Rationale for MEK Inhibitors and Surgery

Author(s):

Ryan Haumschild, PharmD, MS, MBA,Mina Lobbous, MD, MSPH

Panelists discuss how MEK inhibitors, such as trametinib and selumetinib, are emerging as first-line treatments for symptomatic NF1-associated plexiform neurofibromas, especially in progressive or inoperable cases, while emphasizing the importance of surgical resection, pain management, physical therapy, and psychosocial support for improving quality of life.

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

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EP: 8.Rationale for MEK Inhibitors and Surgery

Summary for Physicians

First-Line Therapies for Symptomatic Plexiform Neurofibromas (NF1-PN)
For symptomatic neurofibromatosis type 1–associated plexiform neurofibromas (NF1-PN), MEK inhibitors (eg, trametinib, selumetinib) are emerging as first-line treatments, particularly for patients with progressive or inoperable tumors. These targeted therapies inhibit the MAPK/ERK pathway, addressing the underlying molecular dysfunction in NF1. Surgical resection remains a cornerstone of treatment for accessible and resectable tumors, aimed at relieving symptoms like pain, disfigurement, and functional impairment.

Additional Considerations

Symptomatic management, including pain control (eg, NSAIDs, opioids), physical therapy, and psychosocial support, is also important for improving patient quality of life.