Trametinib in Adults with Neurofibromatosis Type 1-Related Symptomatic Plexiform Neurofibromas.

ABSTRACT:

Objective

Mitogen-activated protein kinase kinase inhibitors have shown promising results in treatment of plexiform neurofibromas in neurofibromatosis type 1 patients, but data in adults are limited. The aim of this phase 2 study was to investigate the efficacy and safety of trametinib in adults with neurofibromatosis type 1.

Methods

Thirty patients with a diagnosis of generalized or mosaic neurofibromatosis type 1 and a symptomatic inoperable plexiform neurofibroma were treated with 2mg trametinib per day. Primary outcome measures were partial response rate on tumor volume at cycle 12 and overall partial response rate. Partial response was defined as ≥20% decrease in tumor volume compared to baseline as measured with 3-dimensional volumetric analysis on magnetic resonance i

SUBMITTER: Noordhoek DC 

PROVIDER: S-EPMC12946598 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature