Table 4.
The 48-week natural history data of 6MWD in placebo group patients (steroid-treated) evaluated every 6 weeks
| Baseline | 6 weeks | 12 weeks | 18 weeks | 24 weeks | 30 weeks | 36 weeks | 42 weeks | 48 weeks | ||
|---|---|---|---|---|---|---|---|---|---|---|
| <7 years 6MWD (m) | n | 6 | 6 | 6 | 6 | 6 | 6 | 6 | 6 | 6 |
| Mean | 382.8 | 417.3 | 413.5 | 425.5 | 418.0 | 410.1 | 405.9 | 427.6 | 416.9 | |
| SD | 42.7 | 55.1 | 30.7 | 30.7 | 32.9 | 25.9 | 35.6 | 40.6 | 35.1 | |
| Mean Δ | 0.0 | 34.5 | 30.7 | 42.7 | 35.2 | 27.4 | 23.1 | 44.8 | 34.1 | |
| SD Δ | 0.0 | 38.5 | 39.4 | 28.7 | 53.8 | 32.7 | 64.5 | 64.2 | 53.9 | |
| 6MWD percent predicted | n | 6.0 | 6.0 | 6.0 | 6.0 | 6.0 | 6.0 | 6.0 | 6.0 | 6.0 |
| Mean | 72.1 | 78.6 | 78.0 | 80.0 | 78.5 | 76.9 | 76.1 | 80.2 | 77.9 | |
| SD | 6.8 | 9.4 | 6.7 | 5.9 | 7.7 | 5.1 | 8.8 | 9.8 | 8.3 | |
| Mean Δ | 0.0 | 6.4 | 5.8 | 7.8 | 6.4 | 4.8 | 4.0 | 8.0 | 5.8 | |
| SD Δ | 0.0 | 7.2 | 7.6 | 5.5 | 10.4 | 6.4 | 12.4 | 12.4 | 10.5 | |
| ≥7 years | ||||||||||
| 6MWD (m) | n | 34 | 34 | 34 | 34 | 34 | 33 | 31 | 34 | 33 |
| Mean | 356.1 | 345.7 | 340.1 | 342.7 | 330.8 | 332.8 | 331.9 | 307.5 | 297.1 | |
| SD | 93.6 | 96.6 | 101.1 | 114.0 | 128.3 | 134.7 | 137.2 | 156.5 | 154.5 | |
| Mean Δ | 0.0 | –10.5 | –16.1 | –13.4 | –25.3 | –26.5 | –32.1 | –48.6 | –58.9 | |
| SD Δ | 0.0 | 29.2 | 33.8 | 42.8 | 59.0 | 66.1 | 68.5 | 88.1 | 81.9 | |
| 6MWD percent predicted | n | 34.0 | 34.0 | 33.0 | 33.0 | 34.0 | 33.0 | 31.0 | 34.0 | 33.0 |
| Mean | 58.5 | 57.2 | 57.0 | 56.6 | 54.8 | 55.3 | 55.1 | 51.0 | 49.4 | |
| SD | 16.7 | 17.0 | 17.7 | 20.3 | 22.3 | 23.3 | 23.7 | 26.8 | 26.3 | |
| Mean Δ | 0.0 | –1.3 | –2.1 | –2.0 | –3.7 | –3.9 | –4.8 | –7.6 | –9.3 | |
| SD Δ | 0.0 | 5.8 | 7.0 | 8.2 | 10.5 | 11.7 | 11.8 | 14.9 | 13.9 | |