Abstract

Historical treatment options and management strategies for neurofibromatosis type 1 (NF1) have been limited, with a relative paucity of clinical studies and the traditional paradigm focused on an ineffectual, and possibly even harmful, trimodal combination of radiation, surgery, and surveillance. Fortunately, an emerging preponderance of trial data supporting the use of targeted medical therapies, including MEK inhibitors and multi-kinase TKIs, is expanding the stale treatment paradigm and broadening horizons for patients. Given the profound novelty of this paradigmatic shift in NF1 management, it is imperative that timely, adaptive, and evidence-based educational initiatives be developed and delivered to the interdisciplinary neuro-oncology treatment team in an effort to bridge both learning and practice gaps. With an appreciation of the intrinsically multi-system nature of NF1, which necessitates a longitudinal treatment approach encompassing the totality of the multidisciplinary team, CEC Oncology designed and tailored targeted NF1 educational activities to improve confidence, advance knowledge, and promote enhanced utilization of targeted medical therapies among pediatric neuro-oncologists, neuro-oncologists, and neurosurgeons who manage patients with NF1. We conducted two independent satellite symposia, one at the 2020 Society for Neuro-Oncology (SNO) Annual Meeting and another at the 2020 AANS/CNS Section on Pediatric Neurological Surgery (PNSS), and achieved robust educational outcomes across Moore’s Levels 1–5. There were notable statistical advancements in clinician confidence, knowledge, and intention to employ targeted medical therapies for NF1-related symptomatic, inoperable plexiform neurofibromas from pre-activity to post-activity assessment, and many of these improvements were retained at 6-week follow-up analysis. In conclusion, our outcomes data evidence the critical need for responsive educational activities in the NF1 treatment space, especially as trial data continue to galvanize evolution of this novel pharmacologic armamentarium. Such activities possess the capacity to improve clinician confidence, advance knowledge, and promote utilization of targeted medical therapies in NF1, thereby optimizing patient outcomes.

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