Table 1.
Characteristics of the subjects according to MPS type.
| Overall (n = 76) | MPS I (n = 27) | MPS II (n = 22) | MPS IVA (n = 19) | MPS VI (n = 8) | p-value | |
|---|---|---|---|---|---|---|
| Sex (n) | M: 46 F: 30 |
M: 12 F: 15 |
M: 22 F: 0 |
M: 8 F: 11 |
M: 4 F: 4 |
<0.001† |
| Age (years) | 11.3 (12.7) | 10.2 (20.4) | 11.8 (15.5) | 10.1 (8.5) | 16.2 (8.3) | 0.491 |
| Number of adults (n) | 23/76 (30%) | 8/27 (29%) | 5/22 (23%) | 7/19 (37%) | 3/8 (38%) | 0.757 |
| Weight (kg) | 25.6 (21.0) | 23.5 (23.4) | 28.0 (36.5) | 21.0 (16.6) | 22.3 (14.9) | 0.103 |
| Height (cm) | 111.0 (35.0) | 108.0 (8.4) | 129.0 (36.0) | 99.5 (19.7) | 106.5 (18.4) | 0.003‡ |
| BSA (m2) | 0.9 (0.5) | 0.9 (0.6) | 1.0 (0.7) | 0.8 (0.3) | 0.8 (0.4) | 0.057 |
| Treated with ERT (n) | 47/76 | 16/27* | 15/22 | 8/19 | 8/8 | 0.029§ |
| Age at the start of ERT (years) | 8.3 (10.1) | 4.0 (15.4) | 6.6 (8.9) | 8.3 (8.1) | 9.7 (2.3) | 0.538 |
| Time on ERT (months) | 41.0 (77.0) | 52.0 (79.5) | 64.0 (76.0) | 22.5 (22.3) | 73.5 (79.0) | 0.317 |
Continuous variables are reported as median and interquartile range.
*
Four patients who received ERT as a concomitant therapy for HSCT are not included in the ERT-treated group. Statistical analysis with chi-squared and partitioning for categorical variables and Kruskal-Wallis test with Dunn's post-hoc test for continuous variables.
†
MPS II is different from other MPS types.
‡
MPS IVA is different from MPS II.
§
MPS IVA is different from MPS VI.