Table 6. Progress in neuroblastoma.
| Discovery | Outcome | Ref. |
|---|---|---|
| Localized NB with normal MYCN | ||
| For resectable stage 1 and 2 NB, surgery without adjuvant chemotherapy is adequate | Stage 1: 5-yr EFS 93%, OS 99%; Stage 2: 5-yr EFS 85%, OS 95% |
(142,144) |
| Infant localized NB with normal MYCN | ||
| Observation does not compromise survival in those with resectable NB | Infant <12 months old: 3-yr OS 99% (n=93); subsequent local or metastatic progression in 30% & 11%. Infant <12 months old & small adrenal mass: 3-yr OS 100% (n=87); 19% required subsequent surgical intervention | (145,146) |
| Omission of anthracyclines in upfront chemotherapy does not compromise survival in those with unresectable NB | 5-yr EFS 90%; OS 99% (n=120); subsequent anthracyclines due to inadequate response in 38% | (147) |
| Intermediate-risk NB | ||
| For favorable biology tumor, reducing chemotherapy from 8 to 4 cycles maintained excellent survival | Favorable biology tumor (4 cycles): 3-yr EFS 90%; OS 98% (n=323). Unfavorable biology tumor (8 cycles): 3-year EFS 83%; OS 93% (n=141) | (141) |
| High-risk NB | ||
| Immunotherapy with ch14.18 (chimeric antibody against GD2) provided the best outcome for post-consolidation biologic therapy | ch14.18 antibody/GM-CSF/IL-2/ isotretinoin: 2-year EFS 66%; OS 86% (n=113). Isotretinoin alone: 2-year EFS 46%; OS 75% (n=113) (P=0.01) | (148) |
| 11q loss of heterozygosity (LOH) | ||
| Poor prognostic factor in stage 2, 3, and 4s tumors without MYCN amplification | <18 months old (stage 2 & 3): 11q LOH, 5-yr EFS 60%; OS 84% (n=176); normal 11q, 5-yr EFS 83%; OS 98% (n=19). ≥18 months old (stage 2 & 3): 1q LOH, 5-yr EFS 61%; OS 73% (n=18); normal 11q, 5-yr EFS 80%; OS 100% (n=49). Stage 4s: 11q LOH, EFS 38% ;OS 63% (n=8); normal 11q, EFS 87%; OS 97% (n=62) | (140) |
IL-2, interleukin-2; OS, overall survival.