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

• Published in: Annals of oncology Vol. 13; no. 6; pp. 956 - 964
• Main Authors: Garaventa, A., Boni, L., Lo Piccolo, M. S., Tonini, G. P., Gambini, C., Mancini, A., Tonegatti, L., Carli, M., di Montezemolo, L. C., Di Cataldo, A., Casale, F., Mazzocco, K., Cecchetto, G., Rizzo, A., De Bernardi, B.
• Format: Journal Article
• Language: English
• Published: Oxford Oxford University Press 01.06.2002
• Subjects: Antineoplastic agents; Antineoplastic Combined Chemotherapy Protocols - administration & dosage; Biological and medical sciences; Biopsy, Needle; Chemotherapy; Child; Child, Preschool; Cyclophosphamide - administration & dosage; Dose-Response Relationship, Drug; Doxorubicin - administration & dosage; Drug Administration Schedule; Female; Humans; Infant; Infant, Newborn; Key words: prognostic factors; Male; Medical sciences; Neoadjuvant Therapy; Neoplasm Staging; Neuroblastoma - drug therapy; Neuroblastoma - mortality; Neuroblastoma - pathology; Neuroblastoma - surgery; Pharmacology. Drug treatments; Prognosis; Proportional Hazards Models; Retrospective Studies; Risk Assessment; Survival Analysis; treatment; Treatment Outcome; unresectable neuroblastoma; Vincristine - administration & dosage; Antineoplastic agent; Human; High risk; Nervous system diseases; Prognosis; Treatment efficiency; Malignant tumor; Neuroblastoma; Chemotherapy; Treatment; Risk factor; Unresectable; Autonomic neuropathy; Predictive factor; High dose; Child; Comparative study; Localized
• ISSN: 0923-7534; 1569-8041
• 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.