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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
  Strongly agree Agree Neither/nor agree Disagree Strongly disagree  
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
  1. GD Gaucher disease, NA not applicable
  2. *Proportional Odds logistic regression adjusted for age, gender and severity score index
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172