Selumetinib in pediatric patients with neurofibromatosis type 1 and plexiform neurofibroma: Propensity score analysis of SPRINT vs. natural history control arm.

ABSTRACT:

Background

Selumetinib is approved in children aged ≥ 2 years (USA) with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PN) based on data from SPRINT (NCT01362803). This analysis estimated PN progression risk with selumetinib in SPRINT versus age-matched patients from the National Cancer Institute NF1 Natural History (NH) study (NCT00924196), accounting for differences in baseline characteristics using propensity score (PS) methods.

Methods

Differences in baseline characteristics between the cohorts were assessed. PSs based on age, sex, race, weight, height, and PN (location, volume, and progression status) were used in 1:1 PS matching without replacement, stabilized inverse probability of treatment weighting (sIPTW), and 1:2 PS matching

SUBMITTER: Adeyemi A 

PROVIDER: S-EPMC12288023 | biostudies-literature | 2025 Jan-Dec

REPOSITORIES: biostudies-literature