Ivacaftor Appears Efficacious, Safe for Younger CF Patients

This article originally appeared here.
Share this content:
Ivacaftor Appears Efficacious, Safe for Younger CF Patients
Ivacaftor Appears Efficacious, Safe for Younger CF Patients

MONDAY, Jan. 25, 2016 (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 Jan. 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 six months, they took two daily doses -- 50 mg for children who weighed less than 14 kg (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.

Abstract
Full Text (subscription or payment may be required)
Editorial (subscription or payment may be required)

Share this content:

is free, fast, and customized just for you!

Already a member?

Sign In Now »

Drug Lookup

Browse drugs by: BrandGenericDisease

Trending Activities

All Professions



Sign up for myCME e-newsletters


More in Home

CDC: No Change in Level of Uninsured in U.S. in 2017

CDC: No Change in Level of Uninsured in ...

Five percent of children uninsured in 2017, 41.3 and 55.0 percent had public and private coverage

Age-Related Racial Disparity Examined in Childhood Suicide

Age-Related Racial Disparity Examined in Childhood Suicide

Significantly higher incidence of suicide for black children aged 5 to 12; lower rate for black teens

Moral Distress for Docs Providing Emergency-Only Hemodialysis

Moral Distress for Docs Providing Emergency-Only Hemodialysis

Docs describe burnout and emotional exhaustion; moral distress at seeing suffering and injustice

is free, fast, and customized just for you!

Already a member?

Sign In Now »