Table 3.
Primary and Secondary Efficacy Outcome
| MSC Group | Control Group | |||||
|---|---|---|---|---|---|---|
| Efficacy Outcomes | n | Mean (SD) | n | Mean (SD) | Mean Difference between Group (SE, 95% CI) | p |
| Primary efficacy outcome | ||||||
| ALSFRS‐R score change from baseline to 4 months | 32 | −1.69 (2.51) | 27 | −4.67 (3.25) | 2.98 (0.75, 1.48 to 4.47) | <0.001 |
| ALSFRS‐R score change from baseline to 6 months | 31 | −3.10 (3.51) | 25 | −6.48 (4.53) | 3.38 (1.07, 1.23 to 5.54) | 0.003 |
| Secondary efficacy outcome | ||||||
| Changes in ALSFRS‐R slope between lead‐in and follow‐up perioda | 32 | 1.13 (1.33) | 27 | 0.03 (1.33) | 1.10 (0.35, 0.40 to 1.79) | 0.003 |
| Changes in ALSFRS‐R slope between lead‐in and follow‐up period,b up to 6 months | 32 | 1.08 (1.34) | 25 | 0.16 (1.46) | 0.92 (0.38, 0.16 to 1.67) | 0.018 |
| AALS score change from baseline to 4 months | 32 | 10.44 (9.24) | 25 | 17.96 (11.78) | −7.18 (2.76, −12.71 to −1.64) | 0.009 |
| Changes in AALS slope between lead‐in and follow‐up period | 31 | −1.51 (3.52) | 25 | 0.29 (5.06) | −1.80 (1.14, −4.08 to 0.48) | 0.119 |
| FVC % change from baseline to 4 months | 31 | −11.28 (10.06) | 25 | −10.75 (8.40) | −0.53 (2.52, −5.58 to 4.51) | 0.833 |
| Changes in FVC % slope between lead‐in and follow‐up period | 31 | −1.54 (3.38) | 25 | −0.80 (3.18) | −0.74 (0.89, −2.52 to 1.03) | 0.406 |
| SF‐36 change from baseline to 4 months | 32 | −9.06 (12.83) | 25 | −11.83 (11.28) | 2.78 (12.18, −3.74 to 9.29) | 0.397 |
| 4 Months | 6 Months | |||||
|---|---|---|---|---|---|---|
| Responder Analysis | MSC, n = 32 | Control, n = 27 | p | MSC, n = 32 | Control, n = 27 | p |
| <50% improvement in ALSFRS‐R slope, poor responders, n (%) | 10 (31%) | 22 (82%) | <0.001 | 12 (38%) | 21 (78%) | 0.002 |
| ≥50% improvement in ALSFRS‐R slope, good responders, n (%) | 22 (69%) | 5 (19%) | <0.001 | 20 (63%) | 6 (22%) | 0.002 |
| ≥75% improvement in ALSFRS‐R slope, n (%) | 18 (56%) | 2 (7%) | <0.001 | 17 (53%) | 3 (11%) | 0.001 |
| ≥100% improvement in ALSFRS‐R slope, n (%) | 13 (41%) | 1 (4%) | 0.001 | 8 (25%) | 1 (4%) | 0.031 |
Baseline was obtained at Visit 5 (just prior to the first MSC injection).
a
Changes in ALSFRS‐R slope = ([V5 − V1]/3 − [V9 − V5]/4).
b
Changes in ALSFRS‐R slope = ([V5 − V1]/3 − [V10 − V5]/6).
AALS = Appel ALS Rating Scale; ALSFRS‐R = Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised; CI = confidence interval; FVC = forced vital capacity; MSC = mesenchymal stem cell; SD = standard deviation; SE = standard error; SF‐36 = 36‐Item Short‐Form Health Survey; V = visit.