Background
Methods
Study design and population
Evaluation protocol
Statistical analysis
Results
Baseline demographics
Unified Multiple System Atrophy Rating Scale progression
UMSARS item | Baseline (n=246) | 1-year follow-up (n=246) | 2-year follow-up (n=97) | p value | Post hoc tests | |||
---|---|---|---|---|---|---|---|---|
Median | IQR | Median | IQR | Median | IQR | |||
Part I | ||||||||
1. Speech | 1 | 0 | 1 | 1 | 2 | 2 | <0.001a | a,b,c |
2. Swallowing | 0 | 1 | 1 | 1 | 1 | 1 | <0.001a | a,b,c |
3. Handwriting | 1 | 0 | 1 | 1 | 2 | 2 | <0.001a | a,b,c |
4. Cutting food/handling utensils | 1 | 1 | 2 | 1 | 2 | 2 | <0.001a | a,b,c |
5. Dressing | 1 | 0 | 1 | 1 | 2 | 2 | <0.001a | a,b,c |
6. Hygiene | 1 | 1 | 2 | 1 | 2 | 2 | <0.001a | a,b,c |
7. Walking | 1 | 1 | 2 | 2 | 3 | 1 | <0.001a | a,b,c |
8. Falls | 0 | 1 | 0 | 1 | 1 | 2 | <0.001a | a,b,c |
9. Orthostatic symptoms | 1 | 2 | 1 | 3 | 2 | 2 | <0.001a | a,b |
10. Urinary function | 2 | 2 | 3 | 2 | 3 | 1 | <0.001a | a,b,c |
11. Sexual function | 1 | 4 | 3 | 4 | 4 | 1 | <0.001a | a,b,c |
12. Bowel function | 1 | 2 | 1 | 2 | 1 | 2 | <0.001a | a,b |
Part II | ||||||||
1. Facial expression | 2 | 1 | 2 | 0 | 2 | 1 | <0.001a | a,b,c |
2. Speech | 1 | 0 | 1 | 1 | 2 | 2 | <0.001a | a,b,c |
3. Ocular motor dysfunction | 0 | 1 | 1 | 1 | 1 | 2 | <0.001a | a,b |
4. Tremor at rest | 0 | 0 | 0 | 0 | 0 | 1 | 0.158 | |
5. Action tremor | 1 | 1 | 1 | 1 | 1 | 1 | <0.001a | a,b,c |
6. Increased tone | 1.5 | 2 | 2 | 1 | 2 | 1 | <0.001a | a,b |
7. Rapid alternating movements | 1 | 1 | 2 | 0 | 2 | 1 | <0.001a | a,b,c |
8. Finger taps | 2 | 1 | 2 | 1 | 2 | 1 | <0.001a | a,b,c |
9. Leg agility | 2 | 1 | 2 | 0 | 2 | 1 | <0.001a | a,b,c |
10. Heel-knee-shin test | 1 | 2 | 2 | 1 | 2 | 1 | <0.001a | a,b,c |
11. Arising from chair | 0 | 1 | 1 | 1 | 2 | 3 | <0.001a | a,b,c |
12. Posture | 1 | 1 | 1 | 1 | 1 | 1 | <0.001a | a,b,c |
13. Body sway | 1 | 1 | 2 | 2 | 3 | 2 | <0.001a | a,b,c |
14. Gait | 1 | 1 | 2 | 2 | 3 | 1 | <0.001a | a,b,c |
UMSARS-I | 13 | 7 | 18 | 10 | 24 | 11 | <0.001a | a,b,c |
UMSARS-II | 15 | 8 | 22 | 9 | 26 | 12 | <0.001a | a,b,c |
UMSARS total | 28 | 15 | 40 | 18 | 48 | 22 | <0.001a | a,b,c |
UMSARS item | Baseline to 1-year follow-up (n=246) | Baseline to 2-year follow-up (n=97) | 1- to 2-year follow-up (n=97) | ||||||
---|---|---|---|---|---|---|---|---|---|
Mean annual change | SD of the change | Standardized effect | Mean annual change | SD of the change | Standardized effect | Mean annual change | SD of the change | Standardized effect | |
Part I | |||||||||
1. Speech | 0.56 | 0.71 | 0.80 | 0.52 | 0.46 | 1.13 | 0.61 | 1.30 | 0.47 |
2. Swallowing | 0.33 | 0.80 | 0.42 | 0.38 | 0.46 | 0.84 | 0.48 | 0.92 | 0.52 |
3. Handwriting | 0.46 | 0.83 | 0.56 | 0.41 | 0.45 | 0.92 | 0.57 | 0.83 | 0.69 |
4. Cutting food/handling utensils | 0.42 | 0.73 | 0.58 | 0.44 | 0.48 | 0.93 | 0.51 | 0.85 | 0.60 |
5. Dressing | 0.40 | 0.66 | 0.61 | 0.44 | 0.48 | 0.94 | 0.51 | 0.78 | 0.66 |
6. Hygiene | 0.48 | 0.71 | 0.67 | 0.51 | 0.50 | 1.01 | 0.55 | 0.78 | 0.71 |
7. Walking | 0.57 | 0.78 | 0.73 | 0.63 | 0.44 | 1.41 | 0.77 | 0.75 | 1.03 |
8. Falls | 0.37 | 1.00 | 0.36 | 0.38 | 0.63 | 0.60 | 0.47 | 1.40 | 0.34 |
9. Orthostatic symptoms | 0.22 | 1.19 | 0.19 | 0.31 | 0.76 | 0.41 | 0.23 | 1.23 | 0.19 |
10. Urinary function | 0.41 | 1.17 | 0.35 | 0.49 | 0.62 | 0.79 | 0.73 | 1.28 | 0.57 |
11. Sexual function | 0.51 | 1.97 | 0.26 | 0.64 | 1.00 | 0.64 | 1.01 | 1.88 | 0.54 |
12. Bowel function | 0.30 | 0.93 | 0.32 | 0.17 | 0.57 | 0.31 | 0.09 | 1.13 | 0.08 |
Part II | |||||||||
1. Facial expression | 0.56 | 0.90 | 0.62 | 0.45 | 0.55 | 0.83 | 0.51 | 0.93 | 0.55 |
2. Speech | 0.50 | 0.68 | 0.74 | 0.48 | 0.45 | 1.07 | 0.52 | 0.78 | 0.67 |
3. Ocular motor dysfunction | 0.31 | 0.96 | 0.32 | 0.21 | 0.48 | 0.44 | 0.09 | 1.07 | 0.08 |
4. Tremor at rest | 0.04 | 0.63 | 0.06 | 0.07 | 0.41 | 0.18 | 0.12 | 0.75 | 0.15 |
5. Action tremor | 0.21 | 0.92 | 0.23 | 0.23 | 0.48 | 0.48 | 0.30 | 0.94 | 0.32 |
6. Increased tone | 0.40 | 1.15 | 0.35 | 0.39 | 0.59 | 0.67 | 0.45 | 1.11 | 0.40 |
7. Rapid alternating movements | 0.40 | 0.85 | 0.47 | 0.32 | 0.49 | 0.65 | 0.30 | 0.82 | 0.37 |
8. Finger taps | 0.38 | 0.70 | 0.55 | 0.36 | 0.46 | 0.77 | 0.32 | 0.85 | 0.37 |
9. Leg agility | 0.42 | 0.75 | 0.56 | 0.44 | 0.42 | 1.05 | 0.48 | 0.70 | 0.68 |
10. Heel-knee-shin test | 0.39 | 0.95 | 0.41 | 0.40 | 0.56 | 0.71 | 0.43 | 0.98 | 0.44 |
11. Arising from chair | 0.79 | 0.91 | 0.87 | 0.77 | 0.57 | 1.36 | 0.95 | 1.00 | 0.95 |
12. Posture | 0.37 | 0.75 | 0.49 | 0.37 | 0.46 | 0.81 | 0.44 | 0.83 | 0.53 |
13. Body sway | 0.62 | 1.00 | 0.61 | 0.61 | 0.64 | 0.95 | 0.66 | 1.11 | 0.59 |
14. Gait | 0.50 | 0.73 | 0.69 | 0.56 | 0.45 | 1.23 | 0.66 | 0.68 | 0.97 |
Non-Motor Symptoms Scale progression
Baseline (n=246) | 1-year follow-up (n=246) | 2-year follow-up (n=97) | p value | Post hoc tests | ||||
---|---|---|---|---|---|---|---|---|
NMSS | Median | IQR | Median | IQR | Median | IQR | ||
D1: Cardiovascular | 2 | 4 | 2 | 5 | 3 | 7 | <0.001a | a,b |
D2: Sleep/fatigue | 5 | 8 | 6 | 7 | 6 | 9 | 0.004a | b |
D3: Mood/apathy | 4 | 16 | 6 | 14 | 6 | 20 | 0.024a | b |
D4: Perceptual problems/hallucinations | 0 | 0 | 0 | 0 | 0 | 0 | <0.001a | a,b |
D5: Attention/memory | 2 | 5 | 3 | 5 | 4 | 6 | <0.001a | a,b,c |
D6: Gastrointestinal | 2.5 | 6 | 4 | 7 | 5 | 8 | <0.001a | a,b,c |
D7: Urinary | 9 | 18 | 9.5 | 16 | 17 | 18 | <0.001a | b,c |
D8: Sexual function | 0 | 8 | 2 | 8 | 8 | 14 | <0.001a | a,b,c |
D9: Miscellaneous | 2 | 8 | 4 | 7 | 3 | 6 | 0.053 | |
NMSS total score | 41 | 45 | 46 | 47 | 65 | 50 | <0.001a | a,b,c |
NMSS | Baseline to 1-year follow-up (n=246) | Baseline to 2-year follow-up (n=97) | 1- to 2-year follow-up (n=97) | ||||||
---|---|---|---|---|---|---|---|---|---|
Mean annual change | SD of the change | Standardized effect | Mean annual change | SD of the change | Standardized effect | Mean annual change | SD of the change | Standardized effect | |
D1: Cardiovascular | 1.09 | 3.84 | 0.28 | 0.95 | 2.42 | 0.39 | 0.37 | 4.03 | 0.09 |
D2: Sleep/fatigue | 0.52 | 6.66 | 0.08 | 1.08 | 3.55 | 0.30 | 1.87 | 6.91 | 0.27 |
D3: Mood/apathy | 0.93 | 14.85 | 0.06 | 2.08 | 7.37 | 0.28 | 3.73 | 14.36 | 0.26 |
D4: Perceptual problems/hallucinations | 0.42 | 1.75 | 0.24 | 0.29 | 0.87 | 0.34 | 0.12 | 2.33 | 0.05 |
D5: Attention/memory | 1.01 | 4.38 | 0.23 | 1.22 | 2.29 | 0.53 | 1.34 | 2.82 | 0.47 |
D6: Gastrointestinal | 2.01 | 5.14 | 0.39 | 1.56 | 2.63 | 0.59 | 1.92 | 5.06 | 0.38 |
D7: Urinary | 1.09 | 9.82 | 0.11 | 3.16 | 3.79 | 0.83 | 5.72 | 7.31 | 0.78 |
D8: Sexual function | 1.02 | 5.32 | 0.19 | 2.20 | 3.91 | 0.56 | 3.78 | 8.57 | 0.44 |
D9: Miscellaneous | 0.51 | 4.24 | 0.12 | 0.52 | 2.71 | 0.19 | 0.69 | 5.47 | 0.13 |