Real-World Safety of CFTR Modulators in the Treatment of Cystic Fibrosis: A Systematic Review.
Real-World Safety of CFTR Modulators in the Treatment of Cystic Fibrosis: A Systematic Review.
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DOI：
10.3390/jcm10010023
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发表时间：
2020-12-23
影响因子：
3.9
通讯作者：
Quon BS
中科院分区：
文献类型：
--
作者：
Dagenais RVE;Su VCH;Quon BS
囊性纤维化跨膜传导调节因子（CFTR）调节剂治疗针对囊性纤维化（CF）的根本原因，并且通常耐受良好;然而，现实世界研究表明停药和不良事件（AE）的频率可能高于临床试验中观察到的频率。本系统性综述的目的是总结市场上可获得的CFTR调节剂报告的真实世界AE（即，依伐卡托（IVA）、鲁玛卡托/依伐卡托（LUM/IVA）、替扎卡托/依伐卡托（TEZ/IVA）和依他卡托/替扎卡托/依伐卡托（ELX/TEZ/IVA）），并确定CF医疗团队的药剂师可能有助于缓解和管理这些AE的方式。检索了MEDLINE、EMBASE、CINAHL和Web of Science Core Collection在线数据库，检索时间为2012年至2020年8月1日。观察性研究、病例系列和病例报告的完整手稿或会议摘要符合入选条件。包括完整的手稿和会议摘要，包括54项观察性研究，5个病例系列和9个病例报告。报告的AE类型通常与临床试验中观察到的一致。在真实世界研究中，LUM/IVA与更高频率的药物相关AE和停药相关。在所有4种CFTR调节剂中确定了精神健康和神经认知AE的信号。有必要在现实环境中采用系统方法监测使用CFTR调节剂的CF患者的AE，以帮助更好地了解潜在AE以及可能与某些AE风险较高相关的患者特征。药剂师在安全启动和监测CF患者接受CFTR调节剂治疗方面发挥着关键作用。
Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies target the underlying cause of cystic fibrosis (CF), and are generally well-tolerated; however, real-world studies indicate the frequency of discontinuation and adverse events (AEs) may be higher than what was observed in clinical trials. The objectives of this systematic review were to summarize real-world AEs reported for market-available CFTR modulators (i.e., ivacaftor (IVA), lumacaftor/ivacaftor (LUM/IVA), tezacaftor/ivacaftor (TEZ/IVA), and elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA)), and to identify ways in which the pharmacist on CF healthcare teams may contribute to mitigating and managing these AEs. The MEDLINE, EMBASE, CINAHL, and Web of Science Core Collection online databases were searched from 2012 to 1 Aug 2020. Full manuscripts or conference abstracts of observational studies, case series, and case reports were eligible for inclusion. The included full manuscripts and conference abstracts comprised of 54 observational studies, 5 case series, and 9 case reports. The types of AEs reported generally aligned with what have been observed in clinical trials. LUM/IVA was associated with a higher frequency of respiratory-related AE and discontinuation in real-world studies. A signal for mental health and neurocognitive AEs was identified with all 4 CFTR modulators. A systematic approach to monitoring for AEs in people with CF on CFTR modulators in the real-world setting is necessary to help better understand potential AEs, as well as patient characteristics that may be associated with higher risk of certain AEs. Pharmacists play a key role in the safe initiation and monitoring of people with CF on CFTR modulator therapies.
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影响因子：
8
作者：
Diab-Caceres, Layla;Maria Giron-Moreno, Rosa;Javier Gomez-de-Terreros-Caro, Francisco
通讯作者：
Javier Gomez-de-Terreros-Caro, Francisco
DOI：
10.1016/j.jcf.2017.07.006
发表时间：
2017-11-01
期刊：
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
影响因子：
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作者：
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通讯作者：
Debray, Dominique
DOI：
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发表时间：
2018-03-01
影响因子：
3.9
作者：
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通讯作者：
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影响因子：
5.2
作者：
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通讯作者：
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DOI：
10.1007/978-3-319-51334-8_3
发表时间：
2017-01-01
期刊：
GLOBAL HISTORIES OF BOOKS: METHODS AND PRACTICES
影响因子：
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作者：
Carter, David
通讯作者：
Carter, David