Efficacy of Targeted CFTR Modulators on Pulmonary Function and Sweat Chloride Metrics in Pediatric Cystic Fibrosis Patients

International Journal of Medicine and Medical Sciences | Vol. 16, No. 8, August 2026 | pp. 73–80

DOI: 10.46882/2026/IJMMS/160073

Original Research Article

Efficacy of Targeted CFTR Modulators on Pulmonary Function and Sweat Chloride Metrics in Pediatric Cystic Fibrosis Patients

Sarah J. Corelli¹, Robert L. Sinclair², Fiona M. Gallagher³*

¹Department of Pediatrics, Hospital for Sick Children, University of Toronto, Toronto, ON, Canada.

²Department of Pulmonology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.

³Institute of Cellular Medicine, Great Ormond Street Hospital, University College London, London, UK.

Abstract:

Small-molecule cystic fibrosis transmembrane conductance regulator (CFTR) modulators have transformed the management of cystic fibrosis, but their continuous efficacy on early-stage pancreatic and pulmonary alterations requires structured pediatric evaluation. This prospective, multi-center cohort study evaluated the clinical efficacy and safety of elexacaftor-tezacaftor-ivacaftor therapy in children possessing at least one F508del allele mutation. We evaluated 120 pediatric patients (aged 6 to 11 years) across three international tertiary centers who completed 24 weeks of targeted triple-combination therapy. The primary endpoints were absolute changes in ppFEV1 (percent predicted forced expiratory volume in 1 second) and sweat chloride concentrations, alongside nutritional indices monitored via body mass index (BMI) Z-scores. Data synthesis showed that by week 24, ppFEV1 increased significantly by a mean of +11.4 ± 2.8 percentage points from baseline markers (p < 0.001). Concurrently, mean sweat chloride concentrations demonstrated a substantial decrease of -42.4 ± 5.1 mmol/L (from 98.2 ± 8.4 mmol/L to 55.8 ± 4.2 mmol/L, p < 0.001), reflecting restoration of systemic ion channel functionality. Nutritional recovery was evidenced by a significant mean increase in BMI Z-scores (+0.34 ± 0.12, p < 0.01). Reported adverse events were primarily mild, with transient elevations in liver transaminases (ALT/AST) observed in 6.7% of the cohort, which resolved without drug discontinuation. Targeted triple-combination CFTR modulators achieve profound, rapid clinical improvements in lung function, clear sweat electrolyte markers, and enhance nutritional velocity in pediatric patients with cystic fibrosis.

Keywords: Cystic Fibrosis, CFTR Modulators, ppFEV1, Sweat Chloride, Pediatric Pulmonology, Nutritional Recovery

Manuscript Timeline: Received: May 10, 2026; Revised: June 18, 2026; Accepted: July 11, 2026; Published: August 11, 2026.

Citation: Corelli SJ, Sinclair RL, Gallagher FM. Efficacy of Targeted CFTR Modulators on Pulmonary Function and Sweat Chloride Metrics in Pediatric Cystic Fibrosis Patients. International Journal of Medicine and Medical Sciences, 2026, 16(8): 73–80. DOI: 10.46882/2026/IJMMS/160073

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