PMID- 32569234 OWN - NLM STAT- MEDLINE DCOM- 20200720 LR - 20221005 IS - 1536-5964 (Electronic) IS - 0025-7974 (Print) IS - 0025-7974 (Linking) VI - 99 IP - 25 DP - 2020 Jun 19 TI - Therapeutic potential of targeting MYCN: A case series report of neuroblastoma with MYCN amplification. PG - e20853 LID - 10.1097/MD.0000000000020853 [doi] LID - e20853 AB - INTRODUCTION: Neuroblastoma (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. METHODS: NB 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. RESULTS: The 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. CONCLUSION: Given the effect of MYCN amplification on poor outcome in NB, novel treatments targeting MYCN should be developed for patients with NB. FAU - Huang, Can AU - Huang C AD - Department of Hematology and Oncology, Children's Hospital of Shanghai, Shanghai Jiao Tong University, Shanghai, China. FAU - Jiang, Shayi AU - Jiang S FAU - Yang, Jingwei AU - Yang J FAU - Liao, Xuelian AU - Liao X FAU - Li, Yanhua AU - Li Y FAU - Li, Shanshan AU - Li S LA - eng PT - Journal Article PL - United States TA - Medicine (Baltimore) JT - Medicine JID - 2985248R RN - 0 (MYCN protein, human) RN - 0 (N-Myc Proto-Oncogene Protein) SB - IM MH - Child, Preschool MH - Combined Modality Therapy MH - Female MH - Humans MH - In Situ Hybridization, Fluorescence MH - Infant MH - Male MH - N-Myc Proto-Oncogene Protein/*metabolism MH - Neuroblastoma/metabolism/mortality/*pathology/therapy MH - Survival Analysis PMC - PMC7310875 COIS- The authors have no conflicts of interest to disclose. EDAT- 2020/06/23 06:00 MHDA- 2020/07/21 06:00 PMCR- 2020/06/19 CRDT- 2020/06/23 06:00 PHST- 2020/06/23 06:00 [entrez] PHST- 2020/06/23 06:00 [pubmed] PHST- 2020/07/21 06:00 [medline] PHST- 2020/06/19 00:00 [pmc-release] AID - 00005792-202006190-00082 [pii] AID - MD-D-19-10197 [pii] AID - 10.1097/MD.0000000000020853 [doi] PST - ppublish SO - Medicine (Baltimore). 2020 Jun 19;99(25):e20853. doi: 10.1097/MD.0000000000020853.