ORPHEE: A Real-World Study on rIX-FP Prophylaxis Use in Adolescent/Adult Patients With Hemophilia B.

IF 2.3 3区医学 Q2 HEMATOLOGY

European Journal of Haematology Pub Date : 2024-12-05 DOI:10.1111/ejh.14357

Fabienne Volot, Sabine Castet, Alexandra Fournel, Birgit Frotscher, Benjamin Gillet, Dominique Desprez, Brigitte Tardy, Antoine Rauch, Pierre Chamouni, Christine Biron-Andreani, Jean-Baptiste Valentin, Annie Harroche, Yesim Dargaud, Brigitte Pan Petesch, Roseline d'Oiron, Claire Berger, Claire Reynes, Thomas Lauvray, Emmanuelle de Raucourt, Abel Hassoun, Aurélien Lebreton, Vincent Cussac, Hasan Catovic, Cédric Martin, Benoit Guillet

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引用次数: 0

Abstract

Objectives: To assess the real-world efficacy and safety of recombinant factor IX albumin fusion protein (rIX-FP) in patients with hemophilia B (HB) in France.

Methods: Data on dosing frequency, weekly consumption, and bleeds before-and-after switching to rIX-FP, were collected from December 2021 to February 2024. Annualized (spontaneous) bleeding rates [A(s)BRs] were calculated only in patients on prophylaxis with a follow-up ≥ 6 months.

Results: This interim analysis focused on 77 patients ≥ 12 years; 62 (81%) had severe HB. After switching to rIX-FP, the infusion interval was 14 (7-14) days. Weekly consumption was 43 (35.5-53) IU/kg. ABRs and AsBRs were 0.5 (0-1.9) and 0 (0-0.7) (n = 63) at 18.2 (12.3-21.9) months of follow-up. Prophylactic efficacy of rIX-FP was considered 'Excellent'/'Good' in 65/68 (95%) patients. Among the 43 patients previously treated with rFIXFc, 21 increased the infusion interval from 7 (7-11) days with rFIXFc to 14 (7-14) days with rIX-FP; 33/43 (77%) reduced weekly factor IX (FIX) consumption from 59.95 (46.35-77.93) to 42.5 (35.88-50.25) IU/kg. Patients maintained good protection against bleeds.

Conclusion: This analysis confirmed that switching to rIX-FP allows for reducing injection frequency and FIX consumption while maintaining good bleed protection.

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来源期刊

European Journal of Haematology 医学-血液学

CiteScore

5.50

自引率

0.00%

发文量

168

审稿时长

4-8 weeks

期刊介绍： European Journal of Haematology is an international journal for communication of basic and clinical research in haematology. The journal welcomes manuscripts on molecular, cellular and clinical research on diseases of the blood, vascular and lymphatic tissue, and on basic molecular and cellular research related to normal development and function of the blood, vascular and lymphatic tissue. The journal also welcomes reviews on clinical haematology and basic research, case reports, and clinical pictures.