CO167 Evaluating Risk of Progression with Selumetinib in Pediatric Patients with Neurofibromatosis Type 1 and Plexiform Neurofibroma: Propensity Score Method (PSM) Analysis of the Sprint Trial Vs a Natural History (NH) Control Arm

Selumetinib is approved for children aged ≥2 (US) or ≥3 (EU) years with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas (NF1-PN) based on data from the single-arm SPRINT trial (NCT01362803) and age-matched patients in the NH trial (NCT00924196). This PSM analysis estimates progression risk with selumetinib in SPRINT versus NH (external control arm), accounting for residual differences in baseline characteristics.