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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