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, Vemurafenib was discontinued, without any
signs of relapse. This case indicates that early combination of target
therapy with standard treatment may induce rapid response and prolonged
disease remission.