Elexacaftor/tezacaftor/ivacaftor and gastrointestinal outcomes in cystic fibrosis: Report of promise-GI

Background: Elexacaftor/tezacaftor/ivacaftor (ETI) improves pulmonary disease in people with cystic fi-brosis (PwCF), but its effect on gastrointestinal symptoms, which also affect quality of life, is not clear.Methods: PROMISE is a 56-center prospective, observational study of ETI in PwCF > 12 years and at least one F508del allele. Gastrointestinal symptoms, evaluated by validated questionnaires: Patient Assessment of Upper Gastrointestinal Disorders-Symptom (PAGI-SYM), Patient Assessment of Constipation-Symptom (PAC-SYM), Patient Assessment of Constipation-Quality of Life (PAC-QOL)), fecal calprotectin, steatocrit and elastase-1 were measured before and 6 months after ETI initiation. Mean difference and 95% confi-dence intervals were obtained from linear regression with adjustment for age and sex.Results: 438 participants fully completed at least 1 questionnaire. Mean (SD) for baseline PAGI-SYM, PAC-SYM, and PAC-QOL total scores were 0.56 (0.59), 0.47 (0.45), and 0.69 (0.53) out of maximum 5, 4, and 5, respectively (higher score indicates greater severity). Corresponding age-and sex-adjusted 6 months

Automated summary

The study aims to investigate the effect of ETI on gastrointestinal symptoms in patients with cystic fibrosis. The results show that there was an overall improvement in gastrointestinal symptoms after 6 months of ETI treatment.