, Version of record, 542.7KB, Terms of use)
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- Publisher copy:
- 10.1111/hae.13712
Authors
- Publisher:
- Wiley
- Journal:
- Haemophilia More from this journal
- Volume:
- 25
- Issue:
- 3
- Pages:
- 373-381
- Publication date:
- 2019-02-28
- Acceptance date:
- 2019-01-28
- DOI:
- EISSN:
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1365-2516
- ISSN:
-
1351-8216
- Pmid:
-
30817066
- Language:
-
English
- Keywords:
- Pubs id:
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1090491
- Local pid:
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pubs:1090491
- Deposit date:
-
2020-03-06
Terms of use
- Copyright holder:
- Curry et al.
- Copyright date:
- 2019
- Rights statement:
- © 2019 The Authors. Haemophilia Published by John Wiley & Sons Ltd. This is an open access article under the terms of the Creative Commons Attribution‐NonCommercial‐NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.
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