Table 2. Primary and Secondary Efficacy End Points.
| End point | Day 90 | Day 365 | ||||||
|---|---|---|---|---|---|---|---|---|
| No. (%) | Adjusted risk difference, % (95% CI) | P value | No. (%) | Adjusted risk difference, % (95% CI) | P value | |||
| MultiStem (n = 104) | Placebo (n = 102) | MultiStem (n = 104) | Placebo (n = 102) | |||||
| Excellent outcome | ||||||||
| Imputationa,b | 12/104 (11.5) | 10/102 (9.8) | 0.5 (−7.3 to 8.3) | .90 | 16/104 (15.4) | 11/102 (10.8) | 3.4 (−5.0 to 11.8) | .43 |
| Observeda,c | 12/103 (11.7) | 10/98 (10.2) | 0.4 (−7.6 to 8.4) | .92 | 14/97 (14.4) | 11/93 (11.8) | 0.4 (−8.1 to 8.9) | .93 |
| mRS score distributionb,d | ||||||||
| 0 | 5 (4.8) | 3 (2.9) | NA | .62 | 9 (8.7) | 1 (1.0) | NA | .42 |
| 1 | 9 (8.7) | 8 (7.8) | NA | 11 (10.6) | 13 (12.7) | NA | ||
| 2 | 20 (19.2) | 12 (11.8) | NA | 18 (17.3) | 13 (12.7) | NA | ||
| 3 | 15 (14.4) | 17 (16.7) | NA | 15 (14.4) | 17 (16.7) | NA | ||
| 4 | 31 (29.8) | 39 (38.2) | NA | 22 (21.2) | 33 (32.4) | NA | ||
| 5 | 18 (17.3) | 20 (19.6) | NA | 16 (15.4) | 15 (14.7) | NA | ||
| 6 | 6 (5.8) | 3 (2.9) | NA | 13 (12.5) | 10 (9.8) | NA | ||
| Global stroke recoveryc | 20/96 (20.8) | 16/94 (17.0) | 0.9 (−9.0 to 10.9) | .86 | NA | NA | NA | NA |
| mRS scorec | ||||||||
| ≤1 | 14/97 (14.4) | 11/95 (11.6) | 1.1 (−7.7 to 9.9) | .81 | NA | NA | NA | NA |
| ≤2 | 34/97 (35.1) | 23/95 (24.2) | 8.3 (−3.6 to 20.2) | .17 | NA | NA | NA | NA |
| NIHSS scorec | ||||||||
| ≤1 | 19/85 (22.4) | 19/85 (22.4) | −2.3 (−13.9 to 9.3) | .71 | NA | NA | NA | NA |
| Improvement ≥75%c | 30/85 (35.3) | 29/85 (34.1) | −1.6 (−15.4 to 12.2) | .82 | NA | NA | NA | NA |
| Barthel index ≥95c | 31/97 (32.0) | 24/93 (25.8) | 3.7 (−8.0 to 15.3) | .54 | NA | NA | NA | NA |
Abbreviations: CMH, Cochran-Mantel-Haenszel; mRS, modified Rankin Scale; NA, not applicable; NIHSS, National Institutes of Health Stroke Scale.
Treatments were compared using the CMH test adjusted for baseline NIHSS score (≤12 or ≥13), receipt of concomitant reperfusion therapy (yes or no), and age (20-74 or ≥75 years). Risk differences and corresponding 2-sided 95% CIs between the MultiStem and placebo groups were adjusted for the same factors applied in the CMH test and calculated using the Mantel-Haenszel method by Sato T.
Imputation was performed by last postrandomization efficacy assessment of excellent outcome and mRS score carried forward.
Observed cases. Regarding excellent outcome, included death cases as worst case and available partial missing case (ie, the case in at least available 1 of 3 composite scoring criteria for excellent outcome) of composite scoring criteria for excellent outcome.
P values were calculated using the CMH test adjusted for baseline NIHSS score (≤12 or ≥13), receipt of concomitant reperfusion therapy (yes or no), and age (20-74 or ≥75 years) using modified ridit scores.