Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B - Aix-Marseille Université
Journal Articles Blood Advances Year : 2023

Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B

Amy Shapiro
  • Function : Author
Roshni Kulkarni
Margaret Ragni
Johnny Mahlangu
  • Function : Author
Johannes Oldenburg
  • Function : Author
Beatrice Nolan
Margareth Ozelo
Meredith Foster
  • Function : Author
Annemieke Willemze
  • Function : Author
Christopher Barnowski
  • Function : Author
Nisha Jain
  • Function : Author
Bent Winding
  • Function : Author
Jennifer Dumont
  • Function : Author
Stefan Lethagen
Chris Barnes
  • Function : Author
K. John Pasi

Abstract

Long-term efficacy and safety of the extended half-life recombinant factor IX Fc fusion protein (rFIXFc) has been established among previously treated patients with severe hemophilia B in 2 phase 3 trials (B-LONG [#NCT01027364] and Kids B-LONG [#NCT01440946]) and a long-term extension study (B-YOND [#NCT01425723]). In this study, we report post hoc analyses of pooled longitudinal data for up to 6.5 years for rFIXFc prophylaxis. In the B-LONG study, subjects ≥12 years received weekly dose-adjusted prophylaxis (WP; starting dose, 50 IU/kg), individualized interval-adjusted prophylaxis (IP; initially, 100 IU/kg every 10 days), or on-demand dosing. In the Kids B-LONG study, subjects <12 years received 50 to 60 IU/kg every 7 days, adjusted as needed. In the B-YOND study, subjects received WP (20-100 IU/kg every 7 days), IP (100 IU/kg every 8-16 days), modified prophylaxis, or on-demand dosing; switching between treatment groups was permitted. A total of 123 subjects from B-LONG and 30 from Kids B-LONG study were included, of whom 93 and 27, respectively, enrolled in the B-YOND study. The median cumulative duration of treatment was 3.63 years (range, 0.003-6.48 years) in B-LONG/B-YOND and 2.88 years (range, 0.30-4.80 years) in Kids B-LONG/B-YOND group. Annualized bleed rates (ABRs) remained low, annualized factor consumption remained stable, and adherence remained high throughout treatment. Low ABRs were also maintained in subjects with dosing intervals ≥14 days or with target joints at baseline. Complete resolution of evaluable target joints and no recurrence in 90.2% of baseline target joints during follow-up were observed. rFIXFc prophylaxis was associated with sustained clinical benefits, including long-term bleed prevention and target joint resolution, for severe hemophilia B.

Dates and versions

hal-04423676 , version 1 (29-01-2024)

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Amy Shapiro, Roshni Kulkarni, Margaret Ragni, Hervé Chambost, Johnny Mahlangu, et al.. Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B. Blood Advances, 2023, 7 (13), pp.3049-3057. ⟨10.1182/bloodadvances.2022009230⟩. ⟨hal-04423676⟩
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