Table 4.
Longitudinal biomarker analysis
| Measurement | Baseline (SD) | 48-week follow-up (SD) | Mean annual change (SD) | Standardized effect size | Power calculation for clinical trials (subjects per group) | |
|---|---|---|---|---|---|---|
| 30% reduction | 50% reduction | |||||
| UMSARS | Points | |||||
| UMSARS-1 (n=22) | 19.9 (4.8) | 26.0 (6.4) | 6.1 (4.5) | 1.30 | 118 | 43 |
| UMSARS-2 (n=22) | 21.4 (5.4) | 27.9 (7.7) | 6.5 (4.6) | 1.40 | 101 | 37 |
| UMSARS-total (n=22) | 41.2 (9.5) | 53.9 (13.2) | 12.6 (8.8) | 1.40 | 100 | 36 |
| Blood biomarkers | pg./ml | |||||
| NfL (N=12) | 23.0 (10.8) | 42.9 (20.6) | 19.9 (15.3) | 1.30 | 104 | 38 |
| αSyn neuronal exosomes (n=14) | 351.4 (299.7) | 69.7 (120.2) | −281.7 (331.5) | −0.8 | 241 | 140 |
| αSyn glial exosomes (n=14) | 1030 (920.9) | 67.1 (80.4) | −962.9 (919.9) | −1.1 | 159 | 58 |
| Retinal OCT | μm | |||||
| RNFL OD (n=8 eyes) | 91.1 (9.5) | 92.9 (11.6) | 1.3 (5.5) | 0.2 | 3123 | 1125 |
| RNFL OS (n=9 eyes) | 90.6 (9.7) | 91.8 (10.5) | 1.1 (5.7) | 0.2 | 4685 | 1687 |
| GCC OD (n=8 eyes) | 78.4 (6) | 79.5 (6.3) | 0.4 (1.8) | 0.2 | 3534 | 1272 |
| GCC OS (n=9 eyes) | 78.3 (5.4) | 78.6 (7.0) | 0.2 (2.7) | 0.1 | >10,000 | >10,000 |
| Volumetric brain MRI | % | |||||
| Whole brain (n=12) | 1353.36 (933) | 1337.9 (913) | −1.11 (0.60) | −1.85 | 49 | 19 |
| Left putamen (n=12) | 2.72 (0.83) | 2.50 (0.80) | −8.26 (2.91) | −2.83 | 22 | 8 |
| Right putamen (n=12) | 3.44 (0.78) | 3.21 (0.64) | −6.38 (2.59) | −2.46 | 29 | 11 |
| Lateral ventricles (n=12) | 26.13 (11.6) | 28.42 (32.2) | 10.14 (9.94) | 1.03 | 168 | 61 |
| Cerebellum (n=12) | 138.2 (231.2) | 136.7 (210.1) | −1.17 (0.73) | −1.60 | 68 | 25 |
| Midbrain (n=12) | 9.4 (0.6) | 9.3 (0.4) | −1.60 (1.44) | −1.11 | 84 | 31 |
| Pons (n=12) | 14.7 (5) | 14 (4) | −4.69 (2.26) | −2.07 | 34 | 13 |
| Medulla (n=12) | 4.2 (0.6) | 4 (0.4) | −3.95 (1.83) | −2.16 | 38 | 14 |
UMSARS: Unified MSA Rating Scale; OCT: Optical Coherence Tomography; OD: Right Eye; OS: Left Eye; SD: Standard Deviation;