Hervé Chambost, Yohann Repessé, Fabienne Genre-Volot, Dominique Desprez, Sabine Marie Castet, Stéphane Vanderbecken, Meriem Zidi, Corinne Gandossi, Eveline Nüesch, Helena Palmborg, Elena Santagostino
{"title":"重组因子IX Fc在血友病B中的实际使用和有效性，来自法国B- sure研究。","authors":"Hervé Chambost, Yohann Repessé, Fabienne Genre-Volot, Dominique Desprez, Sabine Marie Castet, Stéphane Vanderbecken, Meriem Zidi, Corinne Gandossi, Eveline Nüesch, Helena Palmborg, Elena Santagostino","doi":"10.1177/20406207241311535","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>More real-world data are needed to complement existing phase III studies on the efficacy and safety of recombinant factor IX Fc fusion protein (rFIXFc) in people with haemophilia B.</p><p><strong>Objectives: </strong>We report final data from the B-SURE study, evaluating the real-world usage and effectiveness of rFIXFc in France.</p><p><strong>Methods: </strong>Previously treated patients (all ages/severities) received on-demand or prophylactic rFIXFc during B-SURE. Annualised bleeding rate (ABR), injection frequency (IF) and factor consumption (FC) were prospectively evaluated for patients on rFIXFc prophylaxis (primary endpoints). Six months of retrospective factor IX (FIX) data were collected for comparison; patients with ⩾3 months of treatment pre- and post-switch to rFIXFc were analysed.</p><p><strong>Design: </strong>B-SURE was a 24-month, prospective, non-interventional, real-world study across haemophilia treatment centres in France.</p><p><strong>Results: </strong>Ninety-one male patients enrolled across 21 centres (34% <18 years, 89% severe haemophilia B). Eighty-four patients received prophylaxis at rFIXFc initiation; mean prospective observation period was 21.5 months. Sixty-eight of 84 patients had prior FIX prophylaxis; on rFIXFc prophylaxis, these patients achieved low median ABR (1.2), IF (47.45 injections/year) and mean FC (2844 IU/kg/year). Compared with previous FIX, mean ABR was reduced by 40% (<i>n</i> = 63); mean IF and FC were reduced by 38.20 injections/year and 1008 IU/kg/year (<i>n</i> = 57). In patients with prior FIX on-demand (<i>n</i> = 15), mean ABR reduced by 84% on rFIXFc prophylaxis (<i>n</i> = 14), mean IF reduced by 2.13 injections/year and mean FC increased by 381.8 IU/kg/year (<i>n</i> = 15). Most physicians and patients were satisfied/highly satisfied with rFIXFc prophylaxis. rFIXFc was well tolerated with no new safety concerns.</p><p><strong>Conclusion: </strong>Findings support the safety and effectiveness of rFIXFc, with reduced IF and FC while maintaining/improving bleed protection. <b><i>Trial registration</i>:</b> NCT03655340.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":"16 ","pages":"20406207241311535"},"PeriodicalIF":3.4000,"publicationDate":"2025-01-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11770721/pdf/","citationCount":"0","resultStr":"{\"title\":\"Real-world usage and effectiveness of recombinant factor IX Fc in haemophilia B from the B-SURE study in France.\",\"authors\":\"Hervé Chambost, Yohann Repessé, Fabienne Genre-Volot, Dominique Desprez, Sabine Marie Castet, Stéphane Vanderbecken, Meriem Zidi, Corinne Gandossi, Eveline Nüesch, Helena Palmborg, Elena Santagostino\",\"doi\":\"10.1177/20406207241311535\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>More real-world data are needed to complement existing phase III studies on the efficacy and safety of recombinant factor IX Fc fusion protein (rFIXFc) in people with haemophilia B.</p><p><strong>Objectives: </strong>We report final data from the B-SURE study, evaluating the real-world usage and effectiveness of rFIXFc in France.</p><p><strong>Methods: </strong>Previously treated patients (all ages/severities) received on-demand or prophylactic rFIXFc during B-SURE. Annualised bleeding rate (ABR), injection frequency (IF) and factor consumption (FC) were prospectively evaluated for patients on rFIXFc prophylaxis (primary endpoints). Six months of retrospective factor IX (FIX) data were collected for comparison; patients with ⩾3 months of treatment pre- and post-switch to rFIXFc were analysed.</p><p><strong>Design: </strong>B-SURE was a 24-month, prospective, non-interventional, real-world study across haemophilia treatment centres in France.</p><p><strong>Results: </strong>Ninety-one male patients enrolled across 21 centres (34% <18 years, 89% severe haemophilia B). Eighty-four patients received prophylaxis at rFIXFc initiation; mean prospective observation period was 21.5 months. Sixty-eight of 84 patients had prior FIX prophylaxis; on rFIXFc prophylaxis, these patients achieved low median ABR (1.2), IF (47.45 injections/year) and mean FC (2844 IU/kg/year). Compared with previous FIX, mean ABR was reduced by 40% (<i>n</i> = 63); mean IF and FC were reduced by 38.20 injections/year and 1008 IU/kg/year (<i>n</i> = 57). In patients with prior FIX on-demand (<i>n</i> = 15), mean ABR reduced by 84% on rFIXFc prophylaxis (<i>n</i> = 14), mean IF reduced by 2.13 injections/year and mean FC increased by 381.8 IU/kg/year (<i>n</i> = 15). Most physicians and patients were satisfied/highly satisfied with rFIXFc prophylaxis. rFIXFc was well tolerated with no new safety concerns.</p><p><strong>Conclusion: </strong>Findings support the safety and effectiveness of rFIXFc, with reduced IF and FC while maintaining/improving bleed protection. <b><i>Trial registration</i>:</b> NCT03655340.</p>\",\"PeriodicalId\":23048,\"journal\":{\"name\":\"Therapeutic Advances in Hematology\",\"volume\":\"16 \",\"pages\":\"20406207241311535\"},\"PeriodicalIF\":3.4000,\"publicationDate\":\"2025-01-26\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11770721/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Therapeutic Advances in Hematology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1177/20406207241311535\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"Q2\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Therapeutic Advances in Hematology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1177/20406207241311535","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/1/1 0:00:00","PubModel":"eCollection","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
Real-world usage and effectiveness of recombinant factor IX Fc in haemophilia B from the B-SURE study in France.
Background: More real-world data are needed to complement existing phase III studies on the efficacy and safety of recombinant factor IX Fc fusion protein (rFIXFc) in people with haemophilia B.
Objectives: We report final data from the B-SURE study, evaluating the real-world usage and effectiveness of rFIXFc in France.
Methods: Previously treated patients (all ages/severities) received on-demand or prophylactic rFIXFc during B-SURE. Annualised bleeding rate (ABR), injection frequency (IF) and factor consumption (FC) were prospectively evaluated for patients on rFIXFc prophylaxis (primary endpoints). Six months of retrospective factor IX (FIX) data were collected for comparison; patients with ⩾3 months of treatment pre- and post-switch to rFIXFc were analysed.
Design: B-SURE was a 24-month, prospective, non-interventional, real-world study across haemophilia treatment centres in France.
Results: Ninety-one male patients enrolled across 21 centres (34% <18 years, 89% severe haemophilia B). Eighty-four patients received prophylaxis at rFIXFc initiation; mean prospective observation period was 21.5 months. Sixty-eight of 84 patients had prior FIX prophylaxis; on rFIXFc prophylaxis, these patients achieved low median ABR (1.2), IF (47.45 injections/year) and mean FC (2844 IU/kg/year). Compared with previous FIX, mean ABR was reduced by 40% (n = 63); mean IF and FC were reduced by 38.20 injections/year and 1008 IU/kg/year (n = 57). In patients with prior FIX on-demand (n = 15), mean ABR reduced by 84% on rFIXFc prophylaxis (n = 14), mean IF reduced by 2.13 injections/year and mean FC increased by 381.8 IU/kg/year (n = 15). Most physicians and patients were satisfied/highly satisfied with rFIXFc prophylaxis. rFIXFc was well tolerated with no new safety concerns.
Conclusion: Findings support the safety and effectiveness of rFIXFc, with reduced IF and FC while maintaining/improving bleed protection. Trial registration: NCT03655340.
期刊介绍：
Therapeutic Advances in Hematology delivers the highest quality peer-reviewed articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of hematology. The journal has a strong clinical and pharmacological focus and is aimed at clinicians and researchers in hematology, providing a forum in print and online for publishing the highest quality articles in this area.
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
