NEUROFIBROMATOSIS THERAPEUTICS PROGRAM: DEVELOPMENT OF A PROGRAM

Abstract Neurofibromatosis (NF) therapeutics is a vital field in the care of children with NF. Recent developments in the treatment of plexiform neurofibromas (PN) have increased the numbers of patients seen for therapy. The Neurofibromatosis Therapeutics Program (NTP) provides high quality care to patients receiving therapy for brain tumors and PNs, as well as tumors related to NF2. The program at Children’s Hospital Colorado (CHCO) includes a physician, nurse practitioner, and nurse care coordinator. The team collaborates with other disciplines in the care of the NF patient with plexiform neurofibromas and/or CNS tumors. As the program grew, key players were identified in each subspecialty and educated about the NTP. We have ongoing regular communication with a large number of subspecialists regarding protocols, clinical care pathways, and mutual patients. In addition, an extensive tissue collection study of plexiform neurofibromas and brain tumors enhances NTPs devotion to both clinical and lab research. Weekly clinical care meetings ensure continuity in the care of the nearly 140 patients with NF1 and NF2 under our program. Monthly strategy and vision meetings focus on grant applications, education of primary care providers and subspecialists in our large catchment area, development of new clinical pathways, treatment roadmaps, and growth of our program. Over the last two years of being a formalized program, we have increased research on the epigenetics of plexiform neurofibromas, opened a Phase 2 clinical trial for a Mek inhibitor, and increased our patient volume. The Covid pandemic has increased our ability to manage treatment side effects virtually through telehealth and online patient portals. Future goals of the NTP include completion of a program website, quarterly patient and provider newsletters, educational offerings, collaboration with other centers on Mek inhibitor side effects, adolescent and young adult education on tumor risk, and transition to adult care.