Selumetinib in Children with Inoperable Plexiform Neurofibromas.

ABSTRACT:

Background

No approved therapies exist for inoperable plexiform neurofibromas in patients with neurofibromatosis type 1.

Methods

We conducted an open-label, phase 2 trial of selumetinib to determine the objective response rate among patients with plexiform neurofibromas and to assess clinical benefit. Children with neurofibromatosis type 1 and symptomatic inoperable plexiform neurofibromas received oral selumetinib twice daily at a dose of 25 mg per square meter of body-surface area on a continuous dosing schedule (28-day cycles). Volumetric magnetic resonance imaging and clinical outcome assessments (pain, quality of life, disfigurement, and function) were performed at least every four cycles. Children rated tumor pain intensity on a scale from 0 (no pain) to 10 (worst pain

SUBMITTER: Gross AM 

PROVIDER: S-EPMC7305659 | biostudies-literature | 2020 Apr

REPOSITORIES: biostudies-literature