Observation effect of Ivacaftor/lumacaftor on lung function and exercise tolerance in subjects with Cystic Fibrosis and severe lung disease, homozygous for phe508del

Treatment of people living with CF and 2 copies of the delta F 508 gene with Orkambi (lumacaftor/Ivacaftor) has been shown to improve outcomes in those with milder forms of lung disease. In those excluded from the trials with severe disease, this is not known. We carefully assessed the effect of treatment on 10 individuals with severe lung disease and CF. WE showed improvments in exercise tolerance (6 minute walk test) and some effects on lung function, though this took up to 24 weeks to be demonstrated.