Longitudinal effects of ivacaftor and medicine possession ratio in people with theGly551Aspmutation: a 5-year study

Autor:	Peter J. Barry, Katie Stocking, R.M. Mitchell, Philip Foden, Andrew M. Jones
Rok vydání:	2021
Předmět:	Pulmonary and Respiratory Medicine medicine.medical_specialty business.industry Retrospective cohort study medicine.disease Cystic fibrosis Clinical trial Ivacaftor Lung disease Internal medicine Mutation (genetic algorithm) medicine In patient business Body mass index medicine.drug
Zdroj:	Thorax. 76:874-879
ISSN:	1468-3296 0040-6376
Popis:	IntroductionIvacaftor was the first therapy licensed to address the underlying defect in cystic fibrosis (CF). The improvements in lung function, nutritional status and pulmonary exacerbations in patients carrying aGly551Aspmutation were greater than previously seen in clinical trials for other therapies. Limited data are available regarding long-term outcomes and adherence to ivacaftor outside clinical trials.MethodsWe conducted a 5-year single-centre retrospective study of people with CF carrying theGly551Aspmutation who received ivacaftor. Clinical outcome data were extracted from medical notes and databases. Drug delivery data were used to assess medicine possession ratio (MPR).Results35 people were included. After commencing ivacaftor, FEV1improved by 9.6% (SE±1.59%) predicted by 6 months. Thereafter, FEV1declined, and at 5 years had returned to pre-ivacaftor baseline. Ivacaftor did not alter annual rate of FEV1decline (1.57% pre vs 1.82% post, p=0.74). Body mass index (BMI) increased for 4 years. There was a significant reduction in inpatient and total intravenous antibiotic days sustained over 5 years. MPR remained high but declined over time (−2.5±0.9% per year, p=0.007). FEV1was better maintained in patients with higher MPRs.ConclusionThe addition of ivacaftor provides acute benefits for people with theGly551Aspmutation and established lung disease. We report a sustained reduction in intravenous antibiotic use but following acute improvement in lung function, decline continues, and patients will continue to require medical observation and optimisation. Strategies to maintain high adherence should be a priority to prolong the benefits of ivacaftor.
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::5042bb2576d653d97f537bc634ff95a7 https://doi.org/10.1136/thoraxjnl-2020-215556 Zobrazit plný text záznamu