重组凝血因子VIII Fc诱导甲型血友病免疫耐受：一项病历回顾研究的数据

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

作者信息

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

机构信息

Vivantes Klinikum Friedrichshain, Berlin, Germany.

King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia.

出版信息

Eur J Haematol. 2025 Aug;115(2):134-141. doi: 10.1111/ejh.14427. Epub 2025 Apr 27.

DOI:10.1111/ejh.14427

PMID:40289300

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12224564/

Abstract

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.

摘要

目的

报告一项ITI图表回顾性研究（NCT03951103）的数据，该研究针对A型血友病患者首次使用和挽救性使用重组因子VIII Fc融合蛋白（rFVIIIFc）进行免疫耐受诱导（ITI）。

方法

报告一项非干预性、多中心研究的回顾性和前瞻性真实世界数据，该研究的患者曾接受或正在接受rFVIIIFc ITI治疗。报告ITI治疗结果（由研究者定义）和治疗方案。

结果

纳入了来自16个地点的41名患者。24名患者接受首次ITI；16名患者在研究结束时获得ITI结果。13名患者（81.3%）ITI成功，3名失败。rFVIIIFc的中位（范围）消耗量为300（61 - 2800）IU/kg/周，大多数患者（70.8%）使用量≤300 IU/kg/周。绝大多数患者（87.5%）接受的ITI治疗频率低于每日一次。17名患者接受挽救性ITI；16名患者在研究结束时获得ITI结果。8名患者（50.0%）ITI成功/部分成功，7名失败，1名提前退出。rFVIIIFc的中位（范围）消耗量为536（98 - 1435）IU/kg/周；35.3%的患者使用量≤300 IU/kg/周，52.9%的患者使用量>500 IU/kg/周。大多数患者（64.7%）接受每日一次的ITI治疗。