重组因子VIII Fc诱导血友病a的免疫耐受：来自一项图表回顾研究的数据

IF 2.3 3区医学 Q2 HEMATOLOGY

European Journal of Haematology Pub Date : 2025-04-27 DOI:10.1111/ejh.14427

Robert Klamroth, Mahasen Al Saleh, Heidi Glosli, Michele Schiavulli, Benoît Guillet, Linda Bystrická, Anton Schönstein, Stefan Lethagen

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引用次数: 0

摘要

目的：报告一项ITI图表回顾研究（NCT03951103）的数据，该研究针对血友病a患者首次和救援性ITI使用重组因子VIIIFc融合蛋白（rfviii ifc）。方法：回顾性和前瞻性现实世界数据来自一项非介入性、多中心研究，研究对象是曾经或正在接受rfviii ifc ITI治疗的患者。报告ITI治疗结果（由研究者定义）和治疗方案。结果：共纳入16个部位的41例患者。24例患者首次行ITI；16例在研究结束时有ITI结果。13例（81.3%）患者ITI成功，3例失败。中位（范围）rfviii - ifc摄入量为300 (61-2800)IU/kg/周，大多数（70.8%）使用≤300 IU/kg/周。绝大多数患者（87.5%）接受的ITI少于每日。17例患者采用ITI抢救；16例在研究结束时有ITI结果。8例患者（50.0%）ITI成功/部分成功，7例失败，1例提前退出。中位（范围）rfviii ifc摄入量为536 (98-1435)IU/kg/周；35.3%≤300iu /kg/week, 52.9%≤500iu /kg/week。大多数患者（64.7%）每天接受ITI。结论：在首次ITI患者中，rfviii ifc可能会成功，对于以前经历过ITI失败的患者来说，这是一个有效的选择。试验注册：ClinicalTrials.gov标识符：NCT03951103。

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Immune Tolerance Induction With a Recombinant Factor VIII Fc in Haemophilia A: Data From a Chart Review Study.

Objective: To report data from an ITI chart review study (NCT03951103) for first-time and rescue ITI with recombinant factor VIII Fc fusion protein (rFVIIIFc) in persons with haemophilia A.

Methods: Retrospective and prospective real-world data are reported from a non-interventional, multicentre study of patients who had been or were currently being treated with rFVIIIFc ITI. ITI treatment outcome (defined by investigators) and regimens are reported.

Results: Forty-one patients from 16 sites were included. First-time ITI was used in 24 patients; 16 had an ITI outcome at study end. Thirteen patients (81.3%) had ITI success, and three had failure. Median (range) rFVIIIFc consumption was 300 (61-2800) IU/kg/week, and most (70.8%) used ≤ 300 IU/kg/week. The vast majority of patients (87.5%) received less than daily ITI. Rescue ITI was used in 17 patients; 16 had an ITI outcome at study end. Eight patients (50.0%) had ITI success/partial success, seven had failure, and one withdrew early. Median (range) rFVIIIFc consumption was 536 (98-1435) IU/kg/week; 35.3% used ≤ 300 IU/kg/week and 52.9% used > 500 IU/kg/week. Most patients (64.7%) received daily ITI.

Conclusion: ITI with rFVIIIFc is likely to be successful in first-time ITI patients and is an effective option for those who have previously experienced ITI failure.

Trial registration: ClinicalTrials.gov identifier: NCT03951103.

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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.