Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
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.

PubMed Disclaimer

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

References

    1. Birnkrant DJ, Bushby K, Bann CM, Apkon SD, Blackwell A, Brumbaugh D, Case LE, Clemens PR, Hadjiyannakis S, Pandya S, Street N, Tomezsko J, Wagner KR, Ward LM, Weber DR, DMD Care Considerations Working Group (2018). Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol 17(3):251–267. 10.1016/S1474-4422(18)30024-3 - PMC - PubMed
    1. Crisafulli S, Sultana J, Fontana A, Salvo F, Messina S, Trifirò G (2020) Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis. Orphanet J Rare Dis 15(1):141. 10.1186/s13023-020-01430-8 - PMC - PubMed
    1. Ferizovic N, Summers J, de Zárate IBO, Werner C, Jiang J, Landfeldt E, Buesch K (2022) Prognostic indicators of disease progression in Duchenne muscular dystrophy: a literature review and evidence synthesis. PLoS ONE 17(3):e0265879. 10.1371/journal.pone.0265879 - PMC - PubMed
    1. Verhaart IEC, Aartsma-Rus A (2019) Therapeutic developments for Duchenne muscular dystrophy. Nat Rev Neurol 15(7):373–386. 10.1038/s41582-019-0203-3 - PubMed
    1. Ma J, McMillan HJ, Karagüzel G, Goodin C, Wasson J, Matzinger MA, DesClouds P, Cram D, Page M, Konji VN, Lentle B, Ward LM (2017) The time to and determinants of first fractures in boys with Duchenne muscular dystrophy. Osteoporos Int 28(2):597–608. 10.1007/s00198-016-3774-5 - PubMed

LinkOut - more resources