Table 2.
Change in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised overall score and subdomain scores and in forced vital capacity, at treatment end (assessment performed at week 25) and end of follow-up in the evaluable population
| Parameter | Baseline (n = 13) | Week 25 (n = 11) | Week 48 (n = 10) | ||
|---|---|---|---|---|---|
| Change from baseline | p value | Change from baseline | p value | ||
| ALSFRS-R overall, median (IQR) | 42.0 (37.0, 44.0) | − 4.0 (− 8.0, − 3.0) | 0.0010 | − 10.0 (− 14.0, − 7.0) | < 0.0001 |
| ALSFRS-R bulbar, median (IQR) | 10 (9.0, 12.0) | 0.0 (− 2.0, 0.0) | 0.2500 | 2.0 (− 4.0, 0.0) | 0.0313 |
| ALSFRS-R fine and gross motor, median (IQR) | 19.0 (17.0, 21.0) | − 3.0 (− 5.0, − 3.0) | 0.0010 | − 7.0 (− 9.0, − 5.0) | < 0.0001 |
| ALSFRS-R respiratory, median (IQR) | 12.0 (12.0, 12.0) | 0.0 (− 2.0, 0.0) | 0.1875 | 0.0 (− 4.0, 0.0) | 0.0625 |
| FVC (L), median (IQR) | 3.9 (3.0, 4.9) | − 0.4 (− 0.8, 0.0) | 0.0413 | − 0.9 (− 1.8, − 0.3) | 0.0155 |
| FVC as % of predicted, median (IQR) | 87.0 (76.0, 96.0) | − 9.0 (− 23.0, − 6.0) | 0.0068 | − 23.0 (− 38.0, − 9.0) | 0.0006 |
ALSFRS-R scores: overall score ranges from 0 (worst) to 48 (best). Lower scores indicate worse ability; higher scores indicate better ability. p-values in bold stands for p < 0.05
ALSFRS-R, Amyotrophic Lateral Sclerosis Functional Rating Scale- Revised; FVC, forced vital capacity; IQR, interquartile range