Early association of Vemurafenib to standard chemotherapy in multisystemic Langerhans cell histiocytosis in a newborn: take the chance for a better outcome?
Abstract:LCH is an aberrant monoclonal proliferation of dendritic cells, ranging
from a self-limiting local condition to a rapidly progressive
multisystemic disease. Pathogenic cells expressed, in almost 70% of
cases, an activation of the MAPK/ERK signaling pathway, in particular
BRAF V600E mutation. We report on a newborn with multisystemic disease
diagnosed in life-threatening medical conditions, who was successfully
treated with the early association of BRAF inhibitor Vemurafenib to
chemotherapy. After 12 months, Ve… Show more
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