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

  • No new safety concerns were identified.3

  • No serious adverse events were related to treatment with VPRIV.3

  • No serious infusion-related adverse events occurred.3

  • No patient discontinued the study due to an adverse event.3

  • 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

  • Adverse reactions more commonly seen in pediatric patients compared with adult patients include (>10% difference): rash, prolonged aPTT, and pyrexia.1

  • 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

+24.3%
(n=8)3

PLATELET COUNT

Median platelet count at baseline:* 116 × 109/L3
Mean change at 24 months: 79.13 × 109/L3

+93.4%
(n=8)3

-66.3%
(n=7)3

SPLEEN VOLUME

Median spleen volume at baseline:* 14.3 MN3
Mean change at 24 months: −2.55% of body weight3

-27.5%
(n=8)3

LIVER VOLUME

Median liver volume at baseline:* 1.6 MN3
Mean change at 24 months: −1.14% of body weight3

-100

-80

-60

-40

-20

0

20

40

60

80

100

*Baseline is defined as before the first dose of VPRIV in Studies 032 and 039

MN, multiples of normal.