Table 3.
Distribution of answers between patients and their caregivers.
| Patients Cohort (N; %) | Caregivers Cohort (N; %) | x2 p Value | ||
|---|---|---|---|---|
| Question 1 | ||||
| Speaking of motor skills, please indicate if in the last year you: | Remained stable | 29; 64.44% | 28; 62.22% | (x2 (2, N = 90) = 0.461, p = 0.794) |
| Deteriorated | 4; 8.89% | 6; 13.33% | ||
| Improved | 12; 26.67% | 11; 24.44% | ||
| Question 2 | ||||
| What do you expect in the next two years | Stable course | 32; 71.11% | 22; 48.89% | (x2 (2, N = 90) = 4.977, p = 0.083) |
| Deterioration | 2; 4.44% | 2; 4.44% | ||
| Improvement | 21; 46.67% | |||
| Question 3 | ||||
| Would you agree to have participate in a potential clinical trial if, in the absence of side effects or with the possibility of minimal side effects, the prospective was: | Slow down | 29; 64.44% | 33; 73.33% | (x2 (1, N = 90) = 8.29, p = 0.362) |
| Stop deterioration | 37; 82.22% | 36; 80.00% | (x2 (1, N = 90) = 0.73, p = 0.788) | |
| Improve | 45; 100% | 45; 100.00% | N/A | |
| Questions 4–7 | ||||
| Would you consider participating in a clinical trial if it offered the prospect of achieving at least 1, 2, or more than 2 activities on the HFMSE/RULM | ||||
|
HFMSE
Acquisition from score 0 Not applicable in 3 patients |
I would not participate | 3; 7.14% | 0; 0.00% | (x2 (3, N = 84) = 6.205, p = 0.102) |
| At least 1 activity | 21; 50% | 27; 64.29% | ||
| Two activities | 29; 69.05% | 30; 71.43% | ||
| More than 2 activities | 39; 92.86% | 42; 100.00% | ||
|
HFMSE
Improvements from score 1 Not applicable in 6 patients |
I would not participate | 5; 12.82% | 0; 0.00% | (x2 (3, N = 78) = 7.067, p = 0.07) |
| At least 1 activity | 18; 46.15% | 22; 56.41% | ||
| Two activities | 24; 61.54% | 25; 64.10% | ||
| More than 2 activities | 34; 87.18% | 39; 100.00% | ||
|
RULM
Acquisition from score 0 Not applicable in 8 patients |
I would not participate | 1; 2.7% | 0; 0.00% | (x2 (3,N = 74) = 4.100, p = 0.251) |
| At least 1 activity | 16; 43.24% | 24; 64.86% | ||
| Two activities | 21; 56.76% | 27; 72.97% | ||
| More than 2 activities | 36; 97.30% | 37; 100.00% | ||
|
RULM
Improvements from score 1 Not applicable in 12 patients |
I would not participate | 1; 3.03% | 0; 0.00% | (x2 (3,N = 66) = 1.981, p = 0.576) |
| At least 1 activity | 19; 57.58% | 22; 66.67% | ||
| Two activities | 20; 60.61% | 24; 72.73% | ||
| More than 2 activities | 32; 96.97% | 33; 100.00% | ||
| Question 8 | ||||
| With the advent of new pharmacological therapies, how has your perception of the disease changed? | No change | 16; 35.56% | 13; 28.89% | (x2 (2, N = 90) = 0.796, p = 0.672) |
| More positive | 26; 57.78% | 30; 66.67% | ||
| More negative | 0; 0.00% | 0; 0.00% | ||
| Uncertain | 3; 6.67% | 2; 4.44% | ||
| Question 9 | ||||
| What are your expectations regarding the new pharmacological therapies | No change | 12; 26.67% | 6; 13.33% | (x2 (2, N = 90) = 3.362, p = 0.339) |
| More positive | 32; 71.11% | 37; 82.22% | ||
| More negative | 0; 0.00% | 1; 2.22% | ||
| Uncertain | 1; 2.22% | 1; 2.22% | ||