Targeting the RAS/MAPK Pathway in Neurofibromatosis Type 1 and Plexiform Neurofibromas: Improving Patient Outcomes With an Innovative Therapeutic Approach
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.
Jointly provided by Medical Learning Institute, Inc. and PVI, PeerView Institute for Medical Education, this live web broadcast will provide 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 will be presented.
- Describe the genetic etiology, diverse clinical symptomatology, and diagnostic characteristics of neurofibromatosis type 1 (NF-1)
- Review the current treatment landscape and unmet needs for patients with plexiform neurofibromas (PNs) and other tumors
- Evaluate the rationale for MEK inhibitors and other novel targeted therapies in clinical development for the treatment of benign and malignant tumors (eg, plexiform neurofibromas, optic pathway gliomas, and 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 plexiform neurofibromas and other tumors in pediatric and adult patients
- Incorporate MEK inhibitors into treatment plans for patients with plexiform neurofibromas and other tumors, based on the latest evidence, recommendations, and effective multidisciplinary collaboration and coordination of care
Bruce R. Korf, MD PhD
UAB School of Medicine
University of Alabama at Birmingham
Michael J. Fisher, MD
The Children's Hospital of Philadelphia
University of Pennsylvania Perelman School of Medicine
Roger J. Packer, MD
Gilbert Family Neurofibromatosis Institute
Brain Tumor Institute
Children’s National Hospital
The George Washington University Medical Center
This CME/MOC/CNE/CPE activity is jointly provided by Medical Learning Institute, Inc. and PVI, PeerView Institute for Medical Education.
This activity is supported by an independent educational grant from AstraZeneca.
- Categories: Webinar