Life-Changing Cystic Fibrosis Treatment

Teenage patients suffering from cystic fibrosis (CF) now have access to a brand-new triple-combination therapy that gives them the chance to achieve near-normal life expectancy, possibly outliving by more than 45 years CF patients on best supportive care alone.

“These results contribute to accumulating evidence that early intervention in [people with CF] is associated with better outcomes later in life,” as the study states. “Because lung function has been identified as a key predictor of long-term survival, the unprecedented and significant improvements demonstrated in the clinical trial program […] suggest that [Kaftrio] treatment may provide a lifetime therapeutic benefit” for patients.

When patients between the ages of 12 and 17 who have the F508del mutation are administered elexacaftor/tezacaftor/ivacaftor – known as Kaftrio in the United Kingdom and Trikafta in the United States – their median estimated lifespan reaches 82.5 years, close to life expectancy for the general British population which is 83 years. Combining the three distinct medications improves lung function, enhances health-related quality of life, and lowers the frequency of pulmonary exacerbations. Indeed, Kaftrio helps people have 13 fewer pulmonary exacerbations over a lifetime than patients on best supportive care, reducing the annual rate of pulmonary exacerbations by almost 75%.