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

Abstract

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.

• Authors: A Garaventa, L Boni, M S Lo Piccolo, G P Tonini, C Gambini, A Mancini, L Tonegatti, M Carli, L C di Montezemolo, A Di Cataldo, F Casale, K Mazzocco, G Cecchetto, A Rizzo, B Bernardi, Italian Cooperative Group for Neuroblastoma
• Journal: Annals of oncology : official journal of the European Society for Medical Oncology (Ann Oncol) Vol. 13 Issue 6 Pg. 956-64 (Jun 2002) ISSN: 0923-7534 [Print] England
• PMID: 12123342 (Publication Type: Comparative Study, Journal Article, Research Support, Non-U.S. Gov't)

Chemical References

• Vincristine
• Doxorubicin
• Cyclophosphamide

Topics

• 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, mortality, pathology, surgery)
• Prognosis
• Proportional Hazards Models
• Retrospective Studies
• Risk Assessment
• Survival Analysis
• Treatment Outcome
• Vincristine (administration & dosage)