Successful treatment of Langerhans cell histiocytosis in an infant with vemurafenib: a case report and literature review

AbstractLangerhans cell histiocytosis (LCH) is a histiocytic neoplasm characterized by a mass of CD1a + CD207 + histiocytes, exhibiting a diverse range of clinical manifestations from a self-healing rash or single bone destruction to multi-organ disease with potentially fatal consequences. The ident...

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Main Author: Qingqing Li (Author)
Format: Book
Published: Taylor & Francis Group, 2023-12-01T00:00:00Z.
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Summary:AbstractLangerhans cell histiocytosis (LCH) is a histiocytic neoplasm characterized by a mass of CD1a + CD207 + histiocytes, exhibiting a diverse range of clinical manifestations from a self-healing rash or single bone destruction to multi-organ disease with potentially fatal consequences. The identification of MAPK signaling pathway activation, particularly BRAFV600E mutations, has propelled targeted therapy into the forefront of therapeutic research for LCH. Several studies have demonstrated that Vemurafenib, a BRAF inhibitor, exhibits superior clinical efficacy and a more favorable safety profile in LCH. Herein, in this case report, we present a good response to vemurafenib in an infant diagnosed with multisystem Langerhans cell histiocytosis (LCH).
Item Description:10.1080/09546634.2023.2279901
1471-1753
0954-6634