Subject characteristics (N = 153)
| Characteristics . | Data . |
|---|---|
| Age at last follow-up | |
| Median (range), y | 10.4 (0.3-52.8) |
| >18 y | 39 (25.5) |
| ≤18 y | 114 (74.5) |
| Sex | |
| Male | 92 (60.1) |
| Female | 61 (39.9) |
| Disease manifestation | |
| Severe BMF | 8 (5.2) |
| Malignancy | |
| MDS | 16 (10.5) |
| AML | 10 (6.5) |
| Lymphoma | 1 (0.7) |
| Cause of death (n = 20) | |
| Malignancy | 15 (75) |
| BMF | 2 (10) |
| Asphyxiating thoracic dystrophy | 2 (10) |
| Liver failure | 1 (5) |
| Characteristics . | Data . |
|---|---|
| Age at last follow-up | |
| Median (range), y | 10.4 (0.3-52.8) |
| >18 y | 39 (25.5) |
| ≤18 y | 114 (74.5) |
| Sex | |
| Male | 92 (60.1) |
| Female | 61 (39.9) |
| Disease manifestation | |
| Severe BMF | 8 (5.2) |
| Malignancy | |
| MDS | 16 (10.5) |
| AML | 10 (6.5) |
| Lymphoma | 1 (0.7) |
| Cause of death (n = 20) | |
| Malignancy | 15 (75) |
| BMF | 2 (10) |
| Asphyxiating thoracic dystrophy | 2 (10) |
| Liver failure | 1 (5) |
Unless otherwise noted, data are n (%).