ABSTRACT
We report a case of congenital multisystem Langerhans cell histiocytosis with cutaneous and hematopoietic involvement. After the failure of first-line (vinblastine and prednisolone) and second-line (vincristine and cytarabine) therapies, treatment with cobimetinib, a mitogen-activated protein kinase (MEK) inhibitor, led to the remission of disease and a sustained response after 11 months of ongoing treatment. Protein kinase inhibitors targeting BRAF or MEK could represent a promising future therapeutic option, also in children with LCH.
Subject(s)
Azetidines , Histiocytosis, Langerhans-Cell , Piperidines , Humans , Histiocytosis, Langerhans-Cell/drug therapy , Histiocytosis, Langerhans-Cell/congenital , Azetidines/therapeutic use , Piperidines/therapeutic use , Protein Kinase Inhibitors/therapeutic use , Male , Female , InfantABSTRACT
When the diagnosis of penile calciphylaxis is suggested, the evolution of the disease is rapidly unfavorable; in this case, a rapid medical treatment must be established to obtain an improvement of the disease to avoid penectomy.
