TABLE I.
Patient Characteristics at Baseline
| ITT population (n = 40) | |
|---|---|
| Age, years, median (range) | 37 (9–71); 22.5% <18 |
| Gender, n (%) | 18 (45) male/22 (55) female |
| Clinical parameters, median (range) | |
| Hemoglobin concentration, g/dL | 13.8 (10.4–16.5) |
| Platelet count, × 109/L | 162 (29–399) |
| Spleen volume, MNa | 2.5 (1.0–16.0) |
| Liver volume, MNb | 0.8 (0.6–1.6) |
| Biomarkers, median (range) | |
| Chitotriosidase activity, nmol/mL/h (n = 39)c,d | |
| Wild-type (n = 26) | 3072 (404–30,785) |
| Heterozygotes (n = 13) | 2651 (54–6929) |
| CCL18 level, ng/mL | 325 (49–1582) |
| Prior imiglucerase use, months, median (range) | 67.0 (22.0–191.5) |
| Anti-imiglucerase antibody-positive prior to receiving velaglucerase alfa, n (%) | 3 (8) |
ITT, intent-to-treat; MN, multiples of normal.
a
In 36 patients with spleen intact; four patients had undergone a splenectomy prior to enrollment. A normal spleen volume is 0.2% of body weight.
b
A normal liver volume is 2.5% of body weight.
c
One patient had two copies of the 24 bp duplication and was therefore deficient in chitotriosidase activity.
d
Subjects heterozygous for the chitotriosidase gene typically have lower levels of chitotriosidase activity than subjects with the wild-type genotype; thus data for these subgroups are given separately.