MEK Inhibition in the Treatment of Congenital Langerhans Cell Histiocytosis: A Case Report and Review of the Literature
Natalia Wojciechowska,
Sydney Burke,
Anish Ray
Abstract:Langerhans cell histiocytosis (LCH) is a histiocytic disorder that predominantly affects young children, with congenital manifestations being exceedingly rare. Here, we report a male infant with congenital LCH harboring a driving mutation within the mitogen-activated protein kinase pathway, specifically MAP2K1 Q56P. First-line use of targeted therapy with oral MEK inhibitor trametinib led to rapid and complete resolution of the infant’s widespread cutaneous disease. This patient remains clinically well with no… Show more
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