Table 4. Plasma Lyso-Gb3 quantification.
| N215S | non-N215S | |||
|---|---|---|---|---|
| Overall | N = 84 | N = 167 | ||
| Males | Females | Males | Females | |
| N (%) | 25 (43.1) | 33 (56.9) | 39 (23.4) | 72 (43.1) |
| Lyso-Gb3 (nmol/L) | ||||
| ERT naïve, n (%) | 13 (52) | 26 (78.8) | 9 (23.1) | 36 (50) |
| Mean (SD) | 6.7 (2.1) | 1.5 (0.9) | 74.3 (42.2) | 6.8 (4.6) |
| Median (range) | 6.6 (3–9.8) | 1.2 (0.4–4.1) | 77.4 (20.9–126.9) | 6 (0.4–16.5) |
| During ERT, n (%) | 12 (48) | 7 (21.2) | 30 (76.9) | 36 (50) |
| Mean (SD) | 6.3 (3.3) | 1.7 (1.5) | 37.9 (21.3) | 6.7 (3.4) |
| Median (range) | 4.9 (2.6–13.6) | 1.1 (0.4–4.9) | 33.8 (1.4–113.2) | 5.9 (0.4–16.7) |
Lyso-Gb3 = Globotriaosylsphingosine. Upper reference limit Lyso-Gb3 = 0.6.