'Triple therapy (elexacaftor, tezacaftor, ivacaftor) skin rash in patients with cystic fibrosis

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 not yet been listed for lung transplantation, given concerns about low body weight. She had previously tolerated ‘dual’ cystic fibrosis transmembrane conductance regulator therapies, initially LUM/IVA (Orkambi™) and thereafter TEZ/IVA (Symkevi™). Other CF...
Source: Postgraduate Medical Journal - Category: General Medicine Authors: Tags: Adverse drug reactions Source Type: research