Table 1 Comparison of deflazacort and placebo in secondary outcome measures (itt population)
From: Treatment of dysferlinopathy with deflazacort: a double-blind, placebo-controlled clinical trial
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 |