P11.12.A Primary central nervous system neuroblastoma and ganglioneuroblastoma in adult patients. Clinical and molecular features

Abstract

Abstract Background The clinical, morphological, molecular features, treatment modalities, and survival of primary central nervous system neuroblastoma and ganglioneuroblastoma (CNS-NB and CNS-GNB) in adult patients are still poorly understood. Material and Methods The study included 15 patients with CNS-NB and 16 patients with CNS-GNB aged 18 years and older who were treated in our clinic from 2008 to 2020. Clinical, radiological data and surgical results were analyzed. Analysis of tumor molecular profile was performed using the gene panel, which contained the majority of clinically significant genes. Results Progression-free survival (PFS) and overall survival (OS) were higher in patients with CNS-NB than in patients with CNS-GNB. Median PFS in patients with CNS-NB and CNS-GNB was 156 and 46 weeks, respectively (p=0.022), and median OS was 541 and 82 weeks, respectively (p=0.00045). In both groups, disease progression was more favorable for tumor localization in the cerebral hemispheres without involvement of basal structures (p <0.05). Differences for OS in patients with CNS-NB were obtained when tumor volume was removed by more than 50% with a median OS of 541 weeks (p=0.042) compared to biopsy. In patients with CNS-GNB, total resection of the tumor increased both PFS and OS compared to subtotal resection (p=0.014 and p=0.017), respectively. In patients with CNS-NB, no benefit was observed for any of the different first-line chemotherapy regimens (p > 0.05). In the group of patients with CNS-GNB, 6 cycles of temozolomide increased median PFS compared to other chemotherapeutic regimens (p=0.026). In CNS-NB, a high level of expression was observed only in the βIII-tubulin gene (54%, 7/13). In CNS-GNB, high levels of expression were found in three genes: PDGFR-α (54%, 7/13), VEGF (54%, 7/13), and βIII-tubulin (85%, 11/13). No statistically significant effect of mutation in IDH1(R132H) gene on median PFS and OS was observed. The OS of patients with CNS-NB and CNS-GNB was influenced by low or medium levels of VEGF expression (p=0.006), and the OS was influenced by three genes: ERCC1 (p=0.03), MGMT (p=0.029) and VEGF (p=0.002). Conclusion The morphologic type of the tumor determined survival. In both groups, median PFS and OS were influenced by tumor localization. The most important prognostic marker in adult patients with CNS-NB and CNS-GNB was VEGF, while MGMT and ERCC1 were predictive markers.