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Real-World Safety of CFTR Modulators in the Treatment of Cystic Fibrosis: A Systematic Review

Authors
  • Dagenais, Renée V. E.1, 2
  • Su, Victoria C.1, 2
  • Quon, Bradley S.1, 3, 4
  • 1 (B.S.Q.)
  • 2 Department of Pharmacy, St. Paul’s Hospital, Vancouver, BC V6Z 1Y6, Canada
  • 3 Department of Medicine, University of British Columbia, Vancouver, BC V6Z 1Y6, Canada
  • 4 Centre for Heart Lung Innovation, St. Paul’s Hospital, Vancouver, BC V6Z 1Y6, Canada
Type
Published Article
Journal
Journal of Clinical Medicine
Publisher
MDPI AG
Publication Date
Dec 23, 2020
Volume
10
Issue
1
Identifiers
DOI: 10.3390/jcm10010023
PMID: 33374882
PMCID: PMC7795777
Source
PubMed Central
Keywords
Disciplines
  • Review
License
Green

Abstract

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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