The MEK inhibitor selumetinib reduces spinal neurofibroma burden in patients with NF1 and plexiform neurofibromas.
Sadhana JacksonEva H BakerAndrea M GrossPatricia WhitcombAndrea BaldwinJoanne DerdakCecilia TiberyJennifer DesantoAmanda CarbonellKaleb YohayGeraldine O'SullivanAlice P ChenBrigitte C WidemannEva DombiPublished in: Neuro-oncology advances (2020)
This is the first study describing the effect of selumetinib on SNF. Of 24 patients, 18 exhibited some improvement of SNF burden on imaging. These findings suggest that selumetinib may prevent the worsening of cord compression, potentially reducing the need for surgical interventions in select patients or benefitting patients who do not have a surgical option. Prospective evaluation of the clinical benefit of selumetinib for SNF is warranted.