What question did this study set out to answer?

This research aims to evaluate the efficacy and safety of vamorolone in treating Duchenne muscular dystrophy at different doses.

April 18, 2026Open Access

Dose Dependent Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A Systematic Review and Meta Analysis of Randomized Controlled Trials

Puntos clave

This research aims to evaluate the efficacy and safety of vamorolone in treating Duchenne muscular dystrophy at different doses.
Conducted a systematic search in multiple databases for relevant randomized controlled trials.
Compared vamorolone doses of 2 mg/kg/day versus 6 mg/kg/day in boys with DMD.
Assessed primary motor outcomes like time to stand, walk distance, and stair climb, as well as changes in BMI z score.
Six mg/kg/day dose of vamorolone improved motor outcomes significantly compared to 2 mg/kg/day.
Motor outcomes included TTSTAND, 6-MWD, TTRW, and TTCLIMB with statistically significant differences in improvement.
Changes in BMI z scores were similar between the two dosage groups.

Resumen

Background: Duchenne muscular dystrophy (DMD) is a severe inherited neuromuscular disorder causing progressive muscle weakness in boys. Corticosteroids are standard therapy but have significant long-term adverse effects. Vamorolone, a dissociative steroid, may preserve efficacy while reducing toxicity. This systematic review and meta-analysis evaluated whether higher doses improve motor outcomes while maintaining safety in boys with DMD. Methods: A systematic search of PubMed, Web of Science, Embase, Scopus, and the Cochrane Library was conducted to identify randomized controlled trials comparing vamorolone at 2 mg/kg/day versus 6 mg/kg/day in boys with DMD. Primary motor outcomes included time to stand from supine (TTSTAND), six-minute walk distance (6-MWD), time to run or walk 10 meters (TTRW), and time to climb four stairs (TTCLIMB). Changes in BMI z score were assessed as a safety indicator. Data were analyzed using Review Manager (RevMan) 5.4 following PRISMA guidelines. Results: Three randomized controlled trials involving 118 boys met the inclusion criteria. The 6 mg/kg/day dose demonstrated significantly greater improvement in motor outcomes compared with the 2 mg/kg/day dose, including TTSTAND (MD = 0.04, 95% CI 0.020.07, p 0.0001), 6-MWD (MD = 26.27, 95% CI 1.5550.99, p = 0.04), TTRW (MD = 0.13, 95% CI 0.070.19, p 0.0001), and TTCLIMB (MD = 0.04, 95% CI 0.010.07, p = 0.006). BMI z score changes were comparable between groups. Conclusion: Vamorolone 6 mg/kg/day improves motor function more than 2 mg/kg/day without increased safety concerns. Larger long-term trials are required to confirm these findings.

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