J Patient Rep Outcomes. 2025 May 6;9(1):48. doi: 10.1186/s41687-025-00879-0.
ABSTRACT
BACKGROUND: Elexacaftor/Tezacaftor/Ivacaftor (ETI) is a therapy approved for cystic fibrosis (CF) that has given improved clinical outcomes in patients carrying the F508del mutation. There are few published data regarding ETI’s effects on patients’ quality of life (QoL). This study aims to (fill the data gap in current literature by assessing) evaluate the long-term effects of ETI on QoL.
METHODOLOGY: A prospective observational study was conducted with thirty-seven severe patients that received ETI for compassionate use (group A), 184 received it for on-label use (group B). All carried one F508del mutation. Patients were assessed using the CFQ-R (Cystic Fibrosis Questionnaire-Revised). The evaluation time-points were pre-treatment (T0), and after 12 (T1) and 24 months (T2); group A was also assessed after 36 months (T3). Twenty-five patients completed 3 years of treatment and 65 patients completed 2 years of treatment, in groups A and B respectively.
RESULTS: At T1, median values for almost all areas of CFQ-R statistically significant increased in group A, particularly Physical Functioning (+ 25.0), Respiratory (+ 22.2) and Health Perception (+ 22.2).The Social Functioning area statistically significant increased at T2 (+ 5.6). At T3, these improvements remained stable. At T1, all areas of CFQ-R statistically significant increased in group B, particularly the Health Perception (+ 22,2) heading. At T2, these improvements remained stable. For both groups, the changes identified at the last follow-up showed no major differences by gender, age or genetic status.
CONCLUSIONS: Treatment with ETI significantly improved patients’ QoL in both groups at 12-24 months, these improvements remaining stable in patients tested at 36 months.
PMID:40327240 | DOI:10.1186/s41687-025-00879-0
