HealthDay News — Ivacaftor is a potential new treatment to offer children aged 2 years and older with cystic fibrosis and a CFTR gating mutation, according to research published online January 20 in The Lancet Respiratory Medicine.
The clinical trial included 34 cystic fibrosis patients between the ages of 2 and 5. All of the children had a CFTR gating mutation on at least one allele. For 6 months, they took two daily doses – 50 mg for children who weighed less than 14kg (31 pounds) and 75 mg for those who weighed more.
The children showed improvement in several areas, including weight gain, pancreatic function, and sweat chloride levels. The drug was generally well tolerated by the children, the researchers found. The most common problems were coughing and vomiting. Five children had liver function abnormalities, leading one to stop treatment.
The study is “groundbreaking for cystic fibrosis care in children aged 2 to 5 years,” write the authors of an accompanying editorial. “Targeted treatment of this basic defect has potential for both prevention of damage and functional improvement of affected organs. Ivacaftor is safe, and results of secondary outcome measures suggest efficacy in this age group that is similar to that in older patients. Many unknowns remain, however, such as the earliest age of possible application, data for natural fluctuation of new outcome variables, and other points that have been reviewed previously.”
Funding for the study was provided by Vertex Pharmaceuticals, maker of ivacaftor.