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Table 4 Baseline levels and slope changes* of the biomarkers during enzyme-replacement therapy: impact of covariates

From: Bone events and evolution of biologic markers in Gaucher disease before and during treatment

Biomarker

Covariate(s)

N

Baseline

P

CVa

Slope of % decreaseb

P

CVa

Platelets

        
 

No splenectomy

38

108,850 (/mm3)

 

56.5%

7 035 (/mm3/yr)

 

80.7%

 

Splenectomy

20

309,380 (/mm3)

  

-882 (/mm3/yr)

  
 

Effect of splenectomy

  

<0.0001

  

0.0007

 

Chitotriosidase

       

ND

 

No splenectomy-N370S

6

14,721 nmol/mL/h

 

9.8%

-38% (/yr)

  
 

No splenectomy-other genotype

22

3,581 nmol/mL/h

  

-14% (/yr)

  
 

Splenectomy-N370S

2

8,283 nmol/mL/h

  

-27% (/yr)

  
 

Splenectomy-other genotype

11

2,015 nmol/mL/h

  

-2% (/yr)

  
 

Effect of splenectomy

  

0.19

  

0.015

 
 

Effect of genotype

  

0.006

  

0.018

 

Ferritin

 

47

382 ng/L

 

15.4%

-14% (/yr)

 

83.6%

ACE

        
 

Diagnosis before 1991

35

90.3 (IU/L)

 

25.5%

-7% (/yr)

 

190.1%

 

Diagnosis in 1991 and after

11

31.7 (IU/L)

  

4% (/yr)

  
 

Effect of diagnosis data

  

0.020

  

0.055

 

TRAP

        
 

No splenectomy-diagnosis >15 yr

10

4.2 (IU/L)

 

3.6%

-4% (/yr)

 

ND

 

No splenectomy-diagnosis 15 yr

13

6.6 (IU/L)

  

-9% (/yr)

  
 

Splenectomy-diagnosis >15 yr

5

3.5 (IU/L)

  

3% (/yr)

  
 

Splenectomy-diagnosis <15 yr

8

5.4 (IU/L)

  

-2% (/yr)

  
 

Effect of splenectomy

  

0.32

  

0.004

 
 

Effect of age at diagnosis

  

0.016

  

0.057

 
  1. *Estimated with linear-mixed models under treatment. aCoefficient of variation of interpatient variability. bWhen a log-linear model was used, the evolution is expressed as the percent change of the slope (all markers except platelets). ND, not determined.