Localized unresectable neuroblastoma: results of treatment based on clinical prognostic factors

Ann Oncol. 2002 Jun;13(6):956-64. doi: 10.1093/annonc/mdf165.


Background: We previously reported that stage 3 neuroblastoma comprises (i) a low-risk group including all infants (age 0-11 months) as well as older children with non-abdominal primaries, and (ii) a high-risk group made up of children >1 year of age with abdominal primaries. Aggressive chemotherapy was effective only in the latter group.

Patients and treatment: On this basis, in 1990 we designed a new protocol by which all low-risk patients received standard-dose chemotherapy, while the high-risk ones received very aggressive chemotherapy.

Results: Between November 1990 and December 1997 a total of 95 eligible and evaluable children were enrolled: 47 were low-risk (35 infants and 12>1 year of age at diagnosis and having non-abdominal primaries), and 48 were high-risk (being >1 year of age and having abdominal primaries). Of the 47 low-risk patients, five relapsed and four subsequently died. The 5-year overall survival (OS) was 91%. Of the 48 patients in the high-risk group, 22 relapsed or progressed, 18 of whom died from their disease and two from toxicity, and one was lost to follow-up. The 5-year OS was 60%. Univariate analysis showed that age, site of primary, risk-group, urine vanillylmandelic excretion, plasma levels of lactate dehydrogenase, ferritin and neurone-specific enolase, and MYCN status correlated with outcome. However, multivariate analysis showed that only MYCN status retained prognostic value.

Conclusions: In low-risk stage 3 neuroblastoma, standard-dose chemotherapy is associated with an excellent chance of being cured. Aggressive chemotherapy is effective for high-risk patients, but results are still unsatisfactory. MYCN gene amplification is a prognostic indicator for most, but not all, treatment failures.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / administration & dosage*
  • Biopsy, Needle
  • Child
  • Child, Preschool
  • Cyclophosphamide / administration & dosage
  • Dose-Response Relationship, Drug
  • Doxorubicin / administration & dosage
  • Drug Administration Schedule
  • Female
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Neoadjuvant Therapy
  • Neoplasm Staging
  • Neuroblastoma / drug therapy*
  • Neuroblastoma / mortality*
  • Neuroblastoma / pathology
  • Neuroblastoma / surgery
  • Prognosis
  • Proportional Hazards Models
  • Retrospective Studies
  • Risk Assessment
  • Survival Analysis
  • Treatment Outcome
  • Vincristine / administration & dosage


  • Vincristine
  • Doxorubicin
  • Cyclophosphamide