Opinion
Video
Selumetinib as a Therapeutic Option for NF1-PN
Panelists discuss key findings supporting the use of MEK inhibitors, such as selumetinib, in treating NF1-associated plexiform neurofibromas, emphasizing its impact on clinical decision-making, particularly for pediatric patients with progressive, symptomatic tumors who are not surgical candidates, and review current guidelines recommending its use to control tumor growth and improve quality of life.
Key Findings Supporting MEK Inhibition in NF1
Preclinical studies have shown that NF1-associated tumors are driven by RAS/MEK/ERK pathway activation, due to the loss of neurofibromin. This leads to uncontrolled cell proliferation and tumor growth. MEK inhibitors, such as selumetinib, target this pathway, inhibiting tumor growth and improving symptoms in patients with symptomatic plexiform neurofibromas (NF1-PN).
Impact of MEK Inhibitor Approval on Clinical Decision-Making
The approval of selumetinib has significantly influenced treatment strategies, particularly in pediatric patients with progressive, symptomatic NF1-PN who are not candidates for surgery. This approval provides a first-line therapeutic option to control tumor growth and alleviate associated symptoms. Clinicians are now more likely to initiate MEK inhibitors early to prevent further tumor progression and improve quality of life.
Guideline Recommendations for Selumetinib
Current guidelines recommend selumetinib for pediatric patients (2 years and older) with progressive NF1-PN that causes significant morbidity, particularly when tumors are inoperable or have grown to a size where surgical intervention is not feasible. The recommended dose is 25 mg/m² twice daily and treatment should be monitored for adverse effects such as skin rash, edema, and gastrointestinal symptoms.
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