Post hoc Clinical Parameters

Patients meeting pre-defined clinical parameters at 4 years2
Hemoglobin
Hemoglobin concentration2
Platelets
PLATELET COUNT2
Spleen
SPLEEN VOLUME2
Liver
LIVER Volume2

Pre-defined
clinical
parameters:

  • >11 g/L for females or patients 12 years and younger

  • >12 g/dL for males or patients older than 12 years

  • ≥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

  • ≥50% decrease
    compared with
    baseline (or ≤8.0 MN)

  • ≥30% decrease
    compared with
    baseline (or ≤1.5 MN)

Parameter
met by:

19/20 patients (95%)

20/20 patients (100%)

16/16 patients (100%)

15/16 patients (94%)

Pre-defined clinical parameters:

Hemoglobin
Hemoglobin concentration2
  • >11 g/L for females or patients 12 years and younger

  • >12 g/dL for males or patients older than 12 years

Platelet
PLATELET COUNT2
  • ≥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

Spleen
SPLEEN VOLUME2
  • ≥50% decrease
    compared with
    baseline (or ≤8.0 MN)

Liver
LIVER Volume2
  • ≥30% decrease
    compared with
    baseline (or ≤1.5 MN)

Parameter met by:

19/20 patients (95%)

20/20 patients (100%)

16/16 patients (100%)

15/16 patients (94%)

Over the 4 years, pre-defined clinical parameters were also maintained by 100% of patients who had already met these at baseline.2

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.2

MN, multiples of normal.

Exploratory Efficacy Objective — Bone Mineral Density (BMD) Z-Score

Efficacy of VPRIV in improving BMD Z-score was assessed as an exploratory efficacy objective
in treatment-naïve patients.2*
MEAN CHANGE OVER TIME IN LUMBAR SPINE BMD FROM BASELINE2†

Error bars show 95% CI. Clinical significance of these data is unknown.

Mean change, Z-score

At 24 months in Study 044:2

+0.62 SD

mean increase in BMD Z-score
in the lumbar spine (n=31)

Median BMD Z-score at baseline: -1.73 SD (-4.20, 0.78)2

Overall velaglucerase alfa

Overall velaglucerase alfa, no bisphosphonates

Imiglucerase-velaglucerase alfa

Imiglucerase-velaglucerase alfa, no bisphosphonates

Baseline was before the first dose in the initial trials

CI, confidence interval; SD, standard deviation.