August 2026

PULMONARY

LONG-TERM DATA SHOWS IVACAFTOR IMPROVES LUNG FUNCTION AND HEIGHT IN CYSTIC FIBROSIS

Cystic fibrosis (CF) has long been defined by two hallmark complications: progressive lung function decline and stunted growth, both driven by dysfunction in the CFTR protein. While the short-term benefits of CFTR modulators like ivacaftor are well established, a new retrospective cohort study published in the European Respiratory Journal and co-led by Pulmonologist Manu Jain, MD, set out to answer a more pressing question for long-term disease management: Does ivacaftor meaningfully change the growth and pulmonary trajectory of children and young adults with CF over time?

​What They Did
  • Retrospective cohort analysis using the Cystic Fibrosis Foundation Patient Registry, 2012–2020
  • 18,193 patients ages 6 to 20 included
  • 1,794 treated with ivacaftor monotherapy vs. 16,399 receiving no modulator therapy
  • Height and forced vital capacity (FVC) tracked longitudinally via mixed-effects models
  • Results stratified by age, sex, and CFTR variant class

What they found:
  • Ivacaftor-treated patients showed faster growth in both height and lung function
  • Treated patients reached higher final height and FVC than untreated peers
  • At age 20, treated males and females had a mean FVC advantage of 662 mL and 530 mL, respectively (p<0.0001)
  • Benefits were most pronounced in patients with severe CFTR variants
  • Lung function gains held across all ages, driven largely by height, but with an independent respiratory benefit as well

Clinical Impact
For pulmonologists and thoracic surgeons managing CF patients, these findings offer compelling evidence that early initiation of CFTR modulator therapy may shift a patient's growth and respiratory trajectory, with potential downstream effects on long-term morbidity and mortality.

As modulator therapies continue to expand treatment options, this study reinforces the clinical case for early, proactive intervention to optimize peak lung function and physical development in the CF population.
​Manu Jain, MD, Professor of Pulmonary and Critical Care

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