Videos

In this segment, the physicians discuss how they introduce medical therapy for plexiform neurofibromas (PN) when treatment is first considered for a child with NF1. One physician explains that these conversations begin early—often at the moment of tumor detection—because young children may experience faster tumor growth and require close monitoring. He emphasizes how early discussion helps families understand that effective treatments now exist, alleviating the anxiety rooted in decades of limited options. The segment highlights the importance of new, child-friendly formulations such as the selumetinib SPRINKLE preparation, which allows dosing in infants and toddlers, and notes that mirdametinib was also developed with a soluble form suitable for very young patients. The physicians then shift to treatment expectations. They describe pain relief as the earliest and most meaningful sign of clinical improvement, while radiographic responses—when visible—typically emerge only after several months. Because confirmed tumor shrinkage can take a year or longer, families are counseled that therapy requires long-term commitment.

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.