Discontinuation of supportive therapies in people with cystic fibrosis treated with elexacaftor/tezacaftor/ivacaftor : a patient-reported outcomes’ study

Abstract

Background With the introduction of elexacaftor/tezacaftor/ivacaftor (ETI), there has been significant impact on the health and quality of life of people with cystic fibrosis, raising questions about the need for ongoing supportive therapies. This study examines patient-reported outcomes related to ETI and its influence on supportive therapy adherence. Methods An anonymized cross-sectional survey was conducted among 1589 members of the German Cystic Fibrosis Association to assess treatment burden, and quality of life before and during ETI treatment, focusing on adherence and factors affecting the continuation of supportive therapies. Results Of the 406 respondents (effective response rate 25.5 %), 276 were receiving ETI. Analysis showed increased quality of life and decreased symptoms, with a majority reporting reduced or absent cough (93 %) and sputum (91 %) following ETI treatment. The desire to reduce therapy burden was high, with 80.4 % reporting a prior or present reduction of supportive therapies, particularly of mucoactive inhalations and antibiotics. Age was a significant factor in therapy adherence, with higher adherence in adolescents than adults. Improved quality of life and better lung function correlated with greater adherence to antibiotic inhalations, while better lung function increased the likelihood of reducing mucoactive therapies. Conclusions Many people with CF are reducing supportive therapies despite the absence of clear guidelines. Decision-making is influenced by factors such as the desire to lessen therapy burden, reduced symptom severity, and maximizing therapeutic effects. The study highlights the need for comprehensive recommendations and calls for further research to understand the factors involved in therapy adherence and reduction.