Real-world experience with selumetinib in children with neurofibromatosis type 1: a multicentric retrospective study.

ABSTRACT:

Purpose

Selumetinib is a MEK inhibitor indicated for pediatric patients with neurofibromatosis type 1 (NF1) and symptomatic inoperable plexiform neurofibromas (PNs).

Methods

This retrospective study collected data from 70 patients (aged 3 - 18 years) with NF1 and symptomatic inoperable PNs treated with selumetinib as part of compassionate use at 11 Italian centers between October 2018 and October 2024. Assessments included the clinical benefit rate (CBR) after 24 months and at the last observation. Major response (MR) was defined as a ≥ 50% reduction from baseline in tumor volume. Adverse events (AEs), patient-reported pain and quality of life (QoL), and Eastern Cooperative Oncology Group performance status (ECOG PS), were also evaluated.

Results

Of 45/70 patients wit

SUBMITTER: Santoro C 

PROVIDER: S-EPMC12511227 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature