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

Oncotarget
Anne HeisigThomas Lehrnbecher

Abstract

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.

References

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Sep 25, 2004·Pediatric Blood & Cancer·Jean DonadieuC Thomas
Jun 4, 2010·Blood·Gayane Badalian-VeryBarrett J Rollins
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Jun 19, 2015·Journal of the National Comprehensive Cancer Network : JNCCN·Letizia GandolfiPier Luigi Zinzani
Jul 17, 2015·JAMA Oncology·Sébastien HéritierJean Donadieu
Jun 18, 2016·Pediatric Blood & Cancer·R Maarten EgelerUNKNOWN Steering Committee of the Nikolas Symposium
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Apr 27, 2017·British Journal of Haematology·Sébastien HéritierJean Donadieu
May 16, 2017·European Journal of Cancer : Official Journal for European Organization for Research and Treatment of Cancer (EORTC) [and] European Association for Cancer Research (EACR)·Christian U BlankMichael P Brown

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Citations

Sep 13, 2019·Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology·Jean DonadieuSébastien Héritier
Nov 24, 2019·International Journal of Hematology·Kazuko KudoUNKNOWN Histiocytosis study group of the Japanese Society of Pediatric Hematology/Oncology
Sep 25, 2020·Leukemia & Lymphoma·Arturo Bonometti, UNKNOWN for Associazione Italiana Ricerca Istiocitosi AIRI ONLUS
Oct 31, 2020·The Australasian Journal of Dermatology·Arturo BonomettiEmilio Berti
Nov 15, 2020·Journal of Cutaneous Pathology·Arturo BonomettiUNKNOWN for AIRI, Associazione Italiana Ricerca Istiocitosi ONLUS
Dec 15, 2020·Current Opinion in Oncology·Fanélie JouenneAbdellatif Tazi
Mar 18, 2021·Revista española de patología : publicación oficial de la Sociedad Española de Anatomía Patológica y de la Sociedad Española de Citología·Isidro MachadoAntonio Llombart-Bosch
Jul 10, 2019·Archives de pédiatrie : organe officiel de la Sociéte française de pédiatrie·S HéritierJ Donadieu
Aug 24, 2021·The Journal of Pediatric Pharmacology and Therapeutics : JPPT : the Official Journal of PPAG·Liesbeth VanheeswijckJaques van Heerden

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Methods Mentioned

BETA
biopsy
PCR

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