Table 1.
Characterisitcs and outcome of patients with MUYCN-non-amplified NB
| Group A: initial treatment at MSKCC (n = 14) |
Group B: treated at MSKCC after initial treatment elsewhere (n = 39) |
|
|---|---|---|
| M:F ratio | 9:5 | 17:22 |
| Age:median (range) in years | 1.3 (0.1–5.1) | 1.6 (0.1–8.8) |
| Number of patients >24 months | 5 | 16 |
| Number of patients >12 months | 10 | 29 |
| Number of patients <6 months | 1 | 3 |
| Primary site | ||
| Cervical | 1 | 0 |
| Mediastinal | 3 | 4 |
| Retroperitoneal | 5 | 21 |
| Adrenal | 3 | 10 |
| Pelvic | 2 | 4 |
| Epidural disease | 7 | 14 |
| L2 classification by INRG | 13 | 35 |
| Serum LDH (mean ± SD) U/L | 354 ± 154 | 814 ± 687 (n = 20) |
| Serum ferritin (mean ± SD) ng/mL | 64 ± 78 | 64 ± 52 (n = 17) |
| Hyperdiploidy | 11/12 | 24/31 |
| Favourable histology | 12/13 | 28/34 |
| Patients with diploid tumours AND unfavourable histology | 0/12 | 0/29 |
| Pre-surgery chemotherapy | 0 | 39 |
| Intermediate-dose | 34 | |
| COG protocol 3961 | 26 | |
| Protocol 3961 – 1 cycle | 1 | |
| Protocol 3961 – 2 cycles | 4 | |
| Protocol 3961 – 4 cycles | 11 | |
| Protocol 3961 – 8 cycles | 10 | |
| Other intermediate-dose | 8 | |
| High-dose | 0 | 4 |
| High-dose + SCT | 0 | 1 |
| Pre-surgery radiotherapy | 0 | 3 |
| Gross total resection | 5 | 25 |
| Relapses | 3 | 0 |
| Deaths | 1 | 1 |
| Median follow-up (months) | 58.8 | 46.5 |
| 5 year EFS | 84.6 ± 10% | 97.1 ± 3% |
| 10-year OS | 84.6 ± 14% | 97.1 ± 3% |
Abbreviations: COG: Children’s Oncology Group; EFS: event-free survival; INRG: International Neuroblastoma Research Group; MSKCC: Memorial Sloan-Kettering Cancer Centre; OS: overall survival; SCT: stem cell transplant; SD: standard deviation.