PMID- 33374882
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20220317
IS  - 2077-0383 (Print)
IS  - 2077-0383 (Electronic)
IS  - 2077-0383 (Linking)
VI  - 10
IP  - 1
DP  - 2020 Dec 23
TI  - Real-World Safety of CFTR Modulators in the Treatment of Cystic Fibrosis: A 
      Systematic Review.
LID - 10.3390/jcm10010023 [doi]
LID - 23
AB  - 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 Aug 1, 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.
FAU - Dagenais, Renee V E
AU  - Dagenais RVE
AD  - Adult Cystic Fibrosis Program, St. Paul's Hospital, Vancouver, BC V6Z 1Y6, 
      Canada.
FAU - Su, Victoria C H
AU  - Su VCH
AD  - Adult Cystic Fibrosis Program, St. Paul's Hospital, Vancouver, BC V6Z 1Y6, 
      Canada.
FAU - Quon, Bradley S
AU  - Quon BS
AD  - Adult Cystic Fibrosis Program, St. Paul's Hospital, Vancouver, BC V6Z 1Y6, 
      Canada.
AD  - Department of Medicine, University of British Columbia, Vancouver, BC V6Z 1Y6, 
      Canada.
AD  - Centre for Heart Lung Innovation, St. Paul's Hospital, Vancouver, BC V6Z 1Y6, 
      Canada.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20201223
PL  - Switzerland
TA  - J Clin Med
JT  - Journal of clinical medicine
JID - 101606588
EIN - J Clin Med. 2022 Jan 10;11(2):. PMID: 35054167
PMC - PMC7795777
OTO - NOTNLM
OT  - CFTR modulator
OT  - adverse events
OT  - elexacaftor
OT  - ivacaftor
OT  - lumacaftor
OT  - real-world
OT  - safety
OT  - tezacaftor
COIS- The authors declare no conflict of interest
EDAT- 2020/12/31 06:00
MHDA- 2020/12/31 06:01
PMCR- 2020/12/23
CRDT- 2020/12/30 01:02
PHST- 2020/12/03 00:00 [received]
PHST- 2020/12/14 00:00 [revised]
PHST- 2020/12/15 00:00 [accepted]
PHST- 2020/12/30 01:02 [entrez]
PHST- 2020/12/31 06:00 [pubmed]
PHST- 2020/12/31 06:01 [medline]
PHST- 2020/12/23 00:00 [pmc-release]
AID - jcm10010023 [pii]
AID - jcm-10-00023 [pii]
AID - 10.3390/jcm10010023 [doi]
PST - epublish
SO  - J Clin Med. 2020 Dec 23;10(1):23. doi: 10.3390/jcm10010023.