"Neurosurgical Challenges in Neurofibromatosis Type 1: Novel Horizons in the Era of MEK Inhibition
This educational initiative is targeted to pediatric neurosurgeons, neurosurgeons, neuro-oncologists, neuroradiologists, medical oncologists, pediatric hem/onc nurses, and other members of the interprofessional, multidisciplinary neuro-oncology team who care for patients with neurofibromatosis type 1 (NF1). Designed and developed to provide an exhaustive overview of novel and emerging data, as well as establish a foundational context of NF1 disease state complexity, this activity will begin by describing the genetic etiologies and multi-system pathophysiologies that have historically made NF1-associated plexiform neurofibromas so difficult to treat. Attendees will then gain insights from expert faculty regarding the pivotal shortcomings of traditional treatment approaches, with an emphasis on the challenges faced by neurosurgeons and the new horizons that have accompanied the burgeoning emergence of novel pharmacologic therapies, most notably MEK inhibitors. Finally, top-level clinical experts will guide attendees through a case-based section that explores the vital role of the neurosurgeon within the evolving NF1 management paradigm, and provides activity participants an opportunity to design evidence-based treatment regimens that explore how safe and effective medical therapies can augment and optimize surgical strategies for symptomatic, inoperable NF1-related tumors."
Describe the genetic genesis and the complex, multi-system pathophysiology of NF1, with an emphasis on the clinical gravity and historical intractability of plexiform neurofibromas, especially in the pediatric patient population.
Review traditional modalities for treating plexiform neurofibromas in patients with NF1, focusing on surgical strategies, and highlight the crucial clinical chasms that remain.
Examine emerging pharmacologic therapies for managing symptomatic, inoperable plexiform neurofibromas, including an appraisal of completed and ongoing clinical trials and recent FDA approvals of MEK inhibitors.
Evaluate the role of the neurosurgeon within the interprofessional neuro-oncology team, and identify key ways that emerging medical therapies can augment and optimize surgical approaches in NF1.
Using a case-based format, design evidence-based management strategies for inoperable, clinically significant NF1-associated plexiform neurofibromas