Journal of Clinical Medicine
Plethysmography as a Complementary Tool for Monitoring Response to CFTR Modulator Therapy in Children with Cystic Fibrosis: A Real-World Study
By Magdalena Postek, Katarzyna Walicka‐Serzysko, Urszula Borawska-Kowalczyk and 5 others.
Abstract
Background
Sensitive tools for monitoring response to cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy remain needed in children with cystic fibrosis (CF). Plethysmography may provide complementary information to spirometry and multiple-breath nitrogen washout (MBNW).
Aim
To characterize longitudinal changes in plethysmographic parameters before and during CFTR modulator therapy in children and adolescents with CF, using spirometry and MBNW as complementary physiological assessments.
Methods
This retrospective observational study included 67 children with CF. Forty-nine patients received elexacaftor/tezacaftor/ivacaftor (ETI) and 18 received tezacaftor/ivacaftor (TI). Spirometry, plethysmography, and MBNW were performed at baseline and at follow-up, typically 12 months after treatment initiation, but no later than 24 months. Health-related quality of life was assessed using the Cystic Fibrosis Questionnaire–Revised (CFQ-R) in 42 ETI-treated patients with complete paired data. Pulmonary exacerbations requiring intravenous antibiotic treatment were also analyzed.