Article
Reproducible and sustained efficacy of targeted therapy with vemurafenib in patients with BRAF(V600E)-mutated Erdheim-Chester disease.
Journal of clinical oncology : official journal of the American Society of Clinical Oncology - 10 Feb 2015
Haroche Julien, Cohen-Aubart Fleur, Emile Jean-François, Maksud Philippe, Drier Aurélie, Tolédano Dan, Barete Stéphane, Charlotte Frédéric, Cluzel Philippe, Donadieu Jean, Benameur Neïla, Grenier Philippe A, Besnard Sophie, Ory Jean-Paul, Lifermann François, Idbaih Ahmed, Granel Brigitte, Graffin Bruno, Hervier Baptiste, Arnaud Laurent, Amoura Zahir
Abstract excerpt
PURPOSE: Histiocytoses are rare disorders with heterogeneous prognosis. BRAF(V600E) mutations have been observed in half of patients with Langerhans cell histiocytosis (LCH) and in 50% to 100% of patients with Erdheim-Chester disease (ECD) patients. We recently reported short-term efficacy of a BRAF inhibitor (vemurafenib) in three patients with multisystemic ECD. PATIENTS AND METHODS: Vemurafenib was given to...
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