|
Fixed effects
|
Estimates
|
RSE (%)
|
P-value
|
|---|
|
C0F ( μg/L)
|
603
|
9
| |
|
exp(β_ splen)
|
1.9
|
24
|
10−6
|
|
exp(β_ < 15)
|
0.3
|
14
|
< 10−10
|
|
C0C (nmol/h.mL)
|
8140
|
12
| |
|
C0H (g/dL)
|
11.6
|
1
| |
|
exp(β_ man)
|
1.1
|
40
|
0.01
|
|
exp(β_ < 15)
|
0.9
|
19
|
10−8
|
|
C0P (/mm3)
|
68400
|
4
| |
|
exp(β_ splen)
|
2.7
|
8
|
< 10−10
|
|
exp(β_ < 15)
|
1.4
|
24
|
10−6
|
|
rF
|
0.3
|
13
| |
|
exp(β_ splen)
|
0.5
|
29
|
0.0005
|
|
exp(β_ man)
|
1.7
|
32
|
0.002
|
|
rC
|
0.1
|
14
| |
|
rH
|
1.2
|
1
| |
|
exp(β_ splen)
|
0.9
|
25
|
10−5
|
|
rP
|
2.2
|
4
| |
|
exp(β_ splen)
|
0.7
|
19
|
10−8
|
|
k (years−1)
|
1.4
|
11
| |
|
Variabilities
| | | |
|
ω_ C0F
|
63
|
8
| |
|
ω_C0C
|
122
|
7
| |
|
ω_ C0H
|
12
|
6
| |
|
ω_C0P
|
40
|
6
| |
|
ω_rF
|
70
|
10
| |
|
ω_rC
|
113
|
10
| |
|
ω_rH
|
9
|
9
| |
|
ω_rP
|
31
|
9
| |
|
ω_k
|
97
|
10
| |
|
σ_F
|
29
|
4
| |
|
σ_C
|
61
|
4
| |
|
σ_H
|
7
|
2
| |
|
σ_P
|
20
|
2
| |
- 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 covariates splenectomy, <15 indicates patients under 15 years of age and man indicates the covariate sex. ω: standard deviation of inter-individual variability, σ: standard deviation of proportional residual error. RSE: relative standard error.
