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

Abstract

Langerhans 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 BRAF^V600E 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).

Keywords:

• Langerhans cell histiocytosis
• vemurafenib
• infant
• treatment

Acknowledgement

The wish to acknowledge Dr.Zhenglian Ha for her collaboration in working with the pathology of this case, as well as the assistance provided by the pediatrics department at Southern Hospital.

Ethical approval

Informed written patient consent was taken from the patient.

Disclosure statement

No potential conflict of interest was reported by the author.

Immunohistochemistry(IHC) assay

Antibodies used for IHC are as follows: antibodies against CD1a(AQ medical, China, RTU-CD1a-235-QH, clone:MTB1, RTU), CD68(AQ medical, China, NCL-CD68, clone:KP1, 1:400), Langerin(Gene Tech, China, GT209002, clone:12D6, RTU), LCA(LANOU medical, China, LMO-1042, clone:2B11, RTU), S100(AQ medical, China, NCL-L-S100, clone:polyclone, 1:500), and BRAF^V600E(Roche, CH, 07862270001, clone:VE1, RTU).

Data availability statement

The original contributions presented in this study are included in the article/supplementary material, further inquiries can be directed to the author.

Additional information

Funding

The author(s) reported there is no funding associated with the work featured in this article.