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Roger J. Packer, MD - Progressive Pathways: Recent Recommendations and Emerging Therapies for Treating NF1-Associated Tumors
Go online to PeerView.com/AGQ860 to view the activity, download slides and practice aids, and complete the post-test to earn credit. Neurofibromatosis type 1 (NF1) is an incurable genetic disorder that primarily gives rise to nervous system tumors but can also lead to multisystem involvement. Neurofibromas are a key feature of NF1, and approximately 50% of patients will develop plexiform neurofibromas (PNs), which are associated with substantial morbidity and carry the potential to transform into malignant peripheral nerve sheath tumors. Optimal care of children with NF1 and NF1-related plexiform tumors includes awareness of the disease biology and the most recent clinical data on innovative therapies for managing the disease. Recent therapeutic developments for NF1 have been tumor-directed, often leveraging agents developed for the treatment of a variety of cancers. Notably, inhibitors of the MAPK signaling pathway, such as MEK inhibitors, offer rational therapeutic options for patients with NF1. This web broadcast features an expert review of treatment recommendations, current management paradigms, and challenges in the care of NF1 and NF1-associated tumors in children, as well as insight into the expanding landscape of targeted therapies and guidance on effectively incorporating these strategies into patient care. Upon completion of this activity, participants should be better able to: Describe fundamental aspects of NF1 etiology, pathophysiology, and clinical presentation as they relate to pediatric patients, Summarize current recommendations for NF1 diagnosis and treatment of NF1-associated tumors in pediatric patients, Evaluate recent evidence regarding the role of emerging therapies in the treatment of NF1-associated tumors in pediatric patients, including agents that affect the RAS/MAPK pathway, Assess options for patient-centered treatment of PNs and other NF1-associated tumors in pediatric patients.