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Article

Sustained Clinical Remission of BRAF V600E-Mutated Langerhans Cell Histiocytosis With Multiorgan Involvement in an Infant Treated With Dabrafenib.

Journal of pediatric hematology/oncology - 1 May 2025

Cuntz Franziska, Donadieu Jean, Holzhauer Susanne

Abstract excerpt

BACKGROUND: Above 50% of LCH cases show BRAF-mutations, which can be targeted by dabrafenib in refractory disease. OBSERVATIONS: Here, we report on a patient with neonatal multisystem, BRAF-mutated LCH refractory to conventional treatment with vinblastine and prednisolone. Duodenal involvement rendered oral nutrition impossible, and the patient was severely ill with pancytopenia, hepatic dysfunction, cholestasis,...

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Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

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Identifiers

PMID: 40042809
DOI: 10.1097/MPH.0000000000003014

Genes

BRAF

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