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.
