Patient characteristics (N = 30) and number of samples of DBS
| Characteristic . | Value . | |||
|---|---|---|---|---|
| Male sex | 21 (70%) | |||
| Age at pre-HCT collection, median (range), y | 1.1 (0.1-4.7) | |||
| Age at transplantation, median (range), y | 1.3 (0.2-4.8) | |||
| Enzyme activity level post-HCT in normal range | 28 (93%) | |||
| MPS type | DBS (n) | |||
| Pre | 1-year FU | 3-year FU | 10-year FU | |
| MPS | 22 | 14 | 15 | 10 |
| MPS 1 | 16 | 14 | 15 | 10 |
| MPS 2 | 2 | |||
| MPS type 3 | 2 | |||
| MPS 6 | 2 | |||
| Characteristic . | Value . | |||
|---|---|---|---|---|
| Male sex | 21 (70%) | |||
| Age at pre-HCT collection, median (range), y | 1.1 (0.1-4.7) | |||
| Age at transplantation, median (range), y | 1.3 (0.2-4.8) | |||
| Enzyme activity level post-HCT in normal range | 28 (93%) | |||
| MPS type | DBS (n) | |||
| Pre | 1-year FU | 3-year FU | 10-year FU | |
| MPS | 22 | 14 | 15 | 10 |
| MPS 1 | 16 | 14 | 15 | 10 |
| MPS 2 | 2 | |||
| MPS type 3 | 2 | |||
| MPS 6 | 2 | |||
FU, follow-up.