Primary: To evaluate the safety of every-other-week dosing of VPRIV in patients with type 1 Gaucher disease who were previously treated with imiglucerase.1
Secondary: Absolute change from baseline to 12 months in hemoglobin concentration; and percentage change from baseline to 12 months in platelet count, and spleen and liver volumes normalized by body weight.1
mean change from baseline1
Median hemoglobin concentration at baseline: 13.8 g/dL1
Mean change at 12 months: −0.1 g/dL
(within the pre-defined efficacy criterion of ±1 g/dL)1
mean change from baseline1
Median hemoglobin concentration at baseline: 13.8 g/dL1
Mean change at 12 months: −0.1 g/dL
(within the pre-defined efficacy criterion of ±1 g/dL)1
Median platelet count at baseline: 162 × 109/L1
Mean change at 12 months: +7.0%
(within the pre-defined efficacy criterion of ±20%)1
Median spleen volume at baseline: 2.5 MN1
Mean change at 12 months: −5.6%
(within the pre-defined efficacy criterion of ±15%)1
Median liver volume at baseline: 0.8 MN1
Mean change at 12 months: 0.0%
(within the pre-defined efficacy criterion of ±15%)1
EOW, every other week; MN, multiples of normal.
Primary: To evaluate the long-term safety of VPRIV treatment. 1
Secondary: To evaluate the effects of treatment on hemoglobin concentration, platelet count, and liver and spleen volumes.1
MEAN INCREASE IN HEMOGLOBIN CONCENTRATION1
12-month data – baseline:* 13.8 g/dL; mean change from baseline: –0.1 g/dL
(within the pre-defined efficacy criterion of ±1 g/dL)3
24-month data – baseline:* 13.82 g/dL; mean change from baseline: –0.05 g/dL1,4
MEAN INCREASE IN PLATELET COUNT1
12-month data – baseline:* 162 × 109/L; mean change from baseline: +7.0%
(within the pre-defined efficacy criterion of ±20%)3
24-month data – baseline:* 164.5 × 109/L; mean change from baseline: 9.03 × 109/L1,4
MEAN DECREASE IN SPLEEN VOLUME1
12-month data – baseline:* 2.5 MN; mean change from baseline: –5.6% (within the pre-defined efficacy criterion of ±15%)3
24-month data – baseline:* 0.821% of body weight; mean change from baseline: –0.110% of body weight1,4
MEAN DECREASE IN LIVER VOLUME1
12-month data – baseline:*
0.8 MN; mean change from baseline: 0.0%
(within the pre-defined efficacy criterion of ±15%)3
24-month data – baseline:* 2.062% of body weight; mean change from baseline: –0.026% of body weight1,4
*Baseline is defined as before the first dose of VPRIV in Study 034
CI, confidence interval; MN, multiples of normal; EOW, every other week