Table 1 Summary of demographic data and transplant characteristics by disease.
Disease | |||||
|---|---|---|---|---|---|
ALL (N = 23) | AML (N = 29) | MDS (N = 10) | JMML (N = 3) | Overall (N = 65) | |
Sex [n (%)] | |||||
Male | 15 (65.2%) | 19 (65.5%) | 5 (50.0%) | 3 (100.0%) | 42 (64.6%) |
Female | 8 (34.8%) | 10 (34.5%) | 5 (50.0%) | 0 (0.0%) | 23 (35.4%) |
Age [years] | |||||
Mean (SD) | 10.5 (5.6) | 8.2 (5.6) | 11.6 (5.7) | 2.0 (2.0) | 9.3 (5.8) |
Median | 12.0 | 8.0 | 14.0 | 2.0 | 11.0 |
Min., Max. | 1, 17 | 0, 17 | 1, 17 | 0, 4 | 0, 17 |
ICH age group [n (%)] | |||||
28 days to 23 months | 2 (8.7%) | 4 (13.8%) | 1 (10.0%) | 1 (33.3%) | 8 (12.3%) |
2–11 years | 7 (30.4%) | 14 (48.3%) | 2 (20.0%) | 2 (66.7%) | 25 (38.5%) |
12–17 years | 14 (60.9%) | 11 (37.9%) | 7 (70.0%) | 0 (0.0%) | 32 (49.2%) |
Number of HSCT [n (%)] | |||||
1st | 22 (95.7%) | 28 (96.6) | 8 (80) | 2 (66.7%) | 60 (92.3%) |
2nd | 1 (4.3%) | 1 (3.4) | 2 (20) | 1 (33.3%) | 5 (7.7%) |
Number of complete remission [n (%)]a | |||||
1. CR | 16 (69.6%) | 25 (86.2%) | na | na | 41 (63.1%) |
2. CR | 7 (30.4%) | 3 (10.3%) | na | na | 10 (15.4%) |
3. CR (or higher) | 0 (0.0) | 1 (3.4%) | na | na | 1 (1.5%) |
Secondary origin of malignancy [n (%)] | |||||
Yes | 1 (4.3) | 0 (0.0) | 4 (40) | 0 (0.0) | 5 (7.7) |
Treosulfan dose [n (%)] | |||||
10 g/m2/day −6, −5, −4 | 1 (4.3%) | 3 (10.3%) | 0 (0.0%) | 1 (33.3%) | 5 (7.7%) |
12 g/m2/day −6, −5, −4 | 5 (21.7%) | 13 (44.8%) | 3 (30.0%) | 2 (66.7%) | 23 (35.4%) |
14 g/m2/day −6, −5, −4 | 17 (73.9%) | 13 (44.8%) | 7 (70.0%) | 0 (0.0%) | 37 (56.9%) |
Donor type [n (%)] | |||||
Matched sibling | 6 (26.1%) | 4 (13.8%) | 1 (10.0%) | 0 (0.0%) | 11 (16.9%) |
Matched family | 0 (0.0%) | 1 (3.4%) | 0 (0.0%) | 0 (0.0%) | 1 (1.5%) |
Matched unrelated | 17 (73.9%) | 24 (82.8%) | 9 (90.0%) | 3 (100.0%) | 53 (81.5%) |
Source [n (%)] | |||||
Bone marrow | 14 (60.9%) | 13 (44.8%) | 3 (30.0%) | 3 (100.0%) | 33 (50.8%) |
Peripheral blood | 9 (39.1%) | 16 (55.2%) | 7 (70.0%) | 0 (0.0%) | 32 (49.2%) |
