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.

Metadaten
Verfasserangaben:	Anne Heisig, Jan Sörensen GND, Stefanie-Yvonne Zimmermann, Stefan Schöning, Dirk Schwabe, Hans Michael Kvasnicka ORCiD, Raphaela Schwentner, Caroline Hutter ORCiD, Thomas Lehrnbecher ORCiD GND
URN:	urn:nbn:de:hebis:30:3-465795
DOI:	https://doi.org/10.18632/oncotarget.25277
ISSN:	1949-2553
Pubmed-Id:	https://pubmed.ncbi.nlm.nih.gov/29774135
Titel des übergeordneten Werkes (Englisch):	OncoTarget
Verlag:	Impact Journals LLC
Verlagsort:	[s. l.]
Dokumentart:	Wissenschaftlicher Artikel
Sprache:	Englisch
Jahr der Fertigstellung:	2018
Datum der Erstveröffentlichung:	24.04.2018
Veröffentlichende Institution:	Universitätsbibliothek Johann Christian Senckenberg
Datum der Freischaltung:	12.06.2018
Freies Schlagwort / Tag:	BRAF; LCH; Langerhans cell histiocytosis; child; vemurafenib
Jahrgang:	9
Ausgabe / Heft:	31
Seitenzahl:	5
Erste Seite:	22236
Letzte Seite:	22240
Bemerkung:	All site content, except where otherwise noted, is licensed under a Creative Commons Attribution 3.0 License.
HeBIS-PPN:	43382686X
Institute:	Medizin / Medizin
DDC-Klassifikation:	6 Technik, Medizin, angewandte Wissenschaften / 61 Medizin und Gesundheit / 610 Medizin und Gesundheit
Sammlungen:	Universitätspublikationen
Open-Access-Publikationsfonds:	Medizin
Lizenz (Deutsch):	Creative Commons - Namensnennung 3.0

Open Access