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Table 4 Known group analysis

From: How does a preference-based generic health-related quality of life measure perform in patients with a rare disease? Measurement properties of the EQ-5D-Y proxy version among underage patients with neurofibromatosis type 1

Known groups

EQ-5D-Y utility

EQ-5D VAS

Gender, mean (SD)

  

Female (n = 79)

0.86 (0.16)

75.03 (21.06)

Male (n = 75)

0.90 (0.10)

75.76 (20.38)

Δ

-0.03

-0.73

ES (Cohen’s d)

-0.26 (-0.58, 0.06)

-0.04 (-0.35, 0.28)

Z

-0.98

-0.14

p value

0.33

0.89

Annual outpatient visit times, mean (SD)*

  

< 5 (n = 126)

0.90 (0.11)

75.77 (19.78)

>=5 (n = 28)

0.81 (0.20)

73.64 (24.59)

Δ

0.08

2.13

ES (Cohen’s d)

0.65 (0.24, 1.07)

0.10 (-0.31, 0.51)

Z

2.01

0.04

p value

0.04

0.84

Annual hospitalization times, mean (SD)*

  

0 (n = 113)

0.89 (0.13)

76.08 (20.54)

>=1 (n = 41)

0.86 (0.14)

73.46 (21.16)

Δ

0.02

2.62

ES (Cohen’s d)

0.17 (-0.19, 0.53)

0.13 (-0.23, 0.48)

Z

0.91

0.78

p value

0.36

0.44

PedsQL Physical Function score, mean (SD)

  

≤ 75 (n = 84)

0.82 (0.15)

66.31 (20.93)

> 75 (n = 70)

0.95 (0.07)

86.27 (14.12)

Δ

-0.13

-19.96

ES (Cohen’s d)

-1.11 (-1.45, -0.77)

-1.10 (-1.44, -0.76)

Z

-7.16

-6.22

p value

< 0.0001

< 0.0001

PedsQL Emotional Function score, mean (SD)

  

≤ 65 (n = 80)

0.83 (0.13)

67.43 (20.83)

> 65 (n = 74)

0.93 (0.11)

83.99 (16.77)

Δ

-0.10

-16.56

ES (Cohen’s d)

-0.85 (-1.18, -0.52)

-0.87 (-1.20, -0.54)

Z

-6.50

-5.27

p value

< 0.0001

< 0.0001

PedsQL Social Function score, mean (SD)

  

≤ 70 (n = 81)

0.83 (0.13)

68.91 (20.46)

> 70 (n = 73)

0.94 (0.11)

82.56 (18.52)

Δ

-0.11

-13.65

ES (Cohen’s d)

-0.90 (-1.23, -0.57)

-0.70 (-1.02, -0.37)

Z

-6.60

-4.50

p value

< 0.0001

< 0.0001

PedsQL School Function score, mean (SD)

  

≤ 65 (n = 85)

0.83 (0.15)

68.53 (21.16)

> 65 (n = 69)

0.94 (0.07)

83.83 (16.63)

Δ

-0.12

-15.30

ES (Cohen’s d)

-0.96 (-1.29, -0.62)

-0.79 (-1.12, -0.46)

Z

-6.18

-4.87

p value

< 0.0001

< 0.0001

PedsQL pain, mean (SD)

  

≤ 50 (n = 89)

0.83 (0.15)

69.15 (21.64)

> 50 (n = 65)

0.95 (0.07)

83.92 (15.77)

Δ

-0.12

-14.78

ES (Cohen’s d)

-0.96 (-1.30, -0.63)

-0.76 (-1.09, -0.43)

Z

-6.34

-4.63

p value

< 0.0001

< 0.0001

ZBI-22, mean (SD)

  

≤ 34.5 (n = 77)

0.93 (0.09)

82.70 (16.92)

> 34.5 (n = 77)

0.83 (0.15)

68.06 (21.57)

Δ

0.10

14.64

ES (Cohen’s d)

0.83 (0.50, 1.15)

0.76 (0.43, 1.08)

Z

5.44

4.47

p value

< 0.0001

< 0.0001

  1. *Information of annual outpatient visit times and hospitalization times in 2021 were collected
  2. Δ, the difference between the two groups
  3. ES, effect size, is expressed using Cohen’s d and its 95% confidence interval