• Can Huang, Shayi Jiang, Jingwei Yang, Xuelian Liao, Yanhua Li, and Shanshan Li.
• Department of Hematology and Oncology, Children's Hospital of Shanghai, Shanghai Jiao Tong University, Shanghai, China.
• Medicine (Baltimore). 2020 Jun 19; 99 (25): e20853.

IntroductionNeuroblastoma (NB) with MYCN amplification has a poor prognosis and high mortality. The potential molecular biological relationship between clinical features and MYCN amplification should be explored.MethodsNB patients were examined by fluorescence in situ hybridization (FISH) for MYCN amplification in the tumor mass or bone marrow samples to determine whether MYCN was amplified. A series of eleven MYCN-amplified NB patients were included. The age, primary site, tumor size, specific biomarkers, and invaded organs were analyzed. All patients accepted standardized treatment of surgery, chemotherapy, and radiotherapy. Progression-free survival (PFS) and overall survival (OS) were evaluated.ResultsThe median age at diagnosis was 24 months. Nine patients (81.8%) were in stage IV, with high serum neuron-specific enolase (NSE) expression, normal urine vanillylmandelic acid (VMA) level and extensive metastases. All patients accepted a chemotherapy protocol with 8 to 10 cycles, and 9 patients (81.8%) were sensitive to the initial chemotherapy protocol. At the end of follow-up, four patients (36.3%) died with a median OS of 15 months. Five patients (45%) survived with a median PFS of 13 months. Two patients were still receiving chemotherapy.ConclusionGiven the effect of MYCN amplification on poor outcome in NB, novel treatments targeting MYCN should be developed for patients with NB.

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