Table 1.
Characteristics of Patients With Metastatic Neuroblastoma Lacking MYCN Gene Amplification
Characteristic | Training Cohort (age at diagnosis, months) |
Validation Cohorts (age at diagnosis, months) |
||||||
---|---|---|---|---|---|---|---|---|
CCG |
GPOH |
COG |
||||||
< 18 (n = 39) |
≥ 18 (n = 94) |
≥ 18 (n = 39) |
≥ 18 (n = 52) |
|||||
No. | % | No. | % | No. | % | No. | % | |
Age at diagnosis, months | ||||||||
Mean | 9.3 | 53.6 | 56 | 55.9 | ||||
Range | 0.1-17.3 | 18.2-151 | 18.4-182 | 19.5-186 | ||||
COG risk stratification | Intermediate* | High | High | High | ||||
INPC classification | ||||||||
Favorable | 34 | 87 | 2 | 2 | 3 | 8 | 3 | 6 |
Unfavorable | 5 | 13 | 91 | 97 | 34 | 87 | 42 | 81 |
Unknown | 0 | 0 | 1 | 1 | 2 | 5 | 7 | 13 |
Clinical trials | 323P, 3881 | 323P, 321-2, 321-3, 3891 | NB90, NB95, NB97, NB2004 | A3973† | ||||
5-year EFS rate, % | 95 | 23 | 34 | 31 | ||||
95% CI, % | 81 to 99 | 15 to 32 | 19 to 50 | 19 to 44 | ||||
5-year OS rate, % | 95 | 35 | 50 | 38 | ||||
95% CI, % | 81 to 99 | 25 to 44 | 32 to 66 | 23 to 54 |
Abbreviations: CCG, Children's Cancer Group; COG, Children's Oncology Group; EFS, event-free survival; GPOH, German Society for Pediatric Oncology-Hematology (Gesellschaft für Paediatrische Onkologie und Haematologie); INPC, International Neuroblastoma Pathology Classification; OS, overall survival.
Patients age > 12 months were classified as high risk and treated accordingly per CCG guidelines. On the basis of current COG guidelines, four of 12 patients diagnosed at 12 to 18 months of age with unfavorable tumor histology would be considered high risk.
Three patients were treated on ANBL0532.