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Table 2 HQ-CT Change from Baseline at Week 26 Subgroup Analyses Comparing C601/C602 Cohort to PATH for PWS Cohort

From: Behavioral changes in patients with Prader-Willi syndrome receiving diazoxide choline extended-release tablets compared to the PATH for PWS natural history study

 

Number of participants

LS Mean (95% CI)

LS Mean Difference

(95% CI)*

P-value

Subgroup

C601/ C602

PATH

C601/C602

PATH

Age

< 18

91

129

-8.7 (-10.1, -7.3)

-3.0 (-4.2, -1.8)

-5.7 (-7.6, -3.8)

< 0.001

≥ 18

17

100

-7.4 (-10.1, -4.7)

-1.5 (-2.6, -0.3)

-6.0 (-8.9, -3.0)

< 0.001

Sex

Female

63

119

-8.2 (-9.8, -6.6)

-2.9 (-4.1, -1.8)

-5.3 (-7.4, -3.3)

< 0.001

Male

45

110

-8.5 (-10.4, -6.5)

-1.8 (-3.1, -0.6)

-6.6 (-9.0, -4.3)

< 0.001

PWS Subtype

Deletion

69

113

-9.2 (-10.8, -7.7)

-2.1 (-3.3, -0.9)

-7.1 (-9.1, -5.1)

< 0.001

Non-Deletion

39

82

-7.2 (-9.4, -5.0)

-2.8 (-4.3, -1.4)

-4.3 (-7.1, -1.6)

0.002

Growth Hormone Use

Both Visits

93

130

-8.6 (-10.1, -7.2)

-2.8 (-4.0, -1.6)

-5.8 (-7.7, -3.9)

< 0.001

Untreated

15

87

-6.8 (-9.8, -3.8)

-1.9 (-3.1, -0.6)

-4.9 (-8.2, -1.7)

0.004

Region

United States

86

202

-9.2 (-10.6, -7.8)

-2.6 (-3.4, -1.7)

-6.6 (-8.3. -5.0)

< 0.001

Ex-United States

22

27

-5.4 (-8.3, -2.4)

-0.8 (-3.5, 1.8)

-4.5 (-8.6, -0.5)

0.028

Baseline HQ-CT

Above Median (≥ 18)

75

103

-10.7 (-12.3, -9.1)

-2.6 (-3.9, -1.2)

-8.1 (-10.3, -6.0)

< 0.001

Below Median (< 18)

33

126

-4.4 (-6.3, -2.5)

-1.8 (-2.8, -0.9)

-2.6 (-4.7, -0.4)

0.019

  1. *Difference in LS Means and 95% confidence intervals are derived from an ANCOVA model with baseline value as a covariate