Table 2 Responses on the Likert scale questions over the past month in patients receiving Gaucher-disease specific therapy (Yes) and in untreated patients (No)
From: Patient reported outcome measures in a large cohort of patients with type 1 Gaucher disease
Questions | None of the time | A little of the time | Some of the time | Most of the time | All of the time | p value* | |||||
|---|---|---|---|---|---|---|---|---|---|---|---|
Treatment status | Yes | No | Yes | No | Yes | No | Yes | No | Yes | No | |
1. My GD has restricted my education/job | 80 (67%) | 64 (94%) | 17 (14%) | 4 (6%) | 19 (16%) | 0 | 3 (2.5%) | 0 | 1 (0.5%) | 0 | 0.002 |
2. My GD has restricted fun activities with friends | 94 (76%) | 66 (97%) | 14 (11%) | 2 (3%) | 12 (10%) | 0 | 2 (1.5%) | 0 | 1 (0.5%) | 0 | 0.002 |
3. My GD has restricted my ability to have intimate relationships with my spouse/partner | 94 (90%) | 61 (100%) | 6 (6.5%) | 0 | 4 (2%) | 0 | 0 | 0 | 0 | 0 | 0.001 |
4. My GD has restricted my ability to take part in hobbies and leisure activities | 88 (71%) | 61 (90%) | 17 (14%) | 4 (6%) | 12 (10%) | 3 (4%) | 4 (3%) | 0 | 2 (2%) | 0 | 0.013 |
5. I have been concerned that I am an emotional burden to others because of my GD | 94 (77%) | 64 (95.5%) | 13 (11%) | 3 (4.5%) | 10 (7.5%) | 0 | 3 (2.5%) | 0 | 2 (2%) | 0 | 0.016 |
6. Because of my GD, I am concerned I will be at risk of bone disease | 35 (28.5%) | 38 (56%) | 31 (25%) | 16 (23.5%) | 39 (32%) | 13 (19%) | 10 (8%) | 0 | 8 (6.5%) | 1 (1.5%) | 0.007 |
7. Because of my GD, I am concerned I will be at risk of cancers | 78 (65%) | 49 (73%) | 19 (16%) | 10 (15%) | 21 (17.5%) | 7 (10.5%) | 0 | 0 | 2 (1.5%) | 1 (1.5%) | 0.3 |
8. Because of my GD, I am concerned I will be at risk of Parkinson | 43 (36%) | 40 (59%) | 25 (21%) | 15 (22%) | 46 (38%) | 11 (16%) | 3 (2.5%) | 1 (1.5%) | 3 (2.5%) | 1 (1.5%) | 0.1 |
9. Because of my GD, I am concerned I will be a financial burden | 62 (50.5%) | 48 (72%) | 28 (23%) | 11 (16%) | 23 (19%) | 8 (12%) | 3 (2.5%) | 0 | 6 (5%) | 0 | 0.06 |
10. I am concerned I will not get the best therapy because of budget issues | 72 (60%) | 36 (54.5%) | 23 (19.5%) | 19 (29%) | 16 (13%) | 7 (10.5%) | 6 (5%) | 3 (4.5%) | 3 (2.5%) | 1 (1.5%) | 0.9 |
11. I am concerned I may not have an expert physician for advice in the future | 55 (45%) | 32 (47%) | 28 (23%) | 19 (27.5%) | 30 (24%) | 12 (18%) | 6 (5%) | 4 (6%) | 4 (3%) | 1 (1.5%) | 0.3 |
12. My non-Gaucher problems are more concerning than the Gaucher concerns | 9 (7.5%) | 5 (8%) | 12 (10%) | 7 (11%) | 46 (38.5%) | 19 (29%) | 39 (33%) | 17 (26%) | 13 (11%) | 17 (26%) | 0.05 |
| Â | Strongly agree | Agree | Neither/nor agree | Disagree | Strongly disagree | Â | |||||
|---|---|---|---|---|---|---|---|---|---|---|---|
13. My health in general has improved because of my Gaucher-specific medication | 23 (25.5%) | NA | 23 (25.5%) | NA | 16 (18%) | NA | 28 (31%) | NA | 0 | NA | NA |
14. Over the past month, all of my medical concerns have been Gaucher-related | 5 (4%) | 1 (1.5%) | 11 (9%) | 2 (3%) | 15 (12.5%) | 2 (3%) | 53 (43.5%) | 19 (28%) | 38 (31%) | 44 (64.5%) | 0.002 |
15. My current medication has treated my Gaucher-specific concerns | 41 (34%) | 3 (5%) | 31 (26%) | 0 | 11 (9%) | 3 (5%) | 18 (15%) | 10 (16%) | 19 (16%) | 46 (74%) | NA |