Journal article
Long-term safety and efficacy of extended-interval prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) in subjects with haemophilia B
Thrombosis and Haemostasis, Vol.117(3), pp.508-518
2016
Abstract
The safety, efficacy, and prolonged half-life of recombinant factor IX Fc fusion protein (rFIXFc) were demonstrated in the Phase 3 B-LONG (adults/adolescents ≥12 years) and Kids B-LONG (children <12 years) studies of subjects with haemophilia B (≤2 IU/dl). Here, we report interim, long-term safety and efficacy data from B-YOND, the rFIXFc extension study. Eligible subjects who completed B-LONG or Kids B-LONG could enrol in B-YOND. There were four treatment groups: weekly prophylaxis (20–100 IU/kg every 7 days), individualised prophylaxis (100 IU/kg every 8–16 days), modified prophylaxis (further dosing personalisation to optimise prophylaxis), and episodic (on-demand) treatment. Subjects could change treatment groups at any point. Primary endpoint was inhibitor development. One hundred sixteen subjects enrolled in B-YOND. From the start of the parent studies to the B-YOND interim data cut, median duration of rFIXFc treatment was 39.5 months and 21.9 months among adults/adolescents and children, respectively; 68/93 (73.1 %) adults/adolescents and 9/23 (39.1 %) children had ≥100 cumulative rFIXFc exposure days. No inhibitors were observed. Median annualised bleeding rates (ABRs) were low in all prophylaxis regimens: weekly (≥12 years: 2.3; <6 years: 0.0; 6 to <12 years: 2.7), individualised (≥12 years: 2.3; 6 to <12 years: 2.4), and modified (≥12 years: 2.4). One or two infusions were sufficient to control 97 % (adults/adolescents) and 95 % (children) of bleeding episodes. Interim data from B-YOND are consistent with data from B-LONG and Kids B-LONG, and confirm the long-term safety of rFIXFc, absence of inhibitors, and maintenance of low ABRs with prophylactic dosing every 1 to 2 weeks.
Details
- Title
- Long-term safety and efficacy of extended-interval prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) in subjects with haemophilia B
- Authors/Creators
- K.J. Pasi (Author/Creator) - Queen Mary University of LondonK. Fischer (Author/Creator) - University College UtrechtM. Ragni (Author/Creator) - University of PittsburghB. Nolan (Author/Creator) - Our Lady's HospitalD.J. Perry (Author/Creator) - Addenbrooke's HospitalR. Kulkarni (Author/Creator) - Health and Human Development (2HD) Research NetworkM. Ozelo (Author/Creator) - Universidade Estadual de Campinas (UNICAMP)J. Mahlangu (Author/Creator) - University of the WitwatersrandA.D. Shapiro (Author/Creator) - Indiana Hemophilia and Thrombosis CenterR.I. Baker (Author/Creator) - Murdoch UniversityC.M. Bennett (Author/Creator) - Emory UniversityC. Barnes (Author/Creator) - Royal Children's HospitalJ. Oldenburg (Author/Creator) - University of BonnT. Matsushita (Author/Creator) - Nagoya UniversityH. Yuan (Author/Creator) - Biogen (Germany)A. Ramirez-Santiago (Author/Creator) - Biogen (Germany)G.F. Pierce (Author/Creator) - Biogen (United States)G. Allen (Author/Creator) - Biogen (Germany)B. Mei (Author/Creator) - Biogen (United States)
- Publication Details
- Thrombosis and Haemostasis, Vol.117(3), pp.508-518
- Publisher
- Schattauer
- Identifiers
- 991005545065507891
- Copyright
- © 2016 Schattauer Publishers
- Murdoch Affiliation
- Institute for Immunology and Infectious Diseases
- Language
- English
- Resource Type
- Journal article
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- Collaboration types
- Industry collaboration
- Domestic collaboration
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- Citation topics
- 1 Clinical & Life Sciences
- 1.75 Blood Clotting
- 1.75.619 Bleeding Disorders
- Web Of Science research areas
- Hematology
- Peripheral Vascular Disease
- ESI research areas
- Clinical Medicine