Real life experience of lumacaftor/ivacafotor in adult Phe508del homozygous cystic fibrosis patients.

Bonizzoni, Giacomo; Bellocchia, Michela; Bena, Cristina; Clivati, Elisa; Biglia, Carlotta; Demichelis, S.; Bandelli, Gianpiero; Grande, A.; F, Traversa; Albera, Carlo; Messore, Barbara

doi:10.1183/13993003.congress-2018.pa1322

Cited by 2 publications

(2 citation statements)

References 0 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The majority of AEs reported with LUM/IVA were respiratory-related. Chest tightness, dyspnea, increased sputum, and declines in ppFEV 1 were among the most common respiratory AEs and tended to occur within the first few days after initiation, even as quickly as 3–4 h after the first dose [ 25 , 26 , 27 , 31 , 32 , 33 , 34 , 35 , 37 , 38 , 41 , 42 , 43 , 44 , 45 , 60 , 61 , 66 , 67 , 68 , 69 , 70 , 71 , 72 , 73 , 74 , 75 , 76 , 77 , 78 , 83 , 84 , 85 , 90 ]. Bronchodilators were beneficial in mitigating symptoms of chest tightness, wheeze, and increased work of breathing in some individuals [ 31 , 41 , 66 , 67 , 75 ].…”

Section: Resultsmentioning

confidence: 99%

“…One study reported a ppFEV 1 drop 39% below baseline in an individual; the timing of repeat spirometry after LUM/IVA initiation was not specified [ 44 ]. Declines in ppFEV 1 were not always accompanied by respiratory symptoms, and bronchodilators did not consistently improve acute drops in ppFEV 1 [ 32 , 41 , 43 , 66 , 67 , 84 ]. ppFEV 1 could take upwards of three months after initiation to recover to baseline values [ 34 , 43 ].…”

Section: Resultsmentioning

confidence: 99%

See 1 more Smart Citation

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

Dagenais

Quon

2020

JCM

View full text Add to dashboard Cite

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.

show abstract