Abstract
Abstract only e14546 Background: Neuroblastomas (NBs) are a spectrum of primitive tumors that include Esthesioneuroblastoma (ENB), and both neuroblastoma (NB) and ganglioneuroblastoma (GNB). NB and GNB are prevalent in children and rare in adults with a paucity of data on their therapy and outcomes. This review is to investigate the clinicopathologic features, treatment, and outcomes of NBs in adults. In addition, we highlight genomic findings of a 63-year-old man treated at our institution and discuss ALK-inhibition as a novel treatment strategy for ALK-driven adult-onset NB. Methods: PubMed literature search for NB adults (19+ years) for the period 2000 – 2020 pooled with a case from our institution. Results: Of the 163 included cases, 105 were ENB, 40 were NB, and 18 were GNB. Male:female ratio (1.50:1, 1.29:1, and 1.83:1 respectively). Mean age at diagnosis (49.8, 39.3, and 38.8 respectively). Most common primary site was sinonasal (60%) in ENB, retroperitoneal (20%) in NB, and adrenal (22%) in GNB. Metastasis found in 31.4%, 42.0%, and 16.7% with a mortality rate of 45%, 53%, and 33% respectively. Most common treatment modality overall was surgery with radiation in ENB (35%), Surgery with chemotherapy in NB (28%) and surgery alone in GNB (50%). Most common treatment modality in metastatic cases was surgery with chemoradiation in ENB (50%), surgery with chemotherapy in NB (36%), and surgery alone in GNB (33%). In our patient, ALK gene (R1275Q) gain of function mutation found both in molecular profiling and genomic circulating DNA analysis. Conclusions: Metastatic NB in adults is associated with very high mortality. Their rarity, heterogeneity of clinical behaviors, diverse treatment regimens, and variable outcomes make them even more challenging. The findings of this study would improve our understanding and develop multidisciplinary management strategies for this rare malignancy in adults. Moreover, further research focusing on the potential novel treatment with ALK-targeted inhibitors may hold promise to improve survival outcomes in adult-onset ALK-positive NB. [Table: see text]