Physical well-being and burden of care in adults on modulator therapy: A mixed methods study of patient-reported experiences from the Well-ME survey

Background: Despite widespread availability of modulator therapies and improved lung function in many people with cystic fibrosis (CF), physical symptoms may remain burdensome for some people with CF (PwCF). This study identifies the impact of ivacaftor (IVA) and elexacaftor/tezacaftor/ivacaftor (ETI) on self-reported physical well-being and burden of care among adults with CF. Methods: We conducted a secondary analysis of data from the Well-ME Survey. Participants included adults with CF (age≥18) who reported taking IVA or ETI. We used a mixed methods approach to identify self-reported health status, physical well-being, and experience of CF care while taking IVA or ETI. Results: Among 414 eligible respondents, overall health status was reported very good/excellent by 59 % (n = 243), good by 26 % (n = 114), and poor/fair by 14 % (n = 57). While the majority of respondents experienced improvements in respiratory symptoms, PwCF reporting poor/fair health were less likely to report improvement in overall physical health, fatigue, and ability to exercise compared to those with good or very good/excellent health and less likely to report improvement in pain, sinus issues, and cough than those with very good/excellent health. PwCF reporting poor/fair health or good health were less likely to report improvements in gastrointestinal issues or experience reductions in CF medications or treatments, compared to those reporting very good/excellent health. Conclusions: Despite improvements in respiratory symptoms, some adults with CF taking IVA or ETI report their health is poor/fair. A better understanding of physical well-being and burden of care may help identify underrecognized comorbidities to improve care.