Table 3.
Estimated individual characteristics of biomarker response to ERT for the 197 patients
| Median (range) at steady state | Normalization n (%) | |
|---|---|---|
| Half-life of response (years) |
0.5 (0.01-3.1) |
|
| Time to 95% of response (years) |
2.1 (0.4-13.4) |
|
| Ratio (level at steady state/level at ERT initiation) |
|
|
| Ferritin (%) |
38.1 (2.1-101.5) |
|
| Chitotriosidase (%) |
10.1 (0.8-116.7) |
|
| Hemoglobin (%) |
117.3 (88.6-173.1) |
|
| Platelets (%) |
200.4 (83.5-437.8) |
|
| Predicted value at steady state |
|
|
| Ferritin (μg/L) |
202.0 (16.5-1280.1) |
115 (58.4) |
| Chitotriosidase (nmol/h.mL) |
820 (8–18183) |
131 (66.5) |
| Hemoglobin (g/dL) |
13.5 (8.1-17.2) |
174 (88.3) |
| Platelets (/mm3) | 172 000 (48 000–526 000) | 126 (64.0) |
Normalization was defined by: ferritin <250 μg/L, chitotriosidase <100 nmol/mL/h, hemoglobin >12 g/dL, platelet count >150 000/mm3.