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Table 1 Content validation: demographic 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

Characteristic

Round 1

n = 23

Round 2

n = 10

Total

n = 33

Mean (range) age, years

35.7 (13–67)

50.9 (15–73)

40.3 (13–73)

Sex, n (%)

 Female

14 (60.9)

7 (70.0)

21 (63.6)

Ethnicity, n (%)*

 White

11 (47.8)

7 (70.0)

18 (54.5)

 Ashkenazi Jewish

11 (47.8)

3 (30.0)

14 (42.4)

 Other

4 (17.4)

0

4 (12.1)

Highest educational level (adults only), n (%)

 Some high school

1 (4.3)

0

1 (3.0)

 High school diploma or General Educational Development (GED)

3 (13.0)

1 (10.0)

4 (12.1)

 Some years of college

3 (13.0)

1 (10.0)

4 (12.1)

 Certificate program

1 (4.3)

0

1 (3.0)

 University/college

3 (13.0)

2 (20.0)

5 (15.2)

 Graduate or professional degree

5 (21.7)

4 (40.0)

9 (27.3)

 Other

1 (4.3)

1 (10.0)

2 (6.1)

 N/A or missing

6 (26.1)

0

7 (21.2)

Mean (range) duration of disease, years

26.2 (1–60)

35.0 (14–63)

26.8 (1–63)

Disease severity (as rated by the recruiting clinician), n (%)

 Very mild

1 (4.3)

0

1 (3.0)

 Mild

5 (21.7)

2 (20.0)

7 (21.2)

 Moderate

14 (60.9)

4 (40.0)

18 (54.5)

 Severe

3 (13.0)

3 (30.0)

6 (18.2)

 Very severe

0

1 (10.0)

1 (3.0)

  1. *Multiple answers given