| Affiliation: | 1. Pulmonary Department and Adult CF Centre, Cochin Hospital, AP-HP, Paris, France;2. Université Paris Descartes, Sorbonne Paris Cité, Paris, France;3. Pulmonary Department, Arnaud de Villeneuve Hospital, Montpellier, France;4. Pulmonary Department, University Hospital Grenoble Alpes, Grenoble, France;5. Adult CF and Lung Transplantation Centre, Foch Hospital, Suresnes, France;6. Pulmonary Department and Adult CF Centre, Lille University Hospital, Lille, France;7. Pulmonary Department, Centre Intercommunal de Créteil, Créteil, France;8. Pulmonary Department, Charles Nicolle Hospital, Rouen University Hospital, Rouen, France;9. CF centre, Centre héliomarin de Perharidy, Roscoff, France;10. Pulmonary Department and adult CF centre, Bordeaux University Hospital, Bordeaux, France;11. FHU OncoAge, Côte d''Azur University, Nice, France;12. Adult CF Centre, CHU Nord, AP-HM, Marseille, France |
| Abstract: | ObjectiveTo investigate the short-term adverse events and effectiveness of lumacaftor/ivacaftor combination treatment in adults with cystic fibrosis (CF) and severe lung disease in a real life setting.MethodsA multicentre observational study investigated adverse events, treatment discontinuation, FEV1 and body mass index (BMI) one month and three months after lumacaftor/ivacaftor initiation in adults with CF and FEV1 below 40% predicted.ResultsRespiratory adverse events (AEs) were reported by 27 of 53 subjects (51%) and 16 (30%) discontinued treatment. The mean absolute change in FEV1 was + 2.06% after one month of treatment (P = 0.086) and + 3.19% after 3 months (P = 0.009). BMI was unchanged.ConclusionsTreatment with lumacaftor/ivacaftor in patients with CF and severe lung disease was discontinued more frequently than reported in clinical trials, due to respiratory AEs. Nevertheless, the patients who continued treatment had an increase in lung function comparable to what was observed in pivotal trials. |
