Work

Effective vemurafenib monotherapy for refractory Langerhans cell histiocytosis with sustained results post-withdrawal for over two years: a case report

Year: 2025

Type: article

Abstract: 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: 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.

Source: Frontiers in Oncology

DOI: 10.3389/fonc.2025.1688802

Authors: Jiaxin Ren, Ling Gu, Xue Tang

Institutions: Sichuan University, West China Second University Hospital of Sichuan University

Language: English

Topic: Histiocytic Disorders and Treatments

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,801