Bruce R. Korf, MD, PhD - Targeting the RAS/MAPK Pathway in Neurofibromatosis Type 1 and Plexiform Neurofibromas: Improving Patient Outcomes With an Innovative Therapeutic Approach

01.08.2021 - By PeerView Family Medicine & General Practice CME/CNE/CPE Audio Podcast

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Go online to to view the activity, download slides and practice aids, and complete the post-test to earn credit. Neurofibromatosis type 1 (NF1) is a rare autosomal dominant disorder of the nervous system that is associated with significant morbidity, including cutaneous and plexiform neurofibromas, optic pathway gliomas, skin pigmentation, bone deformities, neurocognitive deficits, and an increased risk of several types of cancer. Currently there is only one MEK inhibitor, selumetinib, approved for pediatric patients who have this debilitating rare disease. This on-demand activity provides expert insights into the pathophysiology of NF1, its clinical presentation, and practical guidance for management. In addition, the mechanistic rationale and latest evidence supporting the use of currently approved and emerging targeted therapies for NF1 are presented. Upon completion of this activity, participants will be able to: Describe the genetic etiology, diverse clinical symptomatology, and diagnostic characteristics of neurofibromatosis type 1 (NF1), Review the current treatment landscape and unmet needs for patients with plexiform neurofibromas (PNs) and other NF1-associated tumors, Evaluate the rationale for MEK inhibitors and other novel targeted therapies in clinical development for the treatment of NF1-related benign and malignant tumors (eg, plexiform neurofibromas, optic pathway gliomas, malignant peripheral nerve sheath tumors), Summarize recent evidence on the benefits and risks of MEK inhibitors and other emerging targeted therapy options for the management of NF1-related plexiform neurofibromas and other tumors in pediatric and adult patients, Incorporate MEK inhibitors into treatment plans for patients with NF1-related plexiform neurofibromas and other tumors, based on the latest evidence, recommendations, and effective multidisciplinary collaboration and coordination of care.

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