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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

From: Evaluation of disease burden and response to treatment in adults with type 1 gaucher disease using a validated disease severity scoring system (DS3)

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)