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Table 4 Values of fixed effects and variabilities for the final model with significant 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)

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

 
  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 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.