Table 1.
Baseline Characteristics
| ITT population (n = 10) | Completer population (n = 8) | |
|---|---|---|
| Median agea, years (range) | 36.0 (19–63) | 39.5 (19–63) |
| Male sex, n (%) | 4 (40.0) | 4 (50.0) |
| Gaucher disease genotype, n (%) | ||
| N370S/N370S | 4 (40.0) | 3 (37.5) |
| N370S/L444P | 1 (10.0) | 1 (12.5) |
| N370S/Other | 5 (50.0) | 4 (50.0) |
| Chitotriosidaseb, n (%) | ||
| Not deficient | 9 (90.0) | 7 (87.5) |
| Deficient | 1 (10.0) | 1 (12.5) |
| Clinical activity variables, median (range) | ||
| Hemoglobin concentration, g/dL | 11.10 (9.8–12.9) | 12.00 (10.1–12.9) |
| Platelet count ×109/L | 62.25 (37–98.5) | 64.00 (37–98.5) |
| Spleen volumec, MN | 19.00 (11–32.5) | 19.00 (11–28) |
| Liver volume, MN | 1.760 (1.04–2.32) | 1.620 (1.04–2.32) |
| Chitotriosidase activityd, nmol/mL/h | 23,375 (7391–68,552) | 23,375 (12,191–66,804) |
| CCL18 level, ng/mL | 1978.5 (1837–5247) | 1923.0 (1837–5247) |
Baseline is defined as data collected at the baseline visit in TKT025.
a
Age at the time TKT025 informed consent was obtained.
b
Chitotriosidase deficient is defined as values below 5 nmol/mL/h.
c
One patient was excluded due to an intravascular metallic device which precluded accurate spleen evaluation.
d
One chitotriosidase deficient patient was excluded.
MN: multiple of normal.