Table 2.
Comparison of efficacy outcomes of givinostat
| Parameter (unit) | Intervention group (95% CI) | Control group (95% CI) | Mean difference (effect size) | Clinical impact of givinostat |
|---|---|---|---|---|
| DMD (EPIDYS) (Mercuri et al. [35], 2024) | ||||
| Four-stair climb, s | 1.27 (1.17 to 1.37) | 1.48 (1.32 to 1.66) | −0.21 | Reduced rate of functional decline in stair-climbing ability |
| MRS VLFF, % | 7.63 (6.10 to 9.17) | 10.56 (8.33 to 12.78) | −2.93 | Attenuation of muscle fat infiltration, indicating slower disease progression |
| Time to rise, s | 9.33 (5.82 to 12.84) | 12.61 (7.49 to 17.72) | −3.28 | Slower progression in the time required to rise from the floor |
| 6-min walk test, m | −38.4 (−50.7 to −26.2) | −48.4 (−66.3 to −30.5) | +10.0 | Improvement in walking endurance compared to the control group |
| Knee extension, N/kg | −0.32 (−0.44 to −0.20) | −0.50 (−0.68 to −0.33) | +0.18 | Reduced loss of muscle strength in knee extension |
| Elbow flexion, N/kg | −0.10 (−0.17 to −0.03) | −0.19 (−0.29 to −0.09) | +0.09 | Reduced loss of muscle strength in elbow flexion |
| NSAA: total score | −2.66 (−3.56 to −1.76) | −4.58 (−5.89 to −3.26) | +1.92 | Lesser functional decline as measured by the (NSAA) score |
| BMD (Comi et al. [30], 2023) | ||||
| Four-stair climb, s | 0.87 (0.71 to 1.05) | 0.88 (0.67 to 1.16) | −0.01 | Comparable maintenance of stair-climbing ability between groups |
| MRS VLFF, % | 1.02 (0.95 to 1.09) | 1.07 (0.98 to 1.16) | −0.05 | Stabilization of muscle fat fraction, suggesting slowed disease progression |
| Time to rise, s | 1.39 (−0.97 to 3.75) | 0.77 (−3.01 to 4.56) | +0.62 | No significant difference observed in the time required to rise from the floor |
| 6-min walk test, m | 0.94 (0.89 to 1.00) | 0.96 (0.88 to 1.04) | −0.02 | No clinically meaningful difference observed in walking endurance between groups |
MRS VLFF, vastus lateralis fat fraction measured by magnetic resonance spectroscopy.