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

{"title":"ORPHEE: A Real-World Study on rIX-FP Prophylaxis Use in Adolescent/Adult Patients With Hemophilia B","authors":"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","doi":"10.1111/ejh.14357","DOIUrl":null,"url":null,"abstract":"<div>\n \n \n <section>\n \n <h3> Objectives</h3>\n \n <p>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.</p>\n </section>\n \n <section>\n \n <h3> Methods</h3>\n \n <p>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.</p>\n </section>\n \n <section>\n \n <h3> Results</h3>\n \n <p>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) (<i>n</i> = 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.</p>\n </section>\n \n <section>\n \n <h3> Conclusion</h3>\n \n <p>This analysis confirmed that switching to rIX-FP allows for reducing injection frequency and FIX consumption while maintaining good bleed protection.</p>\n </section>\n </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 3","pages":"508-516"},"PeriodicalIF":2.3000,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14357","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"European Journal of Haematology","FirstCategoryId":"3","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1111/ejh.14357","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}

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.

Abstract Image

查看原文本刊更多论文

ORPHEE：青少年/成人B型血友病患者rIX-FP预防使用的现实世界研究。

目的：评估重组因子IX白蛋白融合蛋白（rIX-FP）在法国B型血友病（HB）患者中的实际疗效和安全性。方法：收集2021年12月至2024年2月期间rIX-FP给药频率、周用量和前后出血数据。年化（自发）出血率[A(s)BRs]仅在随访≥6个月的预防治疗患者中计算。结果：该中期分析集中于77例≥12岁的患者；62例（81%）有严重的HB。改用rIX-FP后，输注间隔为14（7-14）天。每周摄入量为43 (35.5-53)IU/kg。随访18.2（12.3-21.9）个月时，abr和asbr分别为0.5（0-1.9）和0 (0-0.7)（n = 63）。65/68例（95%）患者认为rIX-FP的预防效果为“优秀”/“良好”。在先前接受rFIXFc治疗的43例患者中，21例将rFIXFc的输注间隔从7（7-11）天增加到14（7-14）天；33/43（77%）将每周因子IX （FIX）摄入量从59.95（46.35-77.93）降低到42.5 (35.88-50.25)IU/kg。患者保持了良好的出血保护。结论：该分析证实，切换到rIX-FP可以减少注射频率和FIX消耗，同时保持良好的出血保护。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

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.