Selumetinib in adults with NF1 and inoperable plexiform neurofibroma: a phase 2 trial.

ABSTRACT: The MEK inhibitor selumetinib induces objective responses and provides clinical benefit in children with neurofibromatosis type 1 (NF1) and inoperable plexiform neurofibromas (PNs). To evaluate whether similar outcomes were possible in adult patients, in whom PN growth is generally slower than in pediatric patients, we conducted an open-label phase 2 study of selumetinib in adults with NF1 PNs. The study was designed to evaluate objective response rate (primary objective), tumor volumetric responses, patient-reported outcomes and pharmacodynamic effects in PN biopsies. The objective response rate was 63.6% (21/33 participants). Median maximal PN volume decrease was 23.6% (range: -48.1% to 5.5%). No disease progression relative to baseline PN volumes occurred before data cutoff, with a medi

SUBMITTER: Gross AM 

PROVIDER: S-EPMC12423960 | biostudies-literature | 2025 Jan

REPOSITORIES: biostudies-literature