Drug Combo Tolerated in Younger Children With Cystic Fibrosis

Share this content:
Drug Combo Tolerated in Younger Children With Cystic Fibrosis
Drug Combo Tolerated in Younger Children With Cystic Fibrosis

THURSDAY, Feb. 7, 2019 (HealthDay News) -- Lumacaftor/ivacaftor treatment is generally safe and well tolerated for children aged 2 to 5 years with cystic fibrosis (CF) homozygous for the F508del-CFTR mutation, according to a study published online Jan. 24 in The Lancet Respiratory Medicine.

John J. McNamara, M.D., from Children's Minnesota in Minneapolis, and colleagues enrolled children aged 2 to 5 years weighing ≥8 kg at enrollment who had a confirmed diagnosis of CF homozygous for the F508del-CFTR mutation in an open-label phase 3 study. Patients received lumacaftor 100 mg/ivacaftor 125 mg (body weight <14 kg) or lumacaftor 150 mg/ivacaftor 188 mg (body weight ≥14 kg) orally for 15 days in part A (12 children) and for 24 weeks in part B (60 children).

The researchers found that safety and pharmacokinetics were consistent with the known safety profile. In part B, 98.3 percent of children had one or more treatment-emergent adverse events (AEs); most were of mild-to-moderate severity. The most common AEs were cough, vomiting, pyrexia, and rhinorrhea. Four children experienced serious AEs: infective pulmonary exacerbation of CF, viral gastroenteritis, and constipation. Because of elevated transaminases, 5 percent of the children discontinued treatment. At week 24, there was a 31.7 mmol/L decrease in mean sweat chloride concentrations, improvement in biomarkers of pancreatic function, and increases in growth parameters.

"These data highlight the potential of early intervention with lumacaftor/ivacaftor to improve clinically relevant outcomes," the authors write.

Several authors are employees of and disclosed financial ties to Vertex Pharmaceuticals, which manufactures lumacaftor/ivacaftor and funded the study.

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

More in Home

Operative Treatment Seems Most Effective for Adult Lumbar Scoliosis

Operative Treatment Seems Most Effective for Adult Lumbar ...

Nonoperative tx advised if patient satisfied with spine-related health; surgery may improve outcomes

Intervention Can Reduce Orders for Inpatient Urine Cultures

Intervention Can Reduce Orders for Inpatient Urine Cultures

Intervention includes notifications to providers, changes to order sets, new urine culture reflex tests

Odds of Receiving Tx for CAD Up With Medicare Advantage

Odds of Receiving Tx for CAD Up With ...

However, no significant difference seen in intermediate outcomes for those enrolled in MA, FFS Medicare

is free, fast, and customized just for you!




Already a member?

Sign In Now »