|
Fixed effects
|
Estimates
|
RSE (%)
|
P-value
|
|---|
|
C0F ( μg/L)
|
598
|
8
| |
|
C0C (nmol/h.mL)
|
7920
|
11
| |
|
C0H (g/dL)
|
11.6
|
1
| |
|
C0P (/mm3)
|
74300
|
4
| |
|
exp(β_ splen)
|
2.5
|
8
|
< 10−10
|
|
rF
|
0.4
|
8
| |
|
rC
|
0.1
|
14
| |
|
rH
|
1.2
|
1
| |
|
rP
|
2.1
|
4
| |
|
exp(β_ splen)
|
0.5
|
19
|
10−7
|
|
k (years−1)
|
1.4
|
10
| |
|
Variabilities
| | | |
|
ω_ C0F
|
83
|
8
| |
|
ω_C0C
|
120
|
7
| |
|
ω_ C0H
|
13
|
6
| |
|
ω_C0P
|
42
|
6
| |
|
ω_rF
|
67
|
9
| |
|
ω_rC
|
117
|
10
| |
|
ω_rH
|
10
|
10
| |
|
ω_rP
|
31
|
10
| |
|
ω_k
|
90
|
10
| |
|
σ_F
|
30
|
4
| |
|
σ_C
|
61
|
4
| |
|
σ_H
|
7
|
2
| |
|
σ_P
|
20
|
3
| |
- C0 is initial concentration, r amplitude of variation and k the rate of normalization during ERT. F corresponds to ferritin, C to chitotriosidase, H to hemoglobin and P to platelets. The P-value is for the Wald test. Exp β represents the effect of the covariates. Splen corresponds to the covariate splenectomy. ω: standard deviation of inter-individual variability, σ: standard deviation of proportional residual error. RSE: relative standard error. For example, baseline ferritin concentration is C0F =598 μg/L, and the estimated corrected value at steady state of ERT is C0F × r
F
= 179.4 μg/L. Baseline platelet count in non-splenectomized patients is C0P = 74,300/mm3 and C0P exp(β _ splen) = 185,750 /mm3 in splenectomized patients. The estimated corrected platelet count at steady state of ERT in splenectomized patients is (C0P × exp(β _ splen)) × (r0 × exp(β _ splen)) = 195,037 /mm3.
