Medication adherence to CFTR modulators in patients with cystic fibrosis: a systematic review.

IF 9 1区 医学 Q1 RESPIRATORY SYSTEM European Respiratory Review Pub Date : 2024-08-14 Print Date: 2024-07-01 DOI:10.1183/16000617.0060-2024
Carina M E Hansen, Anna J Breukelman, Patricia M L A van den Bemt, Annelies M Zwitserloot, Liset van Dijk, Job F M van Boven
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Abstract

Background: In the last decade, a fundamental shift in the treatment of cystic fibrosis (CF) took place due to the introduction of CF transmembrane conductance regulator (CFTR) modulators. Adequate medication adherence is a prerequisite for their effectiveness, but little is known about adherence to CFTR modulators. We aimed to assess the extent of medication adherence to CFTR modulators in patients with CF and assess which characteristics are associated with adherence.

Methods: A systematic review following PRISMA guidelines was performed. Studies needed to report adherence to CFTR modulators. Main outcomes were: 1) level of medication adherence and 2) associations of demographic and/or clinical characteristics with adherence.

Results: In total, 4082 articles were screened and 21 full-text papers were assessed for eligibility. Ultimately, seven studies were included. Most studies were retrospective and focused on adherence to ivacaftor or lumacaftor-ivacaftor with only one focusing on elexacaftor-tezacaftor-ivacaftor. The majority used pharmacy refill data with adherence determined with the proportion of days covered (PDC) or the medication possession ratio (MPR). One study additionally used electronic monitoring and patient self-reported adherence. Adherence was 0.62-0.99 based on pharmacy data (PDC or MPR), 61% via electronic monitoring and 100% via self-report. Age <18 years appeared to be associated with good adherence, as was a higher lung function.

Conclusions: Despite the wide variety of adherence methods used, adherence to CFTR modulators is suboptimal, based on objective measures such as pharmacy refill data or electronic monitoring. CFTR modulator adherence measurement and definitions requires more standardisation with a preference for objective and granular methods.

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囊性纤维化患者对 CFTR 调节剂的用药依从性:系统综述。
背景:近十年来,由于CF跨膜传导调节剂(CFTR)调节剂的问世,囊性纤维化(CF)的治疗发生了根本性转变。充分坚持用药是保证其疗效的先决条件,但人们对 CFTR 调节剂的坚持用药情况知之甚少。我们旨在评估CF患者对CFTR调节剂的用药依从性,并评估哪些特征与依从性相关:我们按照 PRISMA 指南进行了系统性回顾。研究需要报告CFTR调节剂的依从性。主要结果有1)用药依从性水平;2)人口统计学特征和/或临床特征与依从性的关系:共筛选出 4082 篇文章,并对 21 篇全文论文进行了资格评估。最终,7 项研究被纳入其中。大多数研究都是回顾性的,重点关注依维莫司或 lumacaftor-ivacaftor 的依从性,只有一项研究关注 elexacaftor-tezacaftor-ivacaftor 的依从性。大多数研究使用了药房续药数据,并通过覆盖天数比例(PDC)或药物持有率(MPR)来确定依从性。一项研究还使用了电子监测和患者自我报告的依从性。根据药房数据(PDC 或 MPR),依从性为 0.62-0.99,61% 通过电子监测,100% 通过自我报告。年龄 结论:尽管使用的依从性方法多种多样,但根据药房续药数据或电子监测等客观测量方法,CFTR调节剂的依从性并不理想。CFTR调节剂依从性的测量和定义需要更加标准化,更倾向于客观和细化的方法。
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来源期刊
European Respiratory Review
European Respiratory Review Medicine-Pulmonary and Respiratory Medicine
CiteScore
14.40
自引率
1.30%
发文量
91
审稿时长
24 weeks
期刊介绍: The European Respiratory Review (ERR) is an open-access journal published by the European Respiratory Society (ERS), serving as a vital resource for respiratory professionals by delivering updates on medicine, science, and surgery in the field. ERR features state-of-the-art review articles, editorials, correspondence, and summaries of recent research findings and studies covering a wide range of topics including COPD, asthma, pulmonary hypertension, interstitial lung disease, lung cancer, tuberculosis, and pulmonary infections. Articles are published continuously and compiled into quarterly issues within a single annual volume.
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