Skip to main content

Table 3 Psychometric validation: demographics characteristics of the study population

From: Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials

 

Main sample

(N = 46)

Test–retest sample

(n = 23)

Characteristic

n

%

n

%

Age, years

 2534

9

19.6

4

17.4

 3544

13

28.3

6

26.1

 4554

3

6.5

1

4.3

 5564

12

26.1

5

21.7

 6574

5

10.9

5

21.7

 ≥75

4

8.7

2

8.7

Gender

 Male

23

50.0

10

43.5

 Female

22

47.8

12

52.2

 Missing

1

2.2

1

4.3

Ethnicity

 White

45

97.8

22

95.7

 Other

1

2.2

1

4.3

Occupational status

 Employed part-time

8

17.4

3

13.0

 Employed full-time

14

30.4

6

26.1

 Self-employed

4

8.7

1

4.3

 Unemployed

5

10.9

2

8.7

 Retired

13

28.3

9

39.1

 Missing

2

4.3

2

8.7

Time since diagnosis

 <1 year

2

4.3

0

0.0

 14 years

3

6.5

1

4.3

 59 years

5

10.9

4

17.4

 1019 years

6

13.0

2

8.7

 ≥20 years

29

63.0

16

69.6

 Missing

1

2.2

0

0.0

Current health

 Very good

7

15.2

4

17.4

 Good

20

43.5

9

39.1

 Fair

13

28.3

6

26.1

 Bad

4

8.7

3

13.0

 Very bad

2

4.3

1

4.3

Gaucher disease severity state

 Mild (GD1-DS3 score < 3.00)

21

45.7

12

52.2

 Moderate (GD1-DS3 score 3.005.99)

7

15.2

2

8.7

 Marked (GD1-DS3 score 6.008.99)

11

23.9

3

13.0

 Severe (GD1-DS3 score 9.0019.00)

7

15.2

6

26.1

  1. GD1-DS3 Gaucher disease type 1- disease severity scoring system (three domains)