Efficacy of MEK inhibition in a recurrent malignant peripheral nerve sheath tumor.
周围神经鞘恶性肿瘤
作者
Sumanth Nagabushan,Loretta Lau,Paulette Barahona,Marie Wong,Alexandra Sherstyuk,Glenn M. Marshall,Vanessa Tyrrell,Eva A. Wegner,Paul G Ekert,Mark J. Cowley,Chelsea Mayoh,Toby Trahair,Philip J. Crowe,Antoinette Anazodo,David S. Ziegler
The prognosis of recurrent malignant peripheral nerve sheath tumors (MPNST) is dismal, with surgical resection being the only definitive salvage therapy. Treatment with chemoradiation approaches has not significantly improved patient outcomes. Similarly, trials of therapies targeting MPNST genomic drivers have thus far been unsuccessful. Improved understanding of the molecular pathogenesis of MPNST indicates frequent activation of the mitogen-activated protein kinase (MAPK) cell signaling pathway. MEK inhibitors have shown activity in preclinical studies; however, their clinical efficacy has not been reported to date. We describe here a case of sustained complete response to MEK inhibition in an adolescent patient with a recurrent metastatic MPNST with multiple alterations in the MAPK pathway, guided by a precision oncology approach.