Risk-based Grouping of Patients and Risk-directed Treatment in Neuroblastoma / 대한소아혈액종양학회지

ABSTRACT

PURPOSE: This study was done to evaluate whether risk-directed treatment can improve survival of patients with high risk neuroblastoma (NBL). METHODS: Forty two patients with NBL were newly diagnosed and treated at Samsung Seoul Hospital from June 1997 to December 2000. Patients were divided into high risk or low risk group according to 3 important prognostic factors. Poor prognostic factors were defined as follows; amplification of N-myc oncogene, age at diagnosis higher than 1 years, and INSS stage 4. Patients with 2 or more poor prognostic factors were defined as high risk patients. While conventional treatment including surgery, radiotherapy, and pre and post-operative chemotherapy was applied to low risk patients, intensive multimodality treatment including single or double high dose chemotherapy (HDCT) and autologous peripheral blood stem cell transplantation (PBSCT) followed by immunotherapy using interleukin-2 (IL-2) and differentiating therapy using 13-cis-retinoic acid (CRA) was applied to high risk patients. RESULTS: Among 42 patients, 30 patients were high risk, 10 patients were low risk, and 2 patients were impossible to classify. Forty five HDCTs and PBSCTs were applied to 28 high risk patients and 2 low risk patients. All of the low risk patients are alive without relapse. Three year event free survival (EFS) after diagnosis in high risk patients was 54.8%. EFS after diagnosis in patients with 2 or 3 risk factors were 81.3%, 39.3% (P=0.0292) respectively. EFS after HDCT was 65.1%. EFS after HDCT in patients with 2 or 3 risk factors were 85.7%, 47.1% (P=0.0527) respectively. CONCLUSION: Risk-based grouping of patients and risk-directed treatment are necessary for better outcome. Multimodality treatment including HDCT and autologous PBSCT followed by immunotherapy using IL-2 and differentiatin therapy using CRA can improve survival in high risk patients.