FOR YOUR TREATMENT-EXPERIENCED PATIENTS
Study 034: 12-Month Switch Study (n=41)1
Study 034: 12-MONTH SWITCH STUDY OBJECTIVES
imiglucerase for ≥30 months
(no washout)
15–60 U/kg EOW (n=40)
-
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
Over 12 months, stability vs. baseline was maintained in all four clinical parameters.1
mean change from baseline1
12 months VPRIV 15–60 U/kg EOW
Hemoglobin
concentration
-
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
12 months VPRIV 15–60 U/kg EOW
Hemoglobin
concentration
-
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
Platelet count
-
Median platelet count at baseline: 162 × 109/L1
-
Mean change at 12 months: +7.0%
(within the pre-defined efficacy criterion of ±20%)1
Spleen volume
-
Median spleen volume at baseline: 2.5 MN1
-
Mean change at 12 months: −5.6%
(within the pre-defined efficacy criterion of ±15%)1
Liver volume
-
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.
FOR YOUR TREATMENT-EXPERIENCED PATIENTS
Study 044: 5-Year Long-Term Extension Study (n=38)1
Study 044: 5-Year Long-Term Extension Study OBJECTIVES
Overall VPRIV (n=40)
EOW (n=38)
-
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
Patients previously treated with imiglucerase maintained stability vs.
baseline
for up to 5 years after switching to VPRIV.1,2
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
