Table 3.
Baseline and follow-up data of aortic root diameter of 16 patients with MPS who received enzyme replacement therapy for 2.0–16.2 years.
| No. | MPS Type | Gender | Age at Baseline (Years) | Age at Follow-Up (Years) | ERT Duration (Years) | AoD (cm) | AoD (z Score) | Change (z Score) |
||
|---|---|---|---|---|---|---|---|---|---|---|
| Baseline | Follow-Up | Baseline | Follow-Up | |||||||
| 1 | I (H/S) | F | 0.7 | 7.7 | 7.0 | 1.70 | 1.90 | 2.60 | 0.60 | −2.00 |
| 2 | I (H/S) | F | 1.1 | 6.9 | 5.8 | 1.40 | 2.00 | −0.10 | 1.20 | 1.30 |
| 3 | I (H/S) | M | 18.2 | 20.2 | 2.0 | 1.63 | 1.88 | −2.00 | −0.61 | 1.39 |
| 4 | II (M) | M | 13.2 | 25.7 | 12.4 | 2.28 | 2.50 | 1.12 | 0.52 | −0.60 |
| 5 | II (M) | M | 14.8 | 23.3 | 8.5 | 2.54 | 3.10 | 2.51 | 2.74 | 0.23 |
| 6 | II (M) | M | 15.4 | 17.7 | 2.2 | 2.85 | 2.14 | 4.47 | 0.29 | −4.18 |
| 7 | II (M) | M | 17.5 | 29.8 | 12.3 | 2.80 | 2.90 | 3.00 | 2.00 | −1.00 |
| 8 | II (M) | M | 18.1 | 26.6 | 8.6 | 3.46 | 3.70 | 4.07 | 4.06 | −0.01 |
| 9 | IVA | M | 1.1 | 7.4 | 6.3 | 1.56 | 2.40 | 0.52 | 4.18 | 3.66 |
| 10 | IVA | M | 2.3 | 6.6 | 4.3 | 1.64 | 1.80 | 0.92 | 1.20 | 0.28 |
| 11 | IVA | M | 7.2 | 10.5 | 3.3 | 2.08 | 2.30 | 2.29 | 2.39 | 0.10 |
| 12 | IVA | F | 14.8 | 19.0 | 4.2 | 2.42 | 2.40 | 4.29 | 3.59 | −0.70 |
| 13 | IVA | F | 16.8 | 23.2 | 6.4 | 2.80 | 2.60 | 5.94 | 3.39 | −2.55 |
| 14 | VI | M | 7.6 | 21.6 | 14.0 | 2.06 | 2.20 | 3.57 | 1.83 | −1.74 |
| 15 | VI | M | 8.3 | 22.4 | 14.1 | 2.38 | 2.80 | 5.07 | 4.18 | −0.89 |
| 16 | VI | M | 11.7 | 27.9 | 16.2 | 2.67 | 2.70 | 4.44 | 3.94 | −0.50 |
| Mean | 10.6 | 18.5 | 8.0 | 2.27 | 2.46 | 2.67 | 2.22 | −0.45 | ||
MPS, mucopolysaccharidosis; ERT, enzyme replacement therapy; AoD, aortic root diameter; H/S: Hurler–Scheie; MPS II (M), mild form; F, female; M, male. p-value: p = 0.501.