Study 044: PEDIATRIC SUBSET
VPRIV’s safety and efficacy profile was similar in pediatric (ages 4–17) and adult patients1
*Not an actual patient.
*Not an actual patient.
In a subset of 24 pediatric patients from Study 044, safety results were consistent with those of the overall population (N=95):3
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No new safety concerns were identified.3
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No serious adverse events were related to treatment with VPRIV.3
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No serious infusion-related adverse events occurred.3
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No patient discontinued the study due to an adverse event.3
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One pediatric patient tested positive for IgG anti-velaglucerase antibodies in Study 044.3
– No apparent impact was noted on the efficacy of VPRIV as assessed by changes in hemoglobin concentration and platelet count.3
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Adverse reactions more commonly seen in pediatric patients compared with adult patients include (>10% difference): rash, prolonged aPTT, and pyrexia.1
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Efficacy and safety have not been established in children younger than 4 years.1
Mean % change in 24 months in treatment-naïve pediatric patients
HEMOGLOBIN CONCENTRATION
Median hemoglobin concentration at baseline:* 10.75 g/dL3
Mean change at 24 months: 2.65 g/dL3
PLATELET COUNT
Median platelet count at baseline:* 116 × 109/L3
Mean change at 24 months: 79.13 × 109/L3
SPLEEN VOLUME
Median spleen volume at baseline:* 14.3 MN3
Mean change at 24 months: −2.55% of body weight3
LIVER VOLUME
Median liver volume at baseline:* 1.6 MN3
Mean change at 24 months: −1.14% of body weight3
*Baseline is defined as before the first dose of VPRIV in Studies 032 and 039
MN, multiples of normal.
