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Updated: Jun 14, 2025

Author Spotlight: Genetically Engineered Mouse Models and Pathological Characterization of Neurofibromatosis Type 1 Associated Tumors
Published on: May 17, 2024
Successful utilization of topical trametinib for neurofibromatosis type I-associated plexiform neurofibroma
Leonid Melnikov1,2, Lars Brichta3, Nathan J Schloemer2
1Department of Hematology-Oncology, East Tennessee Children's Hospital, Knoxville, Tennessee, USA.
Abstract:
An 11-year-old female with neurofibromatosis type 1 (NF-1) and history of optic glioma presented with a progressive cutaneous plexiform neurofibroma of the breast. The lesion was treated with topical application of a mitogen-activated protein kinase inhibitor, trametinib, resulting in stable, non-progression cutaneous plexiform neurofibroma for greater than 2 years. This case demonstrates the potential application of topical trametinib for NF1-associated superficial cutaneous plexiform neurofibroma without the toxicities associated with systemic treatment.
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