Cystic Fibrosis Therapies Reduce Pediatric Lung Transplant Needs Advances in cystic fibrosis (CF) treatment are significantly decreasing the number of children requiring lung transplants, according to recent medical findings. Historically, children with CF-related end-stage lung disease accounted for up to half of pediatric lung transplants in the United States and about two-thirds in Europe. However, the emergence of highly effective CFTR modulator therapies has altered this landscape. CFTR modulators target the underlying defect in the cystic fibrosis transmembrane conductance regulator protein, improving lung function and slowing disease progression. These therapies have demonstrated substantial clinical benefits, including reduced pulmonary exacerbations and improved nutritional status. Many children with CF now experience stabilized respiratory health, delaying or eliminating the need for transplantation. Data from pediatric transplant centers indicate a notable decline in lung transplants performed for CF indications, particularly during and after the COVID-19 pandemic period. Whereas CF remains a leading cause of lung transplantation in children under 18, the overall volume has decreased due to earlier and more effective intervention with modulator drugs. Despite this progress, lung transplantation remains a critical option for children with advanced lung disease who do not respond adequately to medical therapies or who develop complications unrelated to CFTR function. Ongoing research continues to evaluate long-term outcomes and quality of life following transplantation in the CF population. Healthcare providers emphasize that access to CFTR modulators varies globally, and disparities in treatment availability may influence transplant rates across regions. Continued efforts to expand access to these life-changing therapies are essential to further reduce the burden of end-stage lung disease in children with cystic fibrosis.
42