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. 2024 Aug 14;33(173):240060.
doi: 10.1183/16000617.0060-2024. Print 2024 Jul.

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

Carina M E Hansen  1   2 , Anna J Breukelman  3 , Patricia M L A van den Bemt  4 , Annelies M Zwitserloot  5   6 , Liset van Dijk  3   7 , Job F M van Boven  4   2   6
Affiliations

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

Carina M E Hansen et al. Eur Respir Rev. .

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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Conflict of interest statement

Conflict of interest: C.M.E. Hansen received a travel grant for the ECFS in 2023 (money paid to institution). A.M. Zwitserloot reports grants from Vertex Pharmaceuticals (IIS-2018-107391) and participation in an advisory board meeting for Sanofi (money paid to institution). J.F.M. van Boven reports consulting and advisory grants from Vertex (money paid to institution). The other authors declare that they have no know competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

Figures

FIGURE 1
FIGURE 1
Study inclusion flowchart.
FIGURE 2
FIGURE 2
Adherence rates to cystic fibrosis transmembrane conductance regulator modulators. Rates are given as mean±sd medication possession ratio or proportion of days covered. #: electronic monitoring; : intervention group. Iva: ivacaftor; Lum: lumacaftor; Tez: tezacaftor; ETI: elexacaftor–tezacaftor–ivacaftor.
FIGURE 3
FIGURE 3
Adherence rates to cystic fibrosis transmembrane conductance regulator modulators: detailed overview of the a) 12- and b) 24-month follow-ups as shown in figure 2. Rates are given as mean±sd medication possession ratio or proportion of days covered. #: intervention group. Iva: ivacaftor; Lum: lumacaftor; Tez: tezacaftor; ETI: elexacaftor–tezacaftor–ivacaftor.
See this image and copyright information in PMC

References

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