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Table 2 Values of fixed effects and variabilities for the final model of 4 biomarkers without covariates

From: Modeling changes in biomarkers in Gaucher disease patients receiving enzyme replacement therapy using a pathophysiological model

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  
  1. 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.