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

Background: We previously reported that stage 3 neuroblastoma comprises (i) a low-risk groupincluding 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 chemotherapywas effective only in the latter group.Patients and treatment: On this basis, in 1990 we designed a new protocol by which all low-riskpatients received standard-dose chemotherapy, while the high-risk ones received very aggressivechemotherapy.Results: Between November 1990 and December 1997 a total of 95 eligible and evaluable childrenwere enrolled: 47 were low-risk (35 infants and 12 >1 year of age at diagnosis and having non-abdominalprimaries), and 48 were high-risk (being >1 year of age and having abdominal primaries). Of the47 low-risk patients, five relapsed and four subsequently died. The 5-year overall survival (OS) was91%. Of the 48 patients in the high-risk group, 22 relapsed or progressed, 18 of whom died from theirdisease and two from toxicity, and one was lost to follow-up. The 5-year OS was 60%. Univariateanalysis showed that age, site of primary, risk-group, urine vanillylmandelic excretion, plasma levels oflactate 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 anexcellent chance of being cured. Aggressive chemotherapy is effective for high-risk patients, but resultsare still unsatisfactory. MYCN gene amplification is a prognostic indicator for most, but not all, treatmentfailures.