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

Alberto Garaventa, L. Boni, M. S. Lo Piccolo, G. P. Tonini, Claudio Gambini, A. Mancini, L. Tonegatti, Modesta Carli, L. C. di Montezemolo, Andrea Di Cataldo, Fiorina Casale, K. Mazzocco, G. Cecchetto, Antonino Rizzo, Bruno De Bernardi, Teresa Di Tullio, Maurizio Bianchi, Gianni Bisogno, Giovanni Surico, Nicola SantoroAntonia Mancini, Patricia Alvisi, Margherita Lo Curto, Fabio Schumacher, Paolo Tamaro, Monica Cellini, Giancarlo Izzi, Gabriella Bernini, Angela Tamburrini, Pier Emilio Cornelli, Luciano Musi, Augusto Amici, Antonio Acquaviva, Roberto Targhetta, Domenico Gallisai, Massimo Conte, Paolo Magillo, Alberto Donfrancesco, Alessandro Jenker, Clementina De Laurentis

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish
Pages (from-to)956-964
Number of pages9
JournalAnnals of Oncology
Issue number6
Publication statusPublished - 2002


  • Prognostic factors
  • Treatment
  • Unresectable neuroblastoma

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

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