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


METHODS
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.
Garaventa
A
A
Department of Hematology--Oncology, Giannina Gaslini Children's Hospital, Genova, Italy. albertogaraventa@ospedale-gaslini.ge.it
Boni
L
L
Lo Piccolo
M S
MS
Tonini
G P
GP
Gambini
C
C
Mancini
A
A
Tonegatti
L
L
Carli
M
M
di Montezemolo
L C
LC
Di Cataldo
A
A
Casale
F
F
Mazzocco
K
K
Cecchetto
G
G
Rizzo
A
A
Bernardi
B
B
D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage*; D001707:Biopsy, Needle; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage; D004305:Dose-Response Relationship, Drug; D004317:Doxorubicin / Q000008:administration & dosage; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D020360:Neoadjuvant Therapy; D009367:Neoplasm Staging; D009447:Neuroblastoma / Q000188:drug therapy* / Q000401:mortality* / Q000473:pathology / Q000601:surgery; D011379:Prognosis; D016016:Proportional Hazards Models; D012189:Retrospective Studies; D018570:Risk Assessment; D016019:Survival Analysis; D016896:Treatment Outcome; D014750:Vincristine / Q000008:administration & dosage
D014750:Vincristine; D004317:Doxorubicin; D003520:Cyclophosphamide