This retrospective study evaluated the long-term outcomes and prognostic indicators of patients with stage 4 neuroblastoma who were older than 18 months at diagnosis. The medical records of 118 such children who were treated at Shanghai Children's Medical Center, China, from June 1998-December 2013 were reviewed. Event-free survival (EFS) and overall survival (OS) were analyzed by log-rank tests. Of the 118 patients, 14 improving patients did not complete treatment because of parental decisions, and 1 patient died during surgery. Of the 103 patients who completed the comprehensive protocols, 60 (58.3%) achieved very good partial remission (VGPR), 26 (25.2%) achieved partial remission (PR) after four courses of chemotherapy, and 17 (16.5%) progressed during treatment. The response to induction (including VGPR + PR) was 83.5%. After a median follow-up of 105 months (range 36-160 months), the 5- and 10-year OS were 21 and 18%, and the EFS was 19 and 13%, respectively. EFS was significantly better for patients with normal levels of urinary vanillylmandelic acid (VMA) at diagnosis, who had complete resection of the primary tumor, who were minimal residual disease- (MRD-) negative in their bone marrow after four courses of chemotherapy, and who achieved VGPR at the end of treatment (P < 0.05). The prognosis remains poor for patients with stage 4 neuroblastoma who are older than 18 months at diagnosis. Elevated VMA level, incomplete tumor resection, persistent MRD in bone marrow, and poor curative effect are associated with worse prognosis.
Keywords: Neuroblastoma; Risk factors; Stage 4.