Table 6.
Cumulative ERT doses (units/kg) after 1, 2, and 5 years for GD1 patients with marked, moderate and mild DS3 baseline scores and treatment outcomes as determined by “minimal clinically important improvement” defined as a decrease of 3.1 from the baseline score for evaluable patients with marked and moderate baseline disease and 2.0 for patients with mild baseline disease
| Baseline DS3 severity category | Number (%) of patients with clinically significant change in DS3 score | Mean (SD) cumulative ERT dose units/kg | ||||
|---|---|---|---|---|---|---|
| Year 1 | Year 2 | Year 5 | Year 1 | Year 2 | Year 5 | |
| Marked | 16/46 (34.8) | 19/40 (47.5) | 37/49 (75.5) | |||
| ΔDS3 ≥ 3.1 | 16 | 19 | 37 | 991 (447) | 2354 (1136) | 5681 (2277) |
| ΔDS3 ≤ 3.1 | 30 | 21 | 12 | 1249 (477) | 2147 (857) | 5163 (1775) |
| Moderate | 6/42 (14.3) | 6/39 (15.4) | 9/44 (20.5) | |||
| ΔDS3 ≥ 3.1 | 6 | 6 | 9 | 880 (238) | 1835 (668) | 3770 (1239) |
| ΔDS3 ≤ 3.1 | 36 | 33 | 35 | 1064 (391) | 1854 (762) | 4809 (2049) |
| Mild | 2/17 (11.8) | 0/16 (0.0) | 2/16 (12.5) | |||
| ΔDS3 ≥ 2.0 | 2 | 0 | 2 | 1170 (552) | NA | 5850 (2758) |
| ΔDS3 ≤ 2.0 | 15 | 16 | 14 | 1131 (450) | 2188 (925) | 5478 (2493) |