Skip to main content

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
  1. †: The column shows arithmetic mean ± standard deviations ± minimum and maximum values.
  2. *: 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.
  3. ‡: 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.