Cystic fibrosis

Dr Uta Hill reviews the pathophysiology and genetics of cystic fibrosis, then traces the development of CFTR modulators from ivacaftor to the triple combination elexacaftor/tezacaftor/ivacaftor, using trial data, registry outcomes and a case from Papworth to show their effect on lung function, exacerbations and survival. She covers side effects and drug interactions, and the challenges of an ageing population, including weight gain, CF-related diabetes, liver disease and cancer screening. She also discusses reproductive health, including the rise in pregnancies and the current evidence on modulator use in pregnancy and breastfeeding. It suits doctors outside respiratory medicine who may meet patients with CF on modulators.