Table 1.
Characteristics of 159 patients and their CBUs
| Characteristics | Median | Range |
|---|---|---|
| Age, y | 1.50 | 0.05-26.25 |
| Weight at transplantation, kg | 12.0 | 2.74-73.10 |
| Cryopreserved TNCs, × 107/kg | 9.73 | 2.24-50.37 |
| Reinfused TNCs, × 107/kg | 7.57 | 1.49-32.40 |
| Postthaw reinfused CD34+, × 105/kg | 2.14 | 0.42-104.75 |
| Postthaw reinfused CD3+, × 106/kg | 14.15 | 3.26-100.56 |
| Total postthaw reinfused CFUs, × 104/kg | 5.74 | 0.00-105.30 |
| Primary disease | N | % |
| Hurler syndrome | 45 | 28.3 |
| Krabbe disease | 36 | 22.6 |
| Sanfilippo syndrome | 19 | 11.9 |
| Metachromatic leukodystrophy | 15 | 9.4 |
| Adrenoleukodystrophy (ALD) | 13 | 8.2 |
| Tay Sachs disease | 9 | 5.7 |
| Hunter syndrome | 6 | 3.8 |
| Lesch-Nyhan disease | 4 | 2.5 |
| Sandhoff disease | 3 | 1.9 |
| Hurler Scheie | 2 | 1.3 |
| Niemann-Pick B | 2 | 1.3 |
| Alpha mannosidosis | 1 | 0.6 |
| GM1 gangliosidosis | 1 | 0.6 |
| I-cell disease | 1 | 0.6 |
| Maroteaux-Lamy syndrome | 1 | 0.6 |
| Pelizaeus-Merzbacher disease | 1 | 0.6 |
| Performance status < 80 | 66 | 41.5 |
| Recipient CMV serology negative | 128 | 80.5 |
| HLA match | ||
| 6/6 | 7 | 4.4 |
| 5/6 | 75 | 47.2 |
| 4/6 | 73 | 45.9 |
| 3/6 | 4 | 2.5 |
| ABO matching mismatched | 71 | 44.7 |
| Recipient sex female | 62 | 39.0 |
| Unit sex female | 80 | 50.3 |
| Sex matching (recipient/unit) | ||
| F/F | 32 | 20.1 |
| F/M | 30 | 18.9 |
| M/M | 49 | 30.8 |
| M/F | 48 | 30.2 |
| Recipient race white | 133 | 83.7 |
| Donor race white | 126 | 83.4 |
| Race matching (recipient/unit) | ||
| White/white | 113 | 74.8 |
| White/other | 15 | 9.9 |
| Other/other | 10 | 6.6 |
| Other/white | 13 | 8.6 |