Table 1.
Patient characteristics and time of sampling.
| Patient Characteristics | Total (n = 25) |
|---|---|
|
Gender male female |
14 (56%) 11 (44%) |
| Median age at diagnosis, year, (range) | 3.9 (0.3–10.8) |
| Stage 3 disease | 1 (4%) |
| Stage 4 disease | 24 (96%) |
| Genetics | |
|
MYCN Neg Gain Amp |
18 (72%) 2 (8%) 5 (20%) |
|
1p normal partial loss loss gain |
14 (56%) 9 (36%) 1 (4%) 1 (4%) |
|
17q normal partial gain gain unknown |
1 (4%) 10 (40%) 11 (44%) 3 (12%) |
|
ALK mutation Yes no gain unknown |
5 (20%) 16 (64%) 1 (4%) 3 (12%) |
| CR or PR after induction chemotherapy (3× N5/N6) | 14 (56%) |
| HD + ASCT | 20 (80%) |
| Conditioning Regimen | |
| Busulfan/melphalan | 14/20 (70%) |
| Carboplatin/etoposide/melphalan | 6/20 (30%) |
| CD34+ cell dose ×106/kg, (range) | 2.47 (0.59–21.73) |
| Immunotherapy | 16 (64%) |
| Time to immunotherapy, d, (range) | 137 (108–193) |
| Event: progression or relapse | 7 (31%) |
| Event: Refractory Disease | 3 (14%) |
| Event: Toxicity | 1 (5%) |
| Alive at last FU | 14 (56%) |
| Median EFS, year (range) | 1.65 (0.11–3.67) |
| Median follow-up OS, year, (range) | 2.14 (0.65–3.67) |
Abbreviations:CR, complete response; PR, partial response; HD, high-dose; ASCT, autologous stem cell transplantation; FU, follow-up; EFS, event-free survival; OS, overall survival.