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Table 4 Change from baseline in cardiovascular imaging endpoints*

From: Natural history comparison study to assess the efficacy of elamipretide in patients with Barth syndrome

 

TAZPOWER Patients (n = 12)

 

Natural History Controls (n = 12)

 

Slope Model Analysis

    
 

LV stroke volume (mL)

   
  

Mean (SD) change from baseline; range

3.44 (5.26); − 1.7, 18.3

 − 0.26 (2.66); − 4.9, 3.7

  

P value

0.0407

 
 

LV end-diastolic volume (mL)

   
  

Mean (SD) change from baseline; range

4.89 (7.25); − 1.5, 24.7

0.91 (6.15); − 12.3, 11.2

  

P value

0.1609

 
 

LV end-systolic volume (mL)

   
  

Mean (SD) change from baseline; range

1.46 (2.26); − 2.0, 6.4

1.17 (4.29); − 7.4, 9.1

  

P value

0.8417

 

Mixed Model Analysis

    
 

LV stroke volume (mL)

   
  

LS mean change from baseline

1.92 − 4.80

 
  

LS mean difference (95% CI), P value

6.72 (2.75, 10.69); P = 0.0020

 
 

LV end-diastolic volume (mL)

   
  

LS mean change from baseline

2.08 − 2.66

 
  

LS mean difference (95% CI), P value

4.75 (− 2.72, 12.22); P = 0.2006

 
 

LV end-systolic volume (mL)

   
  

LS mean change from baseline

0.52 1.28

 
  

LS mean difference (95% CI), P value

 − 0.77 (− 7.13, 5.60); P = 0.8047

 
  1. *Indexed to baseline body surface area
  2. CI Confidence interval, LS Least squares, LV left ventricular, SD standard deviation