Table 2 Baseline demographic and clinical characteristics of concept elicitation and cognitive interview participants
Characteristic | Concept elicitation participants | Cognitive interview participants | ||
|---|---|---|---|---|
Treated group (n = 20) | Treatment-naïve group (n = 17) | All (N = 37) | All (N = 14) | |
Age (years), mean ± SD | 45.7 ± 12.1 | 43.7 ± 13.8 | 45.7 ± 12.9 | 48.9 ± 15.8 |
Female, n (%) | 11 (55.0) | 11 (64.7) | 22 (59.5) | 8 (53) |
Phenotype | ||||
Classic, n (%) | 19 (95.0) | 15 (88.2) | 34 (91.9) | N/A |
Late-onset, n (%) | 1 (5.0) | 2 (11.8) | 3 (0.1) | N/A |
Marital status, n (%) | ||||
Married/living as married | 14 (70.0) | 11 (64.7) | 25 (67.6) | 5 (36) |
Widowed | 0 | 1 (5.9) | 1 (2.7) | 2 (14) |
Separated/divorced | 2 (10.0) | 1 (5.9) | 3 (8.1) | 4 (29) |
Never married | 4 (20.0) | 4 (23.5) | 8 (21.6) | 3 (21) |
Post-secondary education, n (%) | 13 (65.0) | 13 (76.5) | 26 (70.2) | 12 (86) |
Employment status, n (%) | ||||
Employed full-time, part-time, self-employed | 13 (65.0) | 12 (70.6) | 25 (67.6) | 7 (50) |
Out of work | 1 (5.0) | 2 (11.8) | 3 (8.1) | 2 (14) |
Homemaker | 1 (5.0) | 1 (5.9) | 2 (5.4) | 2 (14) |
Retired | 1 (5.0) | 2 (11.8) | 3 (8.1) | 1 (7) |
Unable to work | 4 (20.0) | 0 | 4 (10.8) | 2 (14) |
Race, n (%) | ||||
White | 15 (88.2) | 17 (85.0) | 32 (86.5) | 14 (100) |
Black | 2 (11.8) | 2 (10.0) | 4 (10.8) | 0 |
Other | 0 | 1 (5.0) | 1 (2.7) | 0 |
Hispanic, Latino, or Spanish origin, n (%) | 2 (11.8) | 1 (5.0) | 3 (8.1) | 0 |
Time since diagnosis, years, mean ± SD | 14.5 ± 13.7 | 3.6 ± 7.6 | 9.6 ± 12.3 | 12.0 ± 9.6 |
Time since confirming genetic test, years, mean ± SD | 11.3 ± 13.7 | 2.5 ± 3.4 | 7.4 ± 10.9 | N/A |
Treated with ERT ≥ 3 years, n (%) | 12 (60.0) | 0 | 12 (32.4) | N/A |
Ever received treatment (Replagal, SRT, or Fabrazyme | N/A | N/A | N/A | 12 (86) |
Treated with Fabrazyme ≥ 6 months, n (%) | N/A | N/A | N/A | 11 (79) |