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Over half of children with inoperable plexiform neurofibromas responded to the mitogen-activated protein kinase (MAPK) kinase (MEK) inhibitor selumetinib, with responses proving durable in most cases, suggested a phase II trial.
Single-agent selumetinib yielded objective responses in 35 of 50 patients, and 28 of the responses lasted for at least a year. The treatment also produced durable improvement in patient-reported pain intensity and other patient- and parent-reported functional and quality-of-life (QoL) outcomes, reported researchers in the New England Journal of Medicine.