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Guideline Recommendations for Newer NF1-PN Therapies

Opinion
Video

Panelists discuss current guidelines recommending mirdametinib for pediatric patients (2 years and older) with progressive, symptomatic NF1-associated plexiform neurofibromas that are inoperable or difficult to manage surgically, and emphasize the importance of early initiation, regular monitoring, and a multidisciplinary approach to optimize treatment outcomes.

Summary for Physicians

Current Guideline Recommendations for Mirdametinib
Current guidelines recommend mirdametinib for pediatric patients (2 years and older) with progressive, symptomatic neurofibromatosis type 1–associated plexiform neurofibromas (NF1-PN) that causes significant morbidity and is inoperable or difficult to manage surgically. It is considered for patients with tumor growth that impacts function or causes pain, and when other treatment options have been ineffective or are not feasible.

Incorporating Guidelines Into Clinical Practice

In clinical practice, these guidelines should guide decisions on early initiation of mirdametinib for progressive tumors that meet the criteria. Regular monitoring for adverse effects and tumor response is essential to assess treatment efficacy and adjust as needed. Integrating multidisciplinary care teams, including oncologists, pain specialists, and genetic counselors, is crucial for optimizing patient outcomes.

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