CASE REPORT article
Front. Oncol.
Sec. Pediatric Oncology
Volume 15 - 2025 | doi: 10.3389/fonc.2025.1688802
This article is part of the Research TopicCase Reports in Pediatric Oncology: 2025View all 5 articles
Effective Vemurafenib Monotherapy for Refractory Langerhans Cell Histiocytosis with Sustained Results Post-Withdrawal for Over Two Years: A Case Report
Provisionally accepted
- 1Sichuan University West China Second University Hospital, Chengdu, China
- 2Department of Pediatrics, West China Second University Hospital, Sichuan University, Chengdu, China
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Background: Langerhans cell histiocytosis (LCH) is a rare, inflammatory myeloid neoplasm. Mitogen-activated protein kinase (MAPK) inhibitors, such as vemurafenib, can quickly control active disease in patients resistant to vinblastine and prednisone, but recurrence often occurs within a year after stopping treatment. Case Presentation: We report the case of a 15-month-old girl with high-risk multisystem LCH and BRAFV600E mutation. The patient initially received treatment according to the LCH-III chemotherapy protocol but exhibited disease progression after two months of maintenance chemotherapy. Following initiation of vemurafenib monotherapy, the patient's condition improved rapidly. The duration of vemurafenib monotherapy was one year and nine months. The patient remained disease-free for over two years after vemurafenib withdrawal. Conclusion: This case highlights the potential of MAPK inhibitor monotherapy for pediatric refractory LCH.
Keywords: langerhans cell histiocytosis, mitogen-activated protein kinase, BRAF, Vemurafenib, chemotherapy
Received: 19 Aug 2025; Accepted: 24 Sep 2025.
Copyright: © 2025 Xin, GU and Xue. 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: Tang Xue, txily0912@126.com
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