Vamorolone Versus Traditional Glucocorticoids in Duchenne Muscular Dystrophy: A Review and Meta-Analysis of Efficacy and Safety.

Objective

This meta-analysis evaluates the efficacy and safety of vamorolone, a dissociative glucocorticoid, compared with traditional glucocorticoids in treating Duchenne muscular dystrophy (DMD), aiming to assess its potential as a safer alternative with comparable therapeutic benefits. Data Sources: A systematic search was conducted in PubMed (MEDLINE), Embase, and Web of Science from inception till June 2024. Search terms included (Vamorolone) AND (Corticosteroids OR Glucocorticoids OR Prednisone) AND (Duchenne Muscular Dystrophy OR related terms). Only randomized controlled trials (RCTs) and observational studies were included, with no language restrictions. Study Selection and Data Extraction: From 276 identified reports, 135 were screened after duplicate removal, and 30 underwent full-text review. Five studies (2 RCTs, 2 observational, 1 nonrandomized trial) involving 370 patients were included. Two reviewers independently extracted data on efficacy (TTSTAND, TTRW, TTCLIMB velocities) and safety (height percentile, body mass index-z score, osteocalcin, P1NP) using Microsoft Excel, resolving discrepancies through consensus. Data Synthesis: Vamorolone significantly improved TTSTAND (MD = -0.03, 95% confidence interval [CI] = -0.06 to -0.004, P = .02), TTRW (MD = -0.11, 95% CI = -0.22 to -0.01, P = .04), and TTCLIMB velocities (MD = -0.04, 95% CI = -0.08 to -0.003, P = .03). Safety outcomes showed enhanced height percentile (MD = 16.28, 95% CI = 6.31-26.25, P = .001) and bone biomarkers (osteocalcin: MD = 15.68, P < .00001; P1NP: MD = 158.34, P < .00001), with no significant body mass index-z score difference.

Conclusions

Vamorolone offers comparable efficacy with traditional glucocorticoids in DMD, with improved motor function and safer profiles in growth and bone health, suggesting its potential to transform DMD management.