Andrea M Gross, Pamela L Wolters, Eva Dombi, Andrea Baldwin, Patricia Whitcomb, Michael J Fisher, Brian Weiss, AeRang Kim, Miriam Bornhorst, Amish C Shah, Staci Martin, Marie C Roderick, Dominique C Pichard, Amanda Carbonell, Scott M Paul, Janet Therrien, Oxana Kapustina, Kara Heisey, D Wade Clapp, Chi Zhang, Cody J Peer, William D Figg, Malcolm Smith, John Glod, Jaishri O Blakeley, Seth M Steinberg, David J Venzon, L Austin Doyle, Brigitte C Widemann
BACKGROUND: No approved therapies exist for inoperable plexiform neurofibromas in patients with neurofibromatosis type 1. METHODS: We conducted an open-label, phase 2 trial of selumetinib to determine the objective response rate among patients with plexiform neurofibromas and to assess clinical benefit. Children with neurofibromatosis type 1 and symptomatic inoperable plexiform neurofibromas received oral selumetinib twice daily at a dose of 25 mg per square meter of body-surface area on a continuous dosing schedule (28-day cycles)...
April 9, 2020: New England Journal of Medicine