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Meta-Analysis
. 2025 May;46(5):2249-2262.
doi: 10.1007/s10072-024-07939-1. Epub 2024 Dec 24.

Meta-analysis of the efficacy and safety of vamorolone in Duchenne muscular dystrophy

Mohamed Said Ibrahim  1   2 , Omar Ahmed Abdelwahab  3   4 , Bashaer Elawfi  3   5 , Fatmaelzahraa Yasser Ali  3   6 , Sarah Amro  3   7 , Shrouk F Mohammed  3   8 , Nour Shaheen  3   8 , Ahmed Negida  3   6   9   10 , Martin Arndt  11 , Mido Max Hijazi  12 , Jochen Schaefer  11 , Timo Siepmann  13   14
Affiliations
Meta-Analysis

Meta-analysis of the efficacy and safety of vamorolone in Duchenne muscular dystrophy

Mohamed Said Ibrahim et al. Neurol Sci. 2025 May.

Abstract

Background: Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder, often leading to wheelchair dependence by age 13 with limited treatment options, largely relying on glucocorticosteroids. We assessed the efficacy and safety of vamorolone, a modified synthetic corticosteroid, for DMD.

Methods: We performed a systematic review and meta-analysis using seven databases including prospective studies comparing vamorolone with glucocorticosteroids or placebo in DMD patients. We extracted data on efficacy and safety outcomes. We built fixed effects models to assess mean differences. (PROSPERO: CRD42023396908).

Results: Out of 210 identified records, two study reports were included in meta-analysis providing data from 210 patients. Vamorolone at 2.0 mg/kg/day was associated with improvement time to climb four stairs velocity (MD = 0.05 95% CI [0.03 to 0.08] P = 0.0002), and time stand from supine velocity (MD = 0.07 95% CI [0.01 to 0.07] P = 0.007). A higher dose of 6.0 mg/kg/day was additionally associated with higher time to run/walk 10 m velocity (MD = 0.10 95% CI [-0.0.1 to 0.21] P = 0.07, I2 = 0%). Among these beneficial effects only improvement in time to climb four stairs velocity was sustained after a follow-up period of 30 months. Vamorolone did not inhibit growth but increased the risk of weight gain, suppression of adrenal function, and insulin resistance.

Conclusion: The results of our systematic review and meta-analyis are suggestive of improved efficacy and safety of vamorolone for DMD compared to standard glucocorticosteroids but the external validity of these findings as well as the medication's long-term effects remain to be determined.

Keywords: 6MWT; Drug; Muscle; NSAA; Steroid; TTCLIMB; TTRW; TTSTAND; Vamorolone.

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Conflict of interest statement

Declarations. Ethical approval and Informed consent: Consent from the ethics committee was not required for the research. Informed consent was not applicable for this systematic review and meta-analysis. Informed consent: Not applicable. Conflicts of interest: The authors declare no conflict of interest.

Figures

Fig. 1
Fig. 1
PRISMA flow diagram of studies' screening and selection. Two of the five studies included in the review were also included in meta-analysis after exclusion of those with overlapping study populations
Fig. 2
Fig. 2
Forest plot comparing the efficacy outcomes of vamorolone 6 mg/kg/d and placebo or DNHS corticosteroid-naïve at 24 weeks
Fig. 3
Fig. 3
Forest plot comparing the efficacy outcomes of vamorolone 2 mg/kg/d and placebo or DNHS corticosteroid-naïve at 24 weeks
See this image and copyright information in PMC

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