title: [Therapeutic Effect and Tolerance of Ipilimumam in Metastatic Malignant Melanoma in Children -  a Case Report].
abstract: BACKGROUND
  Malignant melanoma is a rare malignancy in the pediatric population. Etiology is usually unknown. Clinical symptoms are nonspecific, clinical behavior and biology features may differ from those in an adult population. The most important prognostic factor is spread of disease. Surgical resection is treatment of choice for localized melanoma. Advanced and metastatic melanoma is still an incurable disease.
  
  
  METHODS
  We are presenting an eight-year- old boy with metastatic malignant melanoma of unknown origin based on TP53 mutation (Li Fraumeni syndrome). He underwent surgery and adjuvant chemotherapy (temozolomide as single agent). Complete remission was achieved at the end of treatment. Two years after the end of therapy (and 31 months from diagnosis) he developed metastatic progression to the lungs. He has received immunotherapy with ipilimumab, according to our knowledge as the first child under the age of 12 in Europe. He completed three courses of ipilimumab, with irAE (immune related adverse event) grade III during the first course of anti CTLA 4. Therefore, further doses of ipilimumab were given with corticoids and antihistamines as premedication. Also, asymptomatic thyreoiditis grade II has been confirmed. The best documented treatment response is stable disease. Performance status was excellent. Three years since the first progression, he developed further massive progression to the lungs. Second line immunotherapy with anti-PD 1 monoclonal antibody (pembrolizumab) is currently going on. So far, the overall survival of the patient is 74 months.
  
  
  CONCLUSIONS
  The presented case study supports the administration of immunotherapy in children younger than 12 years. Therapeutic effect has led to significant overall survival with tolerable toxicity. The problem remains significantly limited number of pediatric clinical trials using immunotherapy.
authors.forename: V
authors.initials: V
authors.lastname: Bajčiová
mesh_terms: D000911:Antibodies, Monoclonal / Q000627:therapeutic use*; D060908:CTLA-4 Antigen / Q000037:antagonists & inhibitors*; D002648:Child; D004361:Drug Tolerance; D006801:Humans; D000074324:Ipilimumab; D008297:Male; D008545:Melanoma / Q000188:drug therapy* / Q000556:secondary
chemical_list: D000911:Antibodies, Monoclonal; D060908:CTLA-4 Antigen; D000074324:Ipilimumab