TREATMENT-NAÏVE* STUDIES
VPRIV is a first-line option for your treatment-naïve patients1,2
*Treatment-naïve: all patients were naïve to ERT at the start of the core studies, although they received different ERTs for the first 9 months3
5-YEAR LONG-TERM EXTENSION: STUDY 044
STUDY 044 DESIGN (N=57)3
STUDIES 032 & 039 OVERALL VPRIV
(n=41)
STUDY 039 IMIGLUCERASE
TO VPRIV
(n=16)
VPRIV
60 U/KG EOW
(N=57)†
PRIMARY OBJECTIVE
To evaluate the long-term safety of VPRIV treatment.3
SECONDARY OBJECTIVES
To evaluate the effects of treatment on hemoglobin concentration, platelet count, and liver and spleen volumes.3
†One patient from each group in Study 039 did not participate in Study 044
At 24 months of VPRIV treatment, patients demonstrated improvements from baseline in clinical parameters. Improvements were maintained for up to 5 years.1,3
MEAN INCREASE IN HEMOGLOBIN
CONCENTRATION3
12-month data – baseline‡: 11.0 g/dL;
mean change from baseline: +2.2 g/dL ± 0.2 (SE)4
24-month data – mean change from baseline: +2.8 g/dL3,4
MEAN INCREASE IN PLATELET COUNT3
12-month data – baseline‡: 108.6 × 109/L;
mean change from baseline: +78 × 109/L ± 12 (SE)4
24-month data – mean change from baseline: +87.9 × 109/L4
MEAN DECREASE IN SPLEEN VOLUME3
12-month data – baseline‡: 3.8% of body weight;
mean change from baseline: −2.1% ± 0.3 of body weight (SE)4
24-month data – mean change from baseline: −2.7% of body weight4
MEAN DECREASE IN LIVER VOLUME3
12-month data – baseline‡: 4.0% of body weight;
mean change from baseline: −0.79% ± 0.15 of body weight (SE)4
24-month data – mean change from baseline −1.2% of body weight4
†Baseline is defined as before the first dose of VPRIV in Studies 032 and 039
BL, baseline; EOW, every other week; SE, standard error
VPRIV’s safety and efficacy profile was similar in pediatric (ages 4–17 years) and adult patients.1
In a subset of 24 pediatric patients from Study 044, safety results were consistent with those of the overall population (N=95):
- No new safety concerns were identified5
- No serious adverse events were related to treatment with VPRIV5
- No serious infusion-related adverse events occurred5
- No patient discontinued the study due to an adverse event5
- One pediatric patient tested positive for IgG anti-velaglucerase antibodies in Study
0445
– No apparent impact was noted on the efficacy of VPRIV as assessed by changes in hemoglobin concentration and platelet count5
- Adverse reactions more commonly seen in pediatric patients compared to adult patients include (>10% difference): rash, aPTT prolonged, and pyrexia1
- Safety and efficacy have not been established in children younger than 4 years1
Mean % change in 24 months in treatment-naïve pediatric patients
HEMOGLOBIN CONCENTRATION
Median hemoglobin concentration at baseline§: 10.75 g/dL5
Mean change at 24 months: +2.65 g/dL5
+24.3%
(n=8)5
PLATELET COUNT
Median platelet count at baseline§: 116 × 109/L5
Mean change at 24 months: +79.13 × 109/L5
+93.4%
(n=8)5
−66.3%
(n=7)5
SPLEEN VOLUME
Median spleen volume at baseline§: 14.3 MN5
Mean change at 24 months: −2.55% of body weight5
−27.5%
(n=8)5
LIVER VOLUME
Median liver volume at baseline§: 1.6 MN5
Mean change at 24 months: −1.14% of body weight5
Patients meeting pre-defined clinical parameters at 4 years.6
A post hoc analysis was conducted on 39 treatment-naïve patients, aged 6–62 years, from the Study 032 and 039 populations who enrolled in the 044 extension trial. This analysis assessed achievement of published pre-defined clinical parameters at baseline and yearly intervals after initiation of VPRIV for up to 4 years6
PRIMARY OBJECTIVE: To measure the number of patients who met these pre-defined clinical parameter expectations in hemoglobin and platelet concentration, and spleen and liver volumes6
Benefits of achieving clinical parameters for a patient’s long-term clinical outcomes have not been demonstrated, and composite analyses may be required to assess the effect of ERT on Gaucher disease, instead of individual assessments6
This analysis does not cover the primary or secondary objectives of the 044 extension trial (Hughes et al. publication). This was a post hoc analysis.
These are observational data, therefore, cautious interpretation is advised. The clinical significance of these data is unknown. There are no official treatment guidelines for type 1 Gaucher disease, therefore, clinical parameters were pre-defined by Pastores et al., based on medical literature data and the clinical experience of an international panel of physicians.6
Pre-defined clinical parameters:
Parameters met by:
HEMOGLOBIN CONCENTRATION6
- ≥11 g/L for females or patients 12 years and younger
- ≥12 g/dL for males or patients older than 12 years
19/20 patients (95%)
PLATELET COUNT6
- ≥100% increase if baseline is <60 × 109/L
- ≥100 × 109/L if baseline is ≥60 and <120 × 109/L
- ≥120 × 109/L if baseline is ≥120 × 109/L
20/20 patients (100%)
SPLEEN VOLUME6
- ≥50% decrease compared with baseline (or ≤8.0 MN)
16/16 patients (100%)
LIVER VOLUME6
- ≥30% decrease compared with baseline (or ≤1.5 MN)
15/16 patients (94%)
MN, multiples of normal
Over the 4 years, pre-defined clinical parameters were also maintained by 100% of patients who had already met these at baseline6
