High-Dose Chemotherapy and Autologous Stem Cell Rescue Therapy for High-Risk Neuroblastoma Patients

Abstract

Background: Neuroblastoma (NBL) accounts for 8% to 10% of all childhood cancers, and more than half of these cases are advanced stages. Vigorous research on single, tandem or triple high-dose chemotherapy (HDCT) and autologous stem cell rescue therapy have improved the survival of many high-risk NBL patients, however, patients remain to have a dismal prognosis. In 2002, the Korean National Health Insurance policy extended coverage to include children over 1 year of age diagnosed with stage IV that showed partial response to surgery or chemotherapy and stage III in which complete resection was infeasible. As the burden of medical expenses was lifted, high-risk NBL patients were able to receive tandem HDCT more readily. The objective of this study was to conduct a historical analysis of patient outcome after the implementation of the Korean health care reform. Methods: During the period between October 1997 and December 2010, a retrospective analysis was performed of 60 patients with stage III or IV NBL who received single or tandem HDCT with stem cell rescue therapy at Seoul National University Childrens Hospital (SNUCH). The overall survival (OS) and event-free survival (EFS) were analyzed. Results: Sixty patients diagnosed with NBL received single or tandem HDCT and autologous stem cell rescue therapy. Five-year OS of the single and tandem group were 82.9% (95% CI, 69.4%-96.4%) and 85.6% (95% CI, 72.5%-98.7%), respectively (P=0.590). Five-year EFS of the single and tandem group were 52.9% (95% CI, 30.9%-67.4%) and 71.4% (95% CI, 43.3%-81.0%) (P=0.234). An event was more likely attributed to relapse, rather than treatment related mortality (TRM). Conclusion: With the change of the Korean National Health Insurance policy, an improvement of outcome in NBL patients receiving autologous stem cell rescue was observed. However, relapse remains to be an issue. As subclinical disease activity is correlated with disease relapse, monitoring disease activity before relapse is crucial and identifying molecular targets for future therapies are warranted.