CASE REPORT article
Front. Oncol.
Sec. Neuro-Oncology and Neurosurgical Oncology
Successful response in advanced leptomeningeal disease from pleomorphic xanthoastrocytoma with BRAF / MEK inhibitors: A case report
Provisionally accepted
Maria A Jacome
Robert J Macaulay
Edwin PegueroSheethal CyriacSolmaz Sahebjam
Arnold Etame
Hsiang-Hsuan Michael YuYolanda Pina*- Moffitt Cancer Center, Tampa, United States
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Leptomeningeal disease (LMD) is a rare metastatic complication with a grim prognosis for most patients and limited treatment strategies. Therapy is adjusted to the primary tumor from which it arises. Targeted therapies and personalized medicine have become cornerstones in cancer treatment but its utility in LMD has been limited. In here we report a case of a female patient who developed LMD from a Pleomorphic Xanthoastrocytoma (PXA), BRAFV600-mutated, who has shown successful response to treatment with BRAF/MEKi (Encorafinib/Binimetinib) for over 3 years since initial LMD diagnosis. The effectiveness of therapy in this patient was initially observed as stable disease, with radiographic progression when BRAF/MEKi were withheld, and immediate tumor control achieved when reinstated. Despite being just one case, this hopefully could serve as proof-of-concept for use of targeted therapy for BRAF V600E-mutated tumors with LMD progression, sparing patients from alternative tumor control options such as radiation therapy.
Keywords: Leptomeningeal disease, pleomorphic xanthoastrocytoma, BRAF V600E mutation, BRAF inhibition, MEK inhibition, CNS tumor
Received: 20 Jul 2025; Accepted: 03 Nov 2025.
Copyright: © 2025 Jacome, Macaulay, Peguero, Cyriac, Sahebjam, Etame, Yu and Pina. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Yolanda Pina, yolanda.pina@moffitt.org
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