Early CFTR modulation and clinical outcomes in preschool cystic fibrosis.

This commentary discusses Presti et al.'s multicenter prospective study evaluating lumacaftor/ivacaftor therapy in preschool children with cystic fibrosis homozygous for F508del. The findings provide important real-world evidence that early CFTR modulation improves biomarkers of CFTR function, pulmonary outcomes, and early lung physiology while maintaining an acceptable safety profile. These data support the broader concept that intervention during early childhood may offer an opportunity to modify disease progression before irreversible organ damage develops.