University of Groningen, University Medical Center Groningen (UMCG), Department of Clinical Pharmacy and Pharmacology, Groningen, The Netherlands
Medication Adherence Expertise Center of the Northern Netherlands (MAECON), Groningen, The Netherlands.
Eur Respir Rev. 2024 Aug 14;33(173). doi: 10.1183/16000617.0060-2024. Print 2024 Jul.
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.
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.
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% electronic monitoring and 100% self-report. Age <18 years appeared to be associated with good adherence, as was a higher lung function.
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.
在过去的十年中，由于囊性纤维化（CF）跨膜电导调节剂（CFTR）调节剂的引入，CF 的治疗发生了根本性转变。充分的药物依从性是其有效性的前提，但人们对 CFTR 调节剂的依从性知之甚少。我们旨在评估 CF 患者 CFTR 调节剂的用药依从程度，并评估哪些特征与依从性相关。
按照 PRISMA 指南进行系统评价。研究需要报告 CFTR 调节剂的依从性。主要结局为：1）药物依从程度和 2）人口统计学和/或临床特征与依从性的关联。
共筛选出 4082 篇文章，对 21 篇全文进行了评估以确定其是否符合纳入标准。最终纳入了 7 项研究。大多数研究为回顾性研究，主要集中在 ivacaftor 或 lumacaftor-ivacaftor 的依从性上，只有一项研究关注 elexacaftor-tezacaftor-ivacaftor。大多数研究使用药房补充数据，通过比例用药天数（PDC）或用药持续时间（MPR）来确定依从性。有一项研究还使用了电子监测和患者自我报告的依从性。基于药房数据（PDC 或 MPR），依从性为 0.62-0.99，电子监测为 61%，自我报告为 100%。<18 岁的年龄似乎与良好的依从性相关，肺功能较高也是如此。
尽管使用了多种依从性方法，但根据药房补充数据或电子监测等客观指标，CFTR 调节剂的依从性并不理想。CFTR 调节剂的依从性测量和定义需要更加标准化，最好使用客观和详细的方法。
