Table 2.
Demographic and clinical characteristics
| Individuals with treatment history n=740 |
||
| Gender n (%) | ||
| Female | 362 (48.9) | |
| Male | 378 (51.1) | |
| MPS I phenotype n (%) | ||
| Severe | 424 (57.3) | |
| Attenuated | 291 (39.3) | |
| Undetermined/missing | 25 (3.4) | |
| Primary treatment | ||
|
ERT
(n=471) |
HSCT
n=269 |
|
| Age at diagnosis, year median (IQR) | 3.4 (1.2, 6.6) | 0.9 (0.6 to 1.2) |
| Age distribution, year n (%) | ||
| <5 310 (65.8) | <1 124 (46.1) | |
| 5–<10 93 (19.7) | 1–<2 126 (46.8) | |
| ≥10 68 (14.4) | ≥2 19 (7.1) | |
| Age at start of treatment, year median (IQR) | 6.1 (2.8 to 12.8) | 1.1 (0.7 to 1.4) |
| Age distribution, year n (%) | ||
| <5 203 (43.1) | <1 24 (46.1) | |
| 5–<10 113 (24.0) | 1–<2 126 (46.8) | |
| ≥10 155 (32.9) | ≥2 19 (7.1) | |
| HSCT treatment history | ||
| # HSCT | 305 | |
ERT, enzyme replacement therapy; HSCT, haematopoietic stem cell transplant.