Table 1.
Comparison of deflazacort and placebo in secondary outcome measures (itt population)
| Outcome measures |
Baseline† |
Natural course |
Deflazacort (pooled) |
Placebo (pooled) |
p-value |
|---|---|---|---|---|---|
| (N = 23) |
End of year 1 – begin* |
End – begin of crossover* |
End – begin of crossover* |
Deflazacort vs. placebo‡ |
|
| (N = 23) | (N = 21) | (N = 21) | (N = 19) | ||
| Muscle strength (CIDD) |
78.5 ± 9.6 (56–89) |
−2.0 ± 2.5 |
−2.2 ± 3.0 |
- 1.0 ± 2.2 |
0.4375 |
| Hand-held dynamometry (HDD) (N) |
596 ± 267 (117–1206) |
−72.2 ± 109.6 |
−67.7 ± 67.6 |
- 26.7 ± 73.9 |
0.1309 |
| QSMT – Torque Measurement |
|
|
|
|
|
| Handgrip left (kg) |
20.1 ± 10.3 (8–52) |
−2.2 ± 3.6 |
−1.9 ± 2.9 |
−0.5 ± 2.5 |
0.4852 |
| Handgrip right (kg) |
20.8 ± (10.3) (8–50) |
−2.5 ± 3.5 |
−2.3 ± 2.3 |
−1.2 ± 2.6 |
0.7408 |
| Quadriceps (Nm) |
136 ± 59 (31–220) |
−11.4 ± 90.4 |
−17.5 ± 55.1 |
−25.5 ± 49.5 |
0.9021 |
| Biceps (Nm) |
203 ± 133 (11–310) |
+12.2 ± 186.9 |
−36.0 ± 120.6 |
−63.4 ± 114.4 |
0.3456 |
| Neuromuscular Symptom Score |
35.4 ± 4.9 (23–41) |
−0.3 ± 1.6 |
−2.4 ± 5.0 |
−0.4 ± 1.5 |
0.1467 |
| Timed Function Test (sec.) |
|
|
|
|
|
| Stair climbing |
6.8 ± 5,4 (3–25) |
+0.8 ± 1.8 |
+0.3 ± 1.1 |
+0.5 ± 1.7 |
0.8478 |
| Running 10 meters |
16.9 ± 5.9 (8–30) |
+1.6 ± 2.2 |
+1.8 ± 1.8 |
+1.0 ± 1.8 |
0.3689 |
| Getting up from sitting |
2.0 ± 2.3 (0.5-10) |
+0.3 ± 1.4 |
+0.4 ± 1.0 |
+0.6 ± 1.6 |
0.4875 |
| Getting up from lying |
4.1 ± 4,8 (1–20) |
+0.5 ± 1.1 |
+1.3 ± 3.2 |
+0.5 ± 1.2 |
0.3909 |
| Vignos Scale |
2.4 ± 0.6 (2–4) |
+0.2 ± 0.6 |
+0.3 ± 0.6 |
0 ± 0.3 |
0.0712 |
| Hammersmith Motor Ability Score |
23.8 ± 6.1 (10–35) |
−2.3 ± 3.1 |
−2.1 ± 2.3 |
−1.0 ± 1.5 |
0.0578 |
| Clinical Global Impressions – severity | 4.5 ± 1.4 (3–6) | −0.2 ± 1.0 | +0.3 ± 0.6 | +0.1 ± 1.0 | 0.3517 |
†: The column shows arithmetic mean ± standard deviations ± minimum and maximum values.
*: The column shows arithmetic mean ± standard deviations for changes between end and begin of each study period. Deflazacort and placebo values are pooled across both crossover periods.
‡: p-values associated with the treatment effect in Lehmacher’s (1991) proposal for the non-parametric analysis of crossover-data (deflazacort vs. Placebo). Neither carry-over nor period effects were detected in any analysis.