Table 2. Baseline sample characteristics in each data source.
| Tadalafil DMD Trial Placebo Arm | Leuven | CCHMC | |
|---|---|---|---|
| Number of ~1-year intervals | 92 | 235 | 543 |
| Number of patients | 92 | 67 | 212 |
| Demographics, mean ± SD, median [range] | |||
| Age (years) | 9.36 ± 1.79 9.13 [7.00, 14.58] |
9.10 ± 2.73 8.84 [3.17, 17.42] |
8.81 ± 2.73 8.51 [2.42, 18.54] |
| Height (cm) | 125.17 ± 8.73 126.00 [108.80, 147.20] |
122.08 ± 10.60 122.50 [92.60, 154.00] |
120.81 ± 12.24 120.20 [80.00, 167.30] |
| Weight (kg) | 30.92 ± 8.86 28.40 [17.20, 61.40] |
28.36 ± 9.86 25.40 [14.20, 70.00] |
28.85 ± 10.54 26.75 [12.20, 88.94] |
| BMI (kg/m2) | 19.54 ± 4.31 18.01 [13.16, 35.02] |
18.53 ± 3.77 17.43 [12.10, 32.22] |
19.20 ± 3.72 18.44 [12.73, 37.21] |
| Steroid use | |||
| Steroid duration (months), mean ± SD | 39.19 ± 24.42 35.83 [6.83, 112.46] |
29.11 ± 26.71 23.52 [0.00, 121.43] |
43.05 ± 31.36 40.32 [0.00, 196.32] |
| Current deflazacort, n (%) | 44 (47.83) | 178 (75.74) | 448 (82.50) |
| Current steroid use, n (%) | 92 (100%) | 198 (84.26) | 499 (91.90%) |
| Ambulatory and timed function tests, mean ± SD | |||
| NSAA total score | 22.13 ± 6.03 22.50 [9, 34] |
24.54 ± 6.43 26.00 [10, 33] |
24.72 ± 5.93 26.00 [7, 34] |
| 6MWD (meters) | 348.71 ± 39.29 355.00 [216, 410] |
365.96 ± 87.76 376.00 [0, 620] |
- |
| 4-stair climb, time (seconds) | 5.1 ± 2.3 4.6 [1.5, 11.6] |
3.8 ± 2.4 3.2 [1.1, 11.9] |
2.6 ± 1.5 2.2 [0.8, 10.4] |
| 4-stair climb, velocity (stairs/second) | 0.97 ± 0.46 0.87 [0.34, 2.67] |
1.42 ± 0.75 1.27 [0.34, 3.77] |
1.96 ± 0.91 1.82 [0.38, 5.00] |
| Rise from supine, time (seconds) | 7.6 ± 3.8 6.6 [1.8, 17.2] |
6.5 ± 8.4 4.5 [1.4, 108.0] |
- |
| Rise from supine, velocity (1/seconds) | 0.15 ± 0.11 0.13 [0.00, 0.56] |
0.22 ± 0.14 0.21 [0.00, 0.74] |
|
| 10-meter walk/run, time (seconds) | 6.4 ± 1.7 6.2 [2.9, 10.9] |
5.5 ± 2.3 4.9 [1.5, 20.4] |
- |
| 10-meter walk/run, velocity (meters/second) | 1.69 ± 0.48 1.61 [0.92, 3.45] |
2.07 ± 0.69 2.04 [0.49, 6.54] |
|
| Sit to stand, time (seconds) | - | - | 2.9 ± 2.3 2.1 [0.9, 21.6] |
| Sit to stand, velocity (1/seconds) | - | - | 0.46 ± 0.20 0.48 [0.05, 1.11] |
| 30-foot walk/run, time (seconds) | - | - | 4.4 ± 1.1 4.1 [2.2, 10.7] |
| 30-foot walk/run, velocity (feet/second) | - | - | 7.28 ± 1.64 7.32 [2.80, 13.64] |
6MWD, 6-minute walk distance; BMI, body mass index; CCHMC, Cincinnati Children's Hospital Medical Center; cm, centimeters; DMD, Duchenne muscular dystrophy; kg, kilograms; m2, meters squared; n, number of intervals; NSAA, North Star Ambulatory Assessment; SD, standard deviation.
Timed function tests in baseline table are calculated without imputation.