Patient perspectives following initiation of elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis and advanced lung disease

Backgound. - Elexacaftor-tezacaftor-ivacaftor partially restores cystic fibrosis transmembrane conductance regulator function, and has been shown to induce significant clinical improvement in patients withat least one Phe508del allele. Yet little data exist on patient perspectives following elexacaftor-tezacaftorivacaftor initiation. Methods. - A mixed methods study was conducted using an online 13-item questionnaire (including 9closed questions and 4 open questions), submitted from July 10thto August 21th2020 to French patientsaged 12 years and older with advanced CF who were treated with elexacaftor-tezacaftor-ivacaftor. Theirresponses were summarized as numbers (%), and free-text items were analysed using a grounded theoryapproach. Results. - Of 245 patients who started elexacaftor-tezacaftor-ivacaftor in France, 101 (41%) participated. Median [IQR] age was 35 [28-41] years and duration of elexacaftor-tezacaftor-ivacaftor treatment was4.3 [3.0-5.6] months. Patients generally reported a rapid impact on respiratory symptoms, sleep quality, general well-being and physical self-esteem, and a reduction in overall treatment burden. The majority ofpatients contrasted treatment burden, symptom severity, depression and a closed future marked by deathor transplantation before elexacaftor-tezacaftor-ivacaftor, to renewed and unexpected physical strength, leading to greater self-confidence, autonomy and long-term planning, after treatment initiation. A smallnumber of patients expressed concerns, mainly regarding changes in body representation and/or the fearof becoming dependent on the treatment. Conclusion. - After initiation of elexacaftor-tezacaftor-ivacaftor, CF patients with advanced diseasereported rapid and positive physical, psychological and social effects, which translated into improvedquality of life and the formulation of new life goals.