For a study, it was determined that in controlled clinical trials, the cystic fibrosis (CF) modulator medicine elexacaftor/tezacaftor/ivacaftor (ETI) was highly beneficial for persons with one F508del allele, which occurs in at least 85% of people with CF (PwCF). PROMISE was post-approval research that looked at the long-term effects of ETI in a more diverse US patient population over the course of 30 months, with analyses planned after 6 months. Prospective, observational study of 487 PwCF aged 12 years who were starting ETI for the first time and had one F508del allele. The evaluations took place before, during, and after ETI therapy for 1, 3, and 6 months. The outcomes were changes in ppFEV1, sweat chloride concentration, body mass index, and self-reported respiratory symptoms. The average age of the participants was 25.1 years. About 44.1% used tezacaftor/ivacaftor or lumacaftor/ivacaftor, while 6.7% used ivacaftor, indicating homozygosity for F508del and the G551D allele, respectively. ppFEV1 improved 9.76 percentage points (95% CI 8.76, 10.76) from baseline, the CFQ-R Respiratory Domain score increased 20.4 points (95% CI 18.3, 22.5), and sweat chloride reduced -41.7 mmol/L six months after starting ETI medication (95% CI 43.8, 39.6). BMI also rose dramatically.
Exposure to Changes was more pronounced in individuals to modulators, but they were significant in all groups, including those who ivacaftor at the start. In a varied population naive to modulator drug therapy, either existing two-drug combinations or ivacaftor alone, ETI by clinical prescription resulted in significant improvements in lung function, respiratory symptoms, and BMI. Sweat chloride concentrations were also significantly lower in each group, associated with improved ppFEV1 in the entire study population.
Reference:www.atsjournals.org/doi/abs/10.1164/rccm.202108-1986OC
