Pediatria pre prax 1/2018

Targeted therapy of severe multisystematic Langerhans cell histiocytosis

Langerhans cell histiocytosis (LCH) is a rare disease, with only 4-5 pediatric patients being diagnosed annually in Slovakia. The results of treatment with LCH are very diverse, and the process can resolve spontaneously. Localized disease has an excellent prognosis. Multisystem LCH is treated by systemic chemotherapy and in some cases it is not possible to cure the patient. The new finding is that about half of the LCH cases carry somatic BRAF V600E, which plays a role in the pathophysiology of LCH and opens new options for targeted treatment for patients with an unfavorable prognosis. We describe the case report of a patient with a severe multisystem form of LCH, in which standard therapy failed and child recieved compasionate experimental targeted therapy. In the treatment of patients with a life-threatening form of LCH, new targeted therapy is promising, but these have to be verified in a prospective randomized trial.

Keywords: Langerhans cell histiocytosis, targeted therapy