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Vemurafenib for Refractory Multisystem Langerhans Cell Histiocytosis in Children: An International Observational Study
PURPOSE: Off-label use of vemurafenib (VMF) to treat BRAF(V600E) mutation–positive, refractory, childhood Langerhans cell histiocytosis (LCH) was evaluated. PATIENTS AND METHODS: Fifty-four patients from 12 countries took VMF 20 mg/kg/d. They were classified according to risk organ involvement: live...
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