Table 2.
The 48‐week change in ambulatory function in deflazacort and prednisone groups in the tadalafil DMD trial and ACT DMD placebo arms
| Deflazacort (95% CI) | Prednisone/prednisolone (95% CI) | Difference* (95% CI) | P value | |
|---|---|---|---|---|
| 6MWD (meters) | ||||
| Tadalafil DMD trial PBO | −43.2 (−75.0, −11.4) | −68.0 (−98.2, −37.7) | 24.8 (−7.9, 57.4) | 0.14 |
| ACT DMD PBO | −39.0 (−68.9, −9.2) | −70.6 (−97.2, −44.0) | 31.6 (0.2, 62.9) | 0.05 |
| 4‐stair climb (seconds) | ||||
| Tadalafil DMD trial PBO | 3.8 (1.2, 6.4) | 5.4 (2.9, 7.8) | −1.6 (−4.2, 1.1) | 0.25 |
| ACT DMD PBO | 3.8 (1.5, 6.0) | 6.7 (4.7, 8.6) | −2.9 (−5.3, −0.5) | 0.02 |
| Rise from supine (seconds) | ||||
| Tadalafil DMD trial PBO | 5.4 (2.3, 8.5) | 8.8 (5.9, 11.6) | −3.4 (−6.5, −0.2) | 0.04 |
| ACT DMD PBO | 4.5 (2.0, 7.0) | 7.1 (4.9, 9.3) | −2.6 (−5.2, 0.0) | 0.05 |
| 10‐meter walk/run (seconds) | ||||
| Tadalafil DMD trial PBO | 3.4 (0.8, 6.0) | 2.8 (0.2, 5.3) | 0.6 (−2.0, 3.3) | 0.65 |
| ACT DMD PBO | 3.2 (1.3, 5.0) | 3.3 (1.6, 4.9) | −0.1 (−2.1, 1.9) | 0.93 |
| NSAA total score | ||||
| Tadalafil DMD trial PBO | −3.4 (−5.2, −1.6) | −4.6 (−6.3, −3.0) | 1.2 (−0.6, 3.0) | 0.19 |
| ACT DMD PBO | −3.4 (−4.8, −2.0) | −4.5 (−5.8, −3.2) | 1.1 (−0.4, 2.6) | 0.14 |
| NSAA linearized score | ||||
| Tadalafil DMD trial PBO | −8.3 (−12.6, −4.0) | −11.9 (−15.8, −7.9) | 3.6 (−0.9, 8.0) | 0.12 |
| ACT DMD PBO | −8.2 (−10.9, −5.4) | −10.6 (−13.2, −8.0) | 2.5 (−1.2, 6.2) | 0.19 |
Abbreviations: 6MWD, 6‐minute walk distance; ACT DMD, Ataluren Confirmatory Trial in Duchenne Muscular Dystrophy; CI, confidence interval; PBO, placebo; NSAA, North Star Ambulatory Assessment.
*
Difference calculated as deflazacort − prednisone. Tadalafil DMD trial PBO estimates are from analyses of individual patient data. ACT DMD estimates are extracted from published results. For both trials, differences are based on least‐squares means of 48‐week change from baseline obtained from mixed model with repeated‐measures analyses.