Abstract
BACKGROUND: The purpose of the study was to evaluate outcome and pattern of failure in patients with newly diagnosed medulloblastoma treated at the Department of Pediatric Hematology and Oncology in Prague, Czech Republic between 2000–2015. METHODS: Demographics, treatment and survival data were collected in 94 consecutive patients (34 F, 60 M) with histologicaly proven medulloblastoma. Median patient age at the time of diagnosis was 8.43 (0.29 - 17.6) years. Two patients were lost to follow up. Altogether 92 patients were evaluable, Kaplan-Meier analysis was performed to determine survival. Two out of 16 infants (5 metastatic) deceased before the start of any treatment, 14 were treated with chemotherapy only. Older patients (n=75) were treated with radiotherapy and chemotherapy, 44 as a standard risk (SR) and 31 as high risk (HR) group (14 metastatic). One patient with biallelic BRCA2/FANCD1 mutation underwent individualized treatment. RESULTS: The 5-year PFS and OS of all 92 analyzed patients were 66.8% and 72.3%. Survival was higher in older children treated with radiotherapy with 5-year PFS and OS 75.9% and 76.9% respectively. 5-year OS in SR treatment group was 83.0%, in HR group 67.9%. Overall 23 patients have died from all causes. Strikingly, the incidence of secondary malignancies reached 9% (n=9 in 8 patients) and were responsible for 4 deaths. CONCLUSION: The prognosis of medulloblastoma patients is good with over 70% surviving 5 years from the diagnosis with current therapies. Worrisome is a number of SMN. “Supported by the “Project for Conceptual Development of Research Organization 00064203”