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Cystic fibrosis transmembrane conductance regulator (CFTR) modulators such as ivacaftor (IVA), elexacaftor (ELX), lumacaftor (LUM) and tezacaftor (TEZ) are recently approved novel therapies for cystic fibrosis (CF) patients who have at least one ‘F508 del’ mutation. This mutation occurs in the majority of cystic fibrosis patients. Clinical trials with ‘triple therapy’ have demonstrated significantly improved lung function, sweat chloride and body mass index, with reduced pulmonary exacerbations.1 2
Our patient is a 24-year-old woman with cystic fibrosis (CF) with homozygous F508 del who received ‘compassionate use’ ELX/TEZ/IVA. She had severe bronchiectasis (forced expiratory volume in one second, FEV1 19%) with chronic Pseudomonas aeruginosa infection and required domiciliary oxygen therapy (2 L/min). She had …
Contributors MKH drafted the case report. GW and OD were involved in patient care and edited the manuscript.
Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Competing interests None declared.
Provenance and peer review Not commissioned; internally peer reviewed.
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