Selumetinib Active in Children With Neurofibromatosis Type 1

Share this content:
Selumetinib Active in Children With Neurofibromatosis Type 1
Selumetinib Active in Children With Neurofibromatosis Type 1

THURSDAY, Dec. 29, 2016 (HealthDay News) -- The oral selective inhibitor of MAPK kinase 1 and 2, selumetinib, is active in children with neurofibromatosis type 1 and inoperable plexiform neurofibromas, according to a study published in the Dec. 29 issue of the New England Journal of Medicine.

Eva Dombi, M.D., from the Center for Cancer Research in Bethesda, Md., and colleagues conducted a phase 1 trial of selumetinib in children with neurofibromatosis type 1 and inoperable plexiform neurofibromas. Selumetinib was administered twice daily at a dose of 20 to 30 mg/m² of body-surface area. Selumetinib was also tested using a mouse model of neurofibromatosis type 1-related neurofibroma.

Twenty-four children (median tumor volume, 1,205 ml) received selumetinib. The researchers found that children were able to receive selumetinib on a long-term basis, with a median of 30 cycles (range, six to 56 cycles). A dose of 25 mg/m² was the maximum tolerated. Acneiform rash, gastrointestinal effects, and asymptomatic creatine kinase elevation were the most common toxic effects associated with selumetinib. Seventeen of the 24 children (71 percent) had confirmed partial responses with selumetinib treatment; 67 percent of 18 mice had decreases from baseline in neurofibroma volume. There was no evidence of disease progression to date (tumor volume increase of ≥20 percent from baseline).

"Our early-phase data suggested that children with neurofibromatosis type 1 and inoperable plexiform neurofibromas benefited from long-term dose-adjusted treatment with selumetinib without having excess toxic effects," the authors write.

The trial was partially funded by AstraZeneca, which provided the selumetinib.

Full Text (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



Sign up for myCME e-newsletters




More in Home

Some California Mosquitoes Can Carry Zika Virus

Some California Mosquitoes Can Carry Zika Virus

Aedes aegypti mosquitoes found to transmit Zika virus

Periodontal Inflammation a Risk With Tongue Piercing

Periodontal Inflammation a Risk With Tongue Piercing

And, two young females with tongue piercings have deep lingual infrabony lesions, periodontitis

Risk of OD Highest for First Days of Opioid  Benzodiazepine Use

Risk of OD Highest for First Days of ...

Five-fold increased risk of opioid-related overdose during first 90 days of concurrent use

is free, fast, and customized just for you!




Already a member?

Sign In Now »