Pediatric NF1-PN Treatment Options and Differences Between Available MEK Inhibitors

n this segment, the physicians discuss how they introduce medical treatment options to families when a plexiform neurofibroma is first detected in a child with NF1. One physician explains that he begins the conversation early, emphasizing close monitoring because very young patients may experience more rapid tumor progression. He notes that discussing available therapies up front helps families avoid the fear that nothing can be done, especially given historical limitations in treatment. The conversation includes considerations for both early surgical intervention in small, discrete tumors and the role of long-term medical therapy when surgery is not feasible. The physicians then review the mechanisms of FDA-approved MEK inhibitors used to treat NF1-PN, including selumetinib and mirdametinib. They explain that loss of neurofibromin leads to unchecked RAS activity, driving tumor growth through the MEK pathway. MEK inhibitors disrupt this signaling to slow or halt tumor progression. Although the two agents vary slightly in mechanism, they perform similarly in clinical practice.