Table 5.
Secondary Efficacy Endpoints for the Randomized Cohort at 12 Months
| Allo-MSCs |
Placebo |
Effect Estimate∗ |
|||||||
|---|---|---|---|---|---|---|---|---|---|
| N | Mean | SD | N | Mean | SD | Diff | 95% CI | p Value | |
| Function | |||||||||
| LVEF, % | |||||||||
| Baseline | 12 | 34.51 | 2.86 | 13 | 33.31 | 6.21 | |||
| 12 months | 12 | 37.98 | 7.84 | 13 | 35.99 | 7.98 | |||
| Delta | 12 | 3.47 | 6.00 | 13 | 2.68 | 6.85 | 0.80 | -4.52 to 6.11 | 0.746 |
| Within group p value | 0.070 | 0.184 | |||||||
| Global circumferential strain, % | |||||||||
| Baseline | 11 | −10.21 | 1.83 | 13 | -9.76 | 2.43 | |||
| 12 months | 11 | −11.04 | 2.39 | 13 | -9.88 | 2.53 | |||
| Delta | 11 | −0.83 | 1.70 | 13 | -0.12 | 2.86 | -0.71 | -2.68 to 1.26 | 0.328 |
| Within group p value | 0.136 | 0.883 | |||||||
| Regional longitudinal strain, %‡ | |||||||||
| Baseline | 12 | −12.45 | 2.58 | 13 | -11.17 | 1.95 | |||
| 12 months | 12 | −13.65 | 3.01 | 13 | -10.79 | 3.32 | |||
| Delta | 12 | −1.20 | 3.34 | 13 | 0.38 | 3.73 | -1.58 | -4.51 to 1.35 | 0.071 |
| Within group p value | 0.239 | 0.720 | |||||||
| Structure | |||||||||
| LV end-diastolic volume index | |||||||||
| Baseline | 12 | 103.47 | 27.88 | 13 | 108.64 | 21.57 | |||
| 12 months | 12 | 101.17 | 28.74 | 13 | 105.28 | 26.03 | |||
| Delta | 12 | −2.30 | 16.97 | 13 | -3.36 | 15.18 | 1.06 | -12.33 to 14.45 | 0.935 |
| Within group p value | 0.647 | 0.440 | |||||||
| LV end-systolic volume index | |||||||||
| Baseline | 12 | 68.22 | 20.33 | 13 | 73.35 | 20.83 | |||
| 12 months | 12 | 64.60 | 26.25 | 13 | 69.07 | 24.43 | |||
| Delta | 12 | −3.62 | 15.66 | 13 | -4.28 | 14.18 | 0.66 | -11.75 to 13.08 | 0.919 |
| Within group p value | 0.440 | 0.298 | |||||||
| LV sphericity index | |||||||||
| Baseline | 12 | 0.50 | 0.12 | 13 | 0.56 | 0.09 | |||
| 12 months | 12 | 0.51 | 0.12 | 13 | 0.49 | 0.09 | |||
| Delta | 12 | 0.01 | 0.08 | 13 | -0.07 | 0.09 | 0.077 | 0.003 to 0.15 | 0.124 |
| Within group p value | 0.685 | 0.024 | |||||||
| Morphology | |||||||||
| Scar size percent | |||||||||
| Baseline | 11 | 13.78 | 8.29 | 10 | 8.83 | 5.49 | |||
| 12 months | 11 | 12.72 | 7.41 | 10 | 8.42 | 5.57 | |||
| Delta | 11 | −1.06 | 2.44 | 10 | -0.41 | 2.76 | -0.66 | -3.05 to 1.74 | 0.993 |
| Within group p value | 0.180 | 0.652 | |||||||
| Scar tissue mass, g | |||||||||
| Baseline | 11 | 15.29 | 10.43 | 10 | 10.45 | 7.38 | |||
| 12 months | 11 | 15.05 | 10.75 | 10 | 10.30 | 8.23 | |||
| Delta | 11 | −0.24 | 3.15 | 10 | -0.15 | 3.12 | -0.090 | -2.96 to 2.78 | 0.949 |
| Within group p value | 0.808 | 0.885 | |||||||
| Viable mass, g | |||||||||
| Baseline | 11 | 106.75 | 37.32 | 10 | 112.40 | 24.55 | |||
| 12 months | 11 | 109.73 | 41.63 | 10 | 111.93 | 21.64 | |||
| Delta | 11 | 2.98 | 11.96 | 10 | -0.47 | 13.39 | 3.45 | -8.23 to 15.12 | 0.567 |
| Within group p value | 0.428 | 0.914 | |||||||
| Viable mass percent (transformation)† | |||||||||
| Baseline | 11 | 92.82 | 6.72 | 10 | 95.83 | 3.68 | |||
| 12 months | 11 | 92.92 | 6.67 | 10 | 95.65 | 3.78 | |||
| Delta | 11 | 0.10 | 2.73 | 10 | -0.18 | 2.08 | 0.28 | -1.93 to 2.49 | 0.704 |
| Within group p value | 0.946 | 0.919 | |||||||
| Functional capacity | |||||||||
| 6MWT, m | |||||||||
| Baseline | 12 | 384.38 | 77.88 | 15 | 359.57 | 48.60 | |||
| 12 months | 12 | 419.33 | 93.83 | 15 | 356.50 | 55.46 | |||
| Delta | 12 | 34.96 | 61.62 | 15 | -3.07 | 42.90 | 38.03 | -5.84 to 81.89 | 0.056 |
| Within group p value | 0.075 | 0.786 | |||||||
| MLHFQ score | |||||||||
| Baseline | 12 | 46.63 | 25.92 | 14 | 53.14 | 30.01 | |||
| 12 months | 12 | 21.18 | 17.22 | 14 | 40.51 | 30.98 | |||
| Delta | 12 | −25.45 | 25.65 | 14 | -12.63 | 14.59 | -12.82 | -30.49 to 4.84 | 0.048 |
| Within group p value | 0.006 | 0.006 | |||||||
| Biomarkers | |||||||||
| NT-proBNP, pg/ml (transformation)† | |||||||||
| Baseline | 12 | 1046.58 | 1064.32 | 15 | 695.07 | 624.02 | |||
| 12 months | 12 | 654.00 | 619.46 | 15 | 618.31 | 491.23 | |||
| Delta | 12 | −392.58 | 953.71 | 15 | -76.76 | 410.48 | -315.80 | −947.50 to 315.80 | 0.199 |
| Within group p value | 0.129 | 0.871 | |||||||
6MWT = 6-min walk test; AIC = anthracycline-induced cardiomyopathy; Allo-MSC = allogeneic mesenchymal stromal cells; LV = left ventricular; LVEF = left ventricular ejection fraction; MLHFQ = Minnesota Living with Heart Failure Questionnaire; NT-proBNP = N-terminal pro-brain natriuretic peptide.
Least-squares mean (LS-mean) difference from the analysis of covariance model. Analysis of covariance adjusted for baseline.
Transformation- arc sine square root transformation for viable mass percent; Log transformation for NT-proBNP; p-values were obtained from transformed data.
Longitudinal strain was assessed across 7 segments each from tagged images of one 4-chamber (basal lateral, midventricular lateral, apical lateral, apex, apical septal, midventricular septal, and basal septal regions) and one 2-chamber view (basal anterior, midventricular anterior, apical anterior, apex, apical inferior, midventricular inferior, and basal inferior segments).