New CF Drug Cuts Daily Treatment Time by 21 Minutes for Children
A Swedish study of 62 children found that a combination therapy reduced time spent on inhalation and airway clearance from 92 minutes daily to 70 minutes—a clinically significant drop that could improve medication adherence and quality of life. The finding signals potential cost savings for health systems through reduced treatment burden and hospitalizations.
Originaltitel: Improved Quality of Life in Children With Cystic Fibrosis Who Received Transmembrane Conductance Regulator Modulators
ABSTRACT 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.