Longitudinal disease burden and drug safety profiles of CFTR modulator therapy in cystic fibrosis: a real-world analysis from the NIH All of Us Research Program (NACFC 2026) - Sep 9, 2026 - Abstract #646; " The longitudinal cohort included 85 patients (mean age, 35.2 years [SD 13.8]; 52.9% female; 78.8% White; 38.8% on elexacaftor/tezacaftor/ivacaftor; mean follow-up, 4.4 years [SD 2.3])... CFTR modulator therapy was associated with sustained disease burden reduction and decreased respiratory and GI manifestations, hospitalizations, and IV antibiotic use over up to 8 years. No increased adult cataract risk was observed. New-onset psychiatric diagnoses in 15.3% of previously unaffected patients and gradual return of hepatic events suggest continued pharmacovigilance is warranted."
Clinical • Real-world • Real-world evidence • Cataract • Cystic Fibrosis • Genetic Disorders • Immunology • Infectious Disease • Novel Coronavirus Disease • Ophthalmology • Respiratory Diseases
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C. Zeng 1; K. Raraigh 2; G. Cutting 2; J. Denny 3
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