Table 4.
The secondary endpoints at baseline and W24
| Patient ID | ADL Score | PedsQL TM 4.0 Score | PedsQL TM 3.0 NMM Score | PedsQL TM 2.0FIM Score | Parental Clinical Impression Scale | Blinded video rating | ||||
|---|---|---|---|---|---|---|---|---|---|---|
| W0 | W24 | W0 | W24 | W0 | W24 | W0 | W24 | |||
| 1 | 75.0 | 65.0 | 61.8 | 23.0 | 60.0 | 62.0 | 50.0 | 58.8 | Slightly worse | Slightly worse |
| 2 | 10.0 | 40.0 | 30.0 | 27.8 | 39.0 | 36.0 | 71.5 | 50.0 | Same | Slightly better |
| 3 | 25.0 | 15.0 | 36.0 | 39.5 | 48.0 | 49.0 | 58.8 | 69.0 | Same | Same |
| 4 | 75.0 | 85.0 | 52.8 | 88.0 | 75.0 | 92.0 | 85.8 | 85.3 | Slightly better | Slightly better |
| 5 | 85.0 | 80.0 | 67.5 | 56.5 | 62.0 | 56.0 | 63.5 | 81.8 | Slightly worse | Slightly worse |
| 6 | 10.0 | 10.0 | 29.0 | 25.0 | 35.0 | 30.0 | 50.0 | 73.8 | Slightly worse | Same |
| 7 | 45.0 | 45.0 | 42.5 | 45.0 | 52.0 | 40.0 | 29.0 | 45.3 | Slightly worse | Markedly worse |
| 8 | 50.0 | 55.0 | 61.3 | 60.0 | / | / | 55.5 | 53.5 | Slightly better | Slightly better |
| 9 | 95.0 | 95.0 | 59.8 | 58.8 | 68.0 | 72.0 | 70.3 | 75.8 | Slightly worse | Slightly worse |
| 10 | 25.0 | 25.0 | 16.0 | 42.5 | 38.0 | 62.0 | 41.3 | 40.0 | Same | Same |
| 11 | 85.0 | 90.0 | 35.0 | 73.9 | 77.0 | 81.0 | 50.0 | 59.5 | Slightly better | Slightly better |
| 12 | 50.0 | 20.0 | 23.8 | 38.3 | 31.0 | 44.0 | 55.5 | 79.8 | Markedly worse | Markedly worse |
| 13 | 35.0 | 30.0 | 65.8 | 35.0 | / | / | 41.3 | 40.0 | Slightly worse | Slightly worse |
| 14 | 50.0 | 40.0 | 35.8 | 22.5 | 40.0 | 45.0 | 58.8 | 60.8 | Slightly worse | Slightly worse |
| 15 | 50.0 | 35.0 | 36.0 | 44.8 | 52.0 | 49.0 | 32.5 | 46.5 | Markedly worse | Markedly worse |
| M ± SD | 49.3 ± 28.1 | 53.0 ± 25.1 | 55.2 ± 18.1 | 45.3 ± 19.1 | 52.1 ± 15.4 | 55.2 ± 18.1 | 54.2 ± 15.1 | 61.3 ± 15.4 | / | / |
| t | 1.361a | 0.332a | 1.142a | 2.306a | / | / | ||||
| P | 0.195a | 0.745a | 0.276a | 0.037a | / | / | ||||
“/” means no statistical analysis was performed
“a” means difference between W24 and W0, Paired t test was performed