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

Authors Váradi Z, Bánusz R, Csomor J, Kállay K, Varga E, Kertész G, Csóka M

Received 5 September 2016

Accepted for publication 4 November 2016

Published 24 January 2017 Volume 2017:10 Pages 521—526


Checked for plagiarism Yes

Review by Single-blind

Peer reviewers approved by Dr Manfred Beleut

Peer reviewer comments 3

Editor who approved publication: Dr Yao Dai

Zsófia Váradi,1 Rita Bánusz,1 Judit Csomor,2 Krisztián Kállay,3 Edit Varga,1 Gabriella Kertész,1 Monika Csóka1

1Second Department of Pediatrics, 2First Department of Pathology and Experimental Cancer Research, Semmelweis University, 3Pediatric Hematology and Stem Cell Transplantation Unit, United St István and St László Hospital, Budapest, Hungary

Abstract: 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.

Keywords: vemurafenib, treatment, Erdheim–Chester disease, Langerhans cell histiocytosis, pediatric histiocytic disorders

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