Main Goals in the Management of NF1-PN
Panelists discuss the current management goals for NF1-associated plexiform neurofibromas, focusing on tumor control, symptom management, and prevention of malignant transformation, while highlighting the evolving future goals of targeted therapies, personalized treatment plans, and enhanced early detection to improve outcomes.
EP: 1.Genetic And Molecular Mechanisms of NF1
EP: 2.Diagnosis, Epidemiology, And Risk Factors Of NF1-PN
EP: 3.Hallmark Symptoms and Clinical Features of NF1-PN
EP: 4.Quality of Life in Patients With NF1-PN
EP: 5.Key Clinical Considerations in NF1-PN Management
EP: 6.Unmet Needs of Patients Living With NF1-PN
EP: 7.Main Goals in the Management of NF1-PN
EP: 8.Rationale for MEK Inhibitors and Surgery
Summary for Physicians
Current Management Goals for NF1-PN
The primary goals in managing neurofibromatosis type 1–associated plexiform neurofibromas (NF1-PN) are to control tumor growth, manage symptoms (pain, disfigurement, neurological impairment), and prevent malignant transformation. Treatment strategies focus on monitoring tumor progression, surgical resection when feasible, and symptomatic management (eg, pain relief, functional support).
Future Evolving Goals
As research advances, future management may focus on targeted therapies aimed at the molecular drivers of NF1-PN, such as RAS/MAPK pathway inhibitors or gene therapies. Additionally, there is potential for earlier intervention with personalized treatment plans based on genetic profiling. Enhanced early detection and preventive strategies for malignancy may also become key components of care.