Improved Quality of Life in Children With Cystic Fibrosis Who Received Transmembrane Conductance Regulator Modulators.

AIM: Children with cystic fibrosis (CF) face substantial daily treatment burdens and the effects of transmembrane conductance regulator modulators on these have not been sufficiently described. We evaluated changes in treatment burden after elexacaftor tezacaftor ivacaftor (ETI) was initiated.

METHODS

This prospective observational study comprised children aged six to 17 years from three Swedish paediatric CF centres. They were enrolled from 7 December 2022 to 30 June 2023, before ETI was initiated. Their health-related quality of life, treatment burden, and adherence were assessed at baseline and at six and 12 months.

RESULTS

We studied 62 children who initiated ETI and were followed up to 12 months. The mean standard deviation (SD) treatment burden score improved by 4.9 points (95% confidence interval (CI) -0.7 to 10.6, p = 0.087), from 67.6 (±20.6) at baseline to 72.5 (±17.6) at 12 months. The mean daily time spent on inhalation therapy and airway clearance decreased from 91.9 (±37.3) to 70.4 (±23.6) minutes (p < 0.001). Mean inhalation time per session fell by 7 min (95% CI -10.5 to -3.3, p < 0.001) and airway clearance time by 4 min (95% CI -6.7 to -1.5, p = 0.003).

CONCLUSION

Treatment burden, particularly daily inhalation and airway clearance time, decreased after ETI initiation.