Predictors of survival in patients with high‐risk neuroblastoma who failed tandem high‐dose chemotherapy and autologous stem cell transplantation

Objective This study aims to explore prognostic factors for high‐risk neuroblastoma patients with response failure to tandem high‐dose chemotherapy and autologous stem cell transplantation (HDCT/auto‐SCT). Methods Survival outcomes were compared according to characteristics at initial diagnosis, at relapse/progression, and after relapse/progression in patients who experienced relapse/progression after tandem HDCT/auto‐SCT from 2006 to 2018. Results Forty‐nine patients experienced relapse/progression after tandem HDCT/auto‐SCT during the study period: 43 received salvage treatment and 30 underwent allogeneic SCT (allo‐SCT) after reinduction treatment. Although all six patients who did not undergo salvage treatment died, 13 of the 43 patients who did remain alive. The 3‐year probabilities of event‐free survival (EFS) and overall survival (OS) from initial relapse/progression among the 49 patients were 14.4% ± 5.2% and 21.2% ± 6.4%, respectively. A higher neuron‐specific enolase (NSE) level (>24 ng/mL) at relapse/progression was an independent prognostic factor for worse OS. Nine of 30 patients who underwent allo‐SCT remain alive, and the 3‐year probabilities of EFS and OS from allo‐SCT were 16.5% ± 7.2% and 21.6% ± 8.3%, respectively. A higher NSE level and no incorporation of high‐dose 131I‐metaiodobenzylguanidine (HD‐MIBG) treatment into allo‐SCT were independent prognostic factors for worse EFS and OS after allo‐SCT. Conclusion The results suggest that a higher serum NSE level at relapse/progression is a predictor of worse prognosis in patients with response failure to tandem HDCT/auto‐SCT, and that incorporation of HD‐MIBG treatment into allo‐SCT may improve outcomes.