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Type: Artigo
Title: Long-term Safety And Efficacy Of Extended-interval Prophylaxis With Recombinant Factor Ix Fc Fusion Protein (rfixfc) In Subjects With Haemophilia B
Author: Pasi
K. John; Fischer
Kathelijn; Ragni
Margaret; Nolan
Beatrice; Perry
David J.; Kulkarni
Roshni; Ozelo
Margareth; Mahlangu
Johnny; Shapiro
Amy D.; Baker
Ross I.; Bennett
Carolyn M.; Barnes
Christopher; Oldenburg
Johannes; Matsushita
Tadashi; Yuan
Huixing; Ramirez-Santiago
Alejandra; Pierce
Glenn F.; Allen
Geoffrey; Mei
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.
Subject: Factor Ix
Recombinant Fusion Proteins
Editor: Schattauer GMBH - Verlag Medizin Naturwissenschaften
Citation: Thrombosis And Haemostasis . Schattauer Gmbh - Verlag Medizin Naturwissenschaften , v. 117, p. 508 - 518, 2017.
Rights: fechado
Identifier DOI: 10.1160/TH16-05-0398
Date Issue: 2017
Appears in Collections:Unicamp - Artigos e Outros Documentos

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