Table 1.
Studies of high-risk neuroblastoma with long-term follow-up
| References (years of study)a |
No. of pts |
Stages and age of study population |
Prognostic impact of response/ MYCN(+) |
Staging studies at diagnosis/ follow-up |
Local RT |
CRA | Anti-GD2 immuno- therapy |
Event-free survival |
Overall survival |
|---|---|---|---|---|---|---|---|---|---|
| 1A. Studies in the 1990s | |||||||||
| Zage9 | 142 | 4, >1 yr | …/… | …/post-protocol | yes | no | no | 23% (7 yr) | 28% (7 yr) |
| (1993–95) | 2 or 3, MYCN(+), | every 3–6m, x2 yr | |||||||
| >1 yr | |||||||||
| Matthay10 | 539 | 4, >1 yr | yes/yes | MIBG optional/ | no | ran- | no | 26% (5 yr) | 36% (5 yr) |
| (1991–96) | 4, <1 yr, MYCN(+) | at end of protocol | domised | stage 4: 21% (5 yr) | 32% (5 yr) | ||||
| 3, MYCN(+) | stage 3: 55% (5 yr) | 59%(5 yr)b | |||||||
| 3, UH and/or | |||||||||
| ferritin ≥143 ng/ml | |||||||||
| Berthold7 | 335 | 4, >1 yr | …/… | …/… | no | no | no | 27% (5 yr) | 33% (5 yr) |
| (1990–97) | |||||||||
| DeBernardi6 | 159 | 4, >1 yr | none/yes | …/… | no | no | no | 17% (5 yr) | 28% (5 yr) |
| (1992–97) | |||||||||
| Kaneko5 | 221 | 4, >1 yr | …/none | MIBG optional/ | no | no | no | 34% (5 yr) | 37% (5 yr) |
| (1991–98) | … | ||||||||
| Castel4 | 83 | 4, >1 yr | none/yes | …/… | no | no | no | 33% (4 yr) | 33% (4 yr) |
| (1992–98) | 4, <1 yr, MYCN(+) | … | 20% (5 yr) | ||||||
| 3, MYCN(+) | |||||||||
| Pearson8 | 262 | 4, >1 yr | yes/none | MIBG optional/ | no | no | no | 28% (3 yr) | … |
| (1990–99) | “local preference” | 24% (5 yr) | 26% (5 yr) | ||||||
| 23% (10 yr) | 24%(10yr) | ||||||||
| 1B. Studies completed in the 2000s | |||||||||
| George13 | 97 | 4, >1 yr | …/none | MIBG variable/ | yes | yes | no | 55% (3 yr) | 72% (3 yr) |
| (1994–2002) | 3, 4, 4S, <1 yr, | … | 47% (5 yr) | 60% (5 yr) | |||||
| MYCN(+) | 46% (7 yr) | 53% (7 yr) | |||||||
| 3, >1 yr, UH | |||||||||
| Berthold12 | 295 | 4, >1 yr | yes/yes | …/… | 24 pts | 39 pts | 160 pts | 39% (3 yr) | 58% (3 yr) |
| (1997–2002) | all stages MYCN(+) | 35% (5 yr) | 45% (5 yr) | ||||||
| Canete19 | 35 | 2, 3, 4, 4S, | yes/… | MIBG if available/ | yes | yes | no | 29% (2 yr) | 30% (2 yr) |
| (1999–2004) | <1 yr, MYCN(+) | … | stage 4: 20% (2 yr) | ||||||
| Sung14 | 52 | 4, >1 yr | none/none | MIBG included/ | 35 pts | yes | no | 62% (5 yr) | 64% (5 yr) |
| (1997–2005) | … | ||||||||
| Kreissman15 | 495 | 4, >1 yr | …/… | …/… | yes | yes | 78 pts | 38% (5 yr) | 50% (5 yr) |
| (2001–2006) | 3, 4, 4S, <1 yr, | ||||||||
| MYCN(+) | |||||||||
| 3, >1 yr, UH | |||||||||
Abbreviations: CRA, 13-cis-retinoic acid; MYCN(+), MYCN-amplified; pts, patients; UH, unfavorable histology; RT, radiotherapy
… Indicates not reported or described.
a
Listed chronologically by year of study’s completion.
b
5-yr EFS/OS rates for MYCN(+) stage 3 were 25%/27% and for MYCN(−) stage 3 were 77%/79%.11