Vemurafenib in Langerhans cell histiocytosis : report of a pediatric patient and review of the literature

  • Selective BRAF inhibitors such as vemurafenib have become a treatment option in patients with Langerhans cell Histiocytosis (LCH). To date, only 14 patients receiving vemurafenib for LCH have been reported. Although vemurafenib can stabilize the clinical condition of these patients, it does not seem to cure the patients, and it is unknown, when and how to stop vemurafenib treatment. We present a girl with severe multisystem LCH who responded only to vemurafenib. After 8 months of treatment, vemurafenib was tapered and replaced by prednisone and vinblastine, a strategy which has not been described to date. Despite chemotherapy, early relapse occurred, but remission was achieved by re-institution of vemurafenib. Further investigation needs to address the optimal duration of vemurafenib therapy in LCH and whether and which chemotherapeutic regimen may prevent disease relapse after cessation of vemurafenib.

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Author:Anne Heisig, Jan Sörensen, Stefanie-Yvonne Zimmermann, Stefan Schöning, Dirk Schwabe, Hans-Michael KvasnickaORCiD, Raphaela Schwentner, Caroline Hutter, Thomas LehrnbecherORCiDGND
Pubmed Id:
Parent Title (English):OncoTarget
Publisher:Impact Journals LLC
Place of publication:[s. l.]
Document Type:Article
Year of Completion:2018
Date of first Publication:2018/04/24
Publishing Institution:Universitätsbibliothek Johann Christian Senckenberg
Release Date:2018/06/12
Tag:BRAF; LCH; Langerhans cell histiocytosis; child; vemurafenib
Page Number:5
First Page:22236
Last Page:22240
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Institutes:Medizin / Medizin
Dewey Decimal Classification:6 Technik, Medizin, angewandte Wissenschaften / 61 Medizin und Gesundheit / 610 Medizin und Gesundheit
Licence (German):License LogoCreative Commons - Namensnennung 3.0