Effective BRAF inhibitor vemurafenib therapy in a 2-year-old patient with sequentially diagnosed Langerhans cell histiocytosis and Erdheim-Chester disease

Erdheim-Chester disease (ECD) is a rare histiocytic disorder, characterized by the xanthomatous infiltration of tissues by CD68-positive and CD1a-/CD100-negative foamy histiocytes. In childhood, ECD is exceptionally rare, and only a dozen cases have been published so far. The cooccurence of Langerha...

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Published inOncoTargets and therapy Vol. 10; pp. 521 - 526
Main Authors Váradi, Zsófia, Bánusz, Rita, Csomor, Judit, Kállay, Krisztián, Varga, Edit, Kertész, Gabriella, Csóka, Monika
Format Journal Article
LanguageEnglish
Published New Zealand Dove Medical Press Limited 01.01.2017
Taylor & Francis Ltd
Dove Medical Press
Subjects
Adults
Autoimmune diseases
Bone marrow
Case Report
Case studies
Chemotherapy
Development and progression
Drug dosages
Drug therapy
Histiocytosis
Langerhans cell histiocytosis
Mutation
NMR
Nuclear magnetic resonance
Pediatrics
Senescence
Stem cells
vemurafenib
treatment
Erdheim–Chester disease
Langerhans cell histiocytosis
pediatric histiocytic disorders
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Summary:Erdheim-Chester disease (ECD) is a rare histiocytic disorder, characterized by the xanthomatous infiltration of tissues by CD68-positive and CD1a-/CD100-negative foamy histiocytes. In childhood, ECD is exceptionally rare, and only a dozen cases have been published so far. The cooccurence of Langerhans cell histiocytosis (LCH) and ECD is even rarer. Here, we report a 2-year-old boy, the youngest patient in the literature so far, who was diagnosed with concomitant BRAF mutation-positive LCH and ECD. In his case, conventional LCH treatment proved to be ineffective, but he is the youngest patient who was successfully treated with the BRAF inhibitor vemurafenib.
ISSN:1178-6930
1178-6930
DOI:10.2147/OTT.S121615