Table 1 Treatment Profiles for all Patients
Laronidase Regimen | ||||||
|---|---|---|---|---|---|---|
0.58 mg/kg Weekly | 1.2 mg/kg Every other week | |||||
Patient ID | MPS I Phenotype | Age at Dx | Age at Start | Duration | Age at Start | Duration |
years | ||||||
1 | H | 2.42 | 4.25 | 1.66 | 6.00 | 7.75 |
2 | H | 1.17 | 1.83 | 4.33 | 6.17 | 6.33 |
3 | H | 1.67 | 2.08 | 1.83 | 3.92 | 1.00 |
Mean (SD) | 1.75 (0.63) | 2.72 (1.33) | 2.61 (1.49) | 5.36 (1.25) | 5.03 (3.56) | |
Median (Range) | 1.67 | 2.08 | 1.83 | 6.00 | 6.33 | |
(1.17-2.42) | (1.83-4.25) | (1.66-4.33) | (3.92-6.17) | (1.00-7.75) | ||
4 | H-S | 1.92 | 2.42 | 1.58 | 4.00 | 2.00 |
5 | H-S | 2.50 | 5.08 | 1.50 | 9.92 | 2.08 |
6 | H-S | 5.17 | 8.33 | 1.50 | 13.00 | 2.92 |
7 | H-S | 2.00 | 21.00 | 4.00 | 25.00 | 9.00 |
8 | H-S | 7.25 | 9.42 | 2.70 | 12.33 | 1.10 |
9 | H-S | 5.00 | 6.75 | 1.17 | 7.91 | 2.00 |
10 | H-S | 15.92 | 17.17 | 1.58 | 18.75 | 6.75 |
11 | H-S | 3.25 | 4.75 | 1.33 | 6.08 | 5.42 |
12 | H-S | 7.50 | 12.08 | 1.25 | 13.33 | 6.42 |
Mean (SD) | 5.61(4.4) | 9.67 (6.11) | 1.85 (0.92) | 12.26 (6.49) | 4.19 (2.77) | |
Median (Range) | 5.0 | 8.33 | 1.5 | 12.33 | 2.92 | |
(1.92–15.92) | (2.42–21.0) | (1.33–4.0) | (4.0–25.0) | (1.1–9.0) | ||
13 | S | 5.00 | 6.00 | 2.00 | 8.00 | 6.00 |
14 | S | 6.00 | 6.33 | 3.42 | 9.75 | 2.75 |
15 | S | 7.00 | 10.00 | 3.00 | 13.00 | 6.00 |
16 | S | 12.00 | 12.25 | 3.42 | 15.67 | 2.75 |
17 | S | 15.92 | 20.08 | 1.59 | 21.67 | 6.33 |
18 | S | 10.00 | 19.00 | 5.00 | 24.00 | 3.67 |
19 | S | 14.00 | 18.00 | 5.00 | 23.00 | 3.67 |
20 | S | 20.00 | 21.00 | 4.00 | 25.00 | 5.00 |
Mean (SD) | 11.24 (5.25) | 14.08 (6.20) | 3.43 (1.25) | 17.51 (6.76) | 4.52 (1.49) | |
Median (Range) | 11.00 | 15.13 | 3.42 | 18.67 | 4.34 | |
(5.0–20.0) | (6.0–21.0) | (1.59–5.00) | (8.0–25.0) | (2.75–6.33) | ||