CFTR调节剂的免疫原性不良事件-一项国际调查。

IF 5.4 2区 医学 Q1 RESPIRATORY SYSTEM
Ruth M Urbantat, Laura Behan, Sebastian Wisniewski, Joshua Gardner, Mirjam Stahl, Marcus A Mall, Daniel Peckham, Dean J Naisbitt, Jobst F Roehmel
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引用次数: 0

摘要

背景:CFTR调节剂治疗对CF (pwCF)患者具有前所未有的积极作用。然而,对CFTR调节剂治疗的免疫原性反应可能导致停药。我们的目的是确定pwCF,由于疑似免疫原性不良事件(iAE)而对CFTR调节剂治疗不耐受。方法:本调查评估了再暴露后的反应类型(如皮疹、肝损伤、药物热),由ECFS CTN中心完成。结果:调查回复率为74%。28个国家的89个CF中心治疗了大约12000至17500个pwCF, 75个(84%)CF中心报告停止了CFTR调节剂治疗。37个(41.1%)CF中心报告iAE影响200例(1.1 - 1.7%)pwCF。200个pwCF中有41个(占受影响的20.5%)提供了iae的详细信息。在详细报道的iae中,33/41例(80.5%)与elexaftor /tezacaftor/ivacaftor调节剂治疗相关,6例(14.6%)与lumacaftor/ivacaftor相关,2例(4.9%)与tezacaftor/ivacaftor相关。72%的iAE pwCF患者再次接受CFTR调节剂治疗。32%的再暴露pwCF报告了第二次iAE。皮疹和肝酶升高是最常见的iae报告。结论:iAE多为短暂性。对CFTR调节剂治疗的药物过敏是罕见的,但与个体pwCF高度相关。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Immunogenic adverse events to CFTR modulators - An international survey.

Background: CFTR modulator therapy has unprecedented positive effects on people with CF (pwCF). However, immunogenic reactions to CFTR modulator therapy may lead to drug discontinuation. We aimed to identify pwCF, intolerant to CFTR modulator therapy due to suspected immunogenic adverse events (iAE).

Methods: This survey assessed the types of reaction (e.g. rash, liver injury, drug fever) including reactions after re-exposure and was completed by ECFS CTN Centers.

Results: Response rate to the survey was 74 %. 89 CF centers treating approximately 12000 to 17500 pwCF in 28 countries participated and 75 (84 %) CF centers reported discontinuation of CFTR modulator therapy. 37 (41.1 %) of CF centers reported iAE affecting 200 (1.1 - 1.7 %) pwCF. Detailed information about iAEs was provided for 41 of 200 (20.5 % of affected) pwCF. Of the iAEs reported in detail 33/41 (80.5 %) were associated with elexacaftor/tezacaftor/ivacaftor modulator therapy, 6 (14.6 %) with lumacaftor/ivacaftor and 2 (4.9 %) with tezacaftor/ivacaftor. 72 % of pwCF with iAE were re-exposed to CFTR modulator therapy. 32 % of re-exposed pwCF reported a second iAE. Rash and elevated liver enzymes were most frequently reported iAEs.

Conclusions: iAE were mostly transient. Drug allergy to CFTR modulator therapy was rare, but highly relevant for individual pwCF.

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来源期刊
Journal of Cystic Fibrosis
Journal of Cystic Fibrosis 医学-呼吸系统
CiteScore
10.10
自引率
13.50%
发文量
1361
审稿时长
50 days
期刊介绍: The Journal of Cystic Fibrosis is the official journal of the European Cystic Fibrosis Society. The journal is devoted to promoting the research and treatment of cystic fibrosis. To this end the journal publishes original scientific articles, editorials, case reports, short communications and other information relevant to cystic fibrosis. The journal also publishes news and articles concerning the activities and policies of the ECFS as well as those of other societies related the ECFS.
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