Table 3. Models for Δ4SC velocity in each data source.
| Tadalafil DMD Trial Placebo Arm (n = 92) | Leuven (n = 235) | CCHMC (n = 543) | ||||
|---|---|---|---|---|---|---|
| Base model | Full model | Base model | Full model | Base model | Full model | |
| Coefficient (95% CI) | Coefficient (95% CI) | Coefficient (95% CI) | Coefficient (95% CI) | Coefficient (95% CI) | Coefficient (95% CI) | |
| Intercept | -1 (-1.88, -0.13)* | 5.53 (-0.07, 11.13) | 0.98 (0.66, 1.29) | 0.74 (-3.19, 4.66) | 0.77 (0.55, 0.99)*** | 3.02 (1.15, 4.89)** |
| Age (years) | 0.03 (-0.02, 0.08) | 0.04 (-0.02, 0.09) | -0.08 (-0.11, -0.05)*** | -0.03 (-0.07, 0.01) | -0.06 (-0.08, -0.04)*** | -0.05 (-0.09, -0.02)** |
| Steroids ≥ 1 year 1 vs. 0 | 0.26 (-0.05, 0.56) | 0.42 (0.16, 0.69)** | -0.15 (-0.33, 0.03) | -0.29 (-0.47, -0.11)** | -0.16 (-0.28, -0.04)** | -0.24 (-0.37, -0.10)*** |
| Timed 4-stair climb velocity | 0.29 (-0.09, 0.66) | -0.17 (-0.66, 0.31) | -0.16 (-0.25, -0.07)*** | -0.6 (-0.74, -0.46)*** | -0.12 (-0.16, -0.07)*** | -0.42 (-0.50, -0.34)*** |
| Current deflazacort 1 vs. 0 | 0.16 (-0.01, 0.33) | 0.16 (-0.02, 0.34) | 0.01 (-0.12, 0.14) | |||
| Timed 10-meter walk/run velocity | 0.22 (-0.07, 0.50) | 0.27 (0.17, 0.37)*** | - | |||
| Timed rise from supine velocity | 1.41 (0.25, 2.56)* | 1.83 (1.13, 2.53)*** | - | |||
| Timed 30-foot walk/run velocity | - | - | - | - | 0.13 (0.08, 0.18)*** | |
| Timed sit to stand velocity | - | - | - | - | 0.72 (0.31, 1.13)*** | |
| BMI (kg/m2) | -0.19 (-0.33, -0.05)** | 0.02 (-0.08, 0.12) | -0.1 (-0.14, -0.05)*** | |||
| Height (cm) | -0.05 (-0.10, -0.01)* | -0.01 (-0.04, 0.03) | -0.02 (-0.04, -0.01)** | |||
| Weight (kg) | 0.11 (0.03, 0.20)** | -0.01 (-0.06, 0.05) | 0.06 (0.03, 0.08)*** | |||
| Model R2 (adjusted R2) | 0.08 (0.05) | 0.29 (0.21) | 0.17 (0.16) | 0.36 (0.33) | 0.16 (0.16) | 0.30 (0.29) |
| RMSE | 0.39 | 0.35 | 0.59 | 0.52 | 0.51 | 0.47 |
Δ4SC, annualized change in 4-stair climb; BMI, body mass index; CCHMC, Cincinnati Children's Hospital Medical Center; CI, confidence interval; cm, centimeters; kg, kilograms; m2, meters squared; n, number of participants; RMSE, root-mean squared error. Statistical significance: *** p < 0.001, ** p < 0.01, * p <0.05