What is your approach to systemic treatment of a rapidly progressing, symptomatic, unresectable MPNST arising from a plexiform neurofibroma in a patient with NF1?  

The activity of the MEK inhibitor is selective for the benign NF/PN. For the malignant transformation, with aggressive behavior, the SOC chemotherapy regimens are preferred. The SARC trial tested Dox/Ifex and Ifex/VP-16, and these remain the preferred front-line regimens.

MPNST is a different ballgame than plexiform neurofibroma. Unfortunately, it is not chemotherapy-sensitive. We currently do not have a good option for MPNST. Perhaps clinical trial enrollment works best. We also do NGS to look for targets, apart from NF1. MEK inhibitors have been tried in the past w...