Case Reports:

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

Anne Heisig, Jan Sörensen, Stefanie-Yvonne Zimmermann, Stefan Schöning, Dirk Schwabe, Hans-Michael Kvasnicka, Raphaela Schwentner, Caroline Hutter and Thomas Lehrnbecher _

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Oncotarget. 2018; 9:22236-22240. https://doi.org/10.18632/oncotarget.25277

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Anne Heisig1, Jan Sörensen2, Stefanie-Yvonne Zimmermann1, Stefan Schöning1, Dirk Schwabe1, Hans-Michael Kvasnicka3, Raphaela Schwentner4, Caroline Hutter4 and Thomas Lehrnbecher1

1Division of Pediatric Hematology and Oncology, Hospital for Children and Adolescents, Johann Wolfgang Goethe-University, Frankfurt, Germany

2Division of Stem Cell Transplantation and Immunology, Hospital for Children and Adolescents, Johann Wolfgang Goethe-University, Frankfurt, Germany

3Institute of Pathology, Johann Wolfgang Goethe-University, Frankfurt, Germany

4St. Anna Kinderspital, Vienna, Austria

Correspondence to:

Thomas Lehrnbecher, email: [email protected]

Keywords: Langerhans cell histiocytosis; LCH; child; vemurafenib; BRAF

Received: March 12, 2018    Accepted: April 06, 2018    Published: April 24, 2018


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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