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Questions {❓}

• Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better?

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         description: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. 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). 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. 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.
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            address:
               name:Medical Research Group of Egypt, Negida Academy, Arlington, USA
               type:PostalAddress
            type:Organization
            name:Al-Azhar University
            address:
               name:Faculty of Medicine, Al-Azhar University, Cairo, Egypt
               type:PostalAddress
            type:Organization
      name:Bashaer Elawfi
      affiliation:
            name:Negida Academy
            address:
               name:Medical Research Group of Egypt, Negida Academy, Arlington, USA
               type:PostalAddress
            type:Organization
            name:Sana’a University
            address:
               name:Faculty of Medicine, Sana’a University, Sana’a, Yemen
               type:PostalAddress
            type:Organization
      name:Fatmaelzahraa Yasser Ali
      affiliation:
            name:Negida Academy
            address:
               name:Medical Research Group of Egypt, Negida Academy, Arlington, USA
               type:PostalAddress
            type:Organization
            name:Zagazig University
            address:
               name:Faculty of Medicine, Zagazig University, Zagazig, Egypt
               type:PostalAddress
            type:Organization
      name:Sarah Amro
      affiliation:
            name:Negida Academy
            address:
               name:Medical Research Group of Egypt, Negida Academy, Arlington, USA
               type:PostalAddress
            type:Organization
            name:Al-Najah National University
            address:
               name:Al-Najah National University, Nablus, Palestine
               type:PostalAddress
            type:Organization
      name:Shrouk F. Mohammed
      affiliation:
            name:Negida Academy
            address:
               name:Medical Research Group of Egypt, Negida Academy, Arlington, USA
               type:PostalAddress
            type:Organization
            name:Alexandria University
            address:
               name:Faculty of Medicine, Alexandria University, Alexandria, Egypt
               type:PostalAddress
            type:Organization
      name:Nour Shaheen
      affiliation:
            name:Negida Academy
            address:
               name:Medical Research Group of Egypt, Negida Academy, Arlington, USA
               type:PostalAddress
            type:Organization
            name:Alexandria University
            address:
               name:Faculty of Medicine, Alexandria University, Alexandria, Egypt
               type:PostalAddress
            type:Organization
      name:Ahmed Negida
      affiliation:
            name:Negida Academy
            address:
               name:Medical Research Group of Egypt, Negida Academy, Arlington, USA
               type:PostalAddress
            type:Organization
            name:Zagazig University
            address:
               name:Faculty of Medicine, Zagazig University, Zagazig, Egypt
               type:PostalAddress
            type:Organization
            name:University of Portsmouth
            address:
               name:School of Pharmacy and Biomedical Sciences, University of Portsmouth, Portsmount, UK
               type:PostalAddress
            type:Organization
            name:Harvard Medical School
            address:
               name:Department of Global Health and Social Medicine, Harvard Medical School, Boston, USA
               type:PostalAddress
            type:Organization
      name:Martin Arndt
      affiliation:
            name:Medical Faculty and University Hospital Carl Gustav Carus, TUD Dresden University of Technology
            address:
               name:Department of Neurology, Medical Faculty and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany
               type:PostalAddress
            type:Organization
      name:Mido Max Hijazi
      affiliation:
            name:Medical Faculty and University Hospital Carl Gustav Carus, TUD Dresden University of Technology
            address:
               name:Department of Neurosurgery, Medical Faculty and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany
               type:PostalAddress
            type:Organization
      name:Jochen Schaefer
      affiliation:
            name:Medical Faculty and University Hospital Carl Gustav Carus, TUD Dresden University of Technology
            address:
               name:Department of Neurology, Medical Faculty and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany
               type:PostalAddress
            type:Organization
      name:Timo Siepmann
      url:http://orcid.org/0000-0003-2196-5904
      affiliation:
            name:Dresden International University, Division of Medicine
            address:
               name:Dresden International University, Division of Medicine, Dresden, Germany
               type:PostalAddress
            type:Organization
            name:Medical Faculty and University Hospital Carl Gustav Carus, TUD Dresden University of Technology
            address:
               name:Department of Neurology, Medical Faculty and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany
               type:PostalAddress
            type:Organization
      email:[email protected]
PostalAddress:
      name:Dresden International University, Division of Medicine, Dresden, Germany
      name:Medical Research Group of Egypt, Cairo, Egypt
      name:Medical Research Group of Egypt, Negida Academy, Arlington, USA
      name:Faculty of Medicine, Al-Azhar University, Cairo, Egypt
      name:Medical Research Group of Egypt, Negida Academy, Arlington, USA
      name:Faculty of Medicine, Sana’a University, Sana’a, Yemen
      name:Medical Research Group of Egypt, Negida Academy, Arlington, USA
      name:Faculty of Medicine, Zagazig University, Zagazig, Egypt
      name:Medical Research Group of Egypt, Negida Academy, Arlington, USA
      name:Al-Najah National University, Nablus, Palestine
      name:Medical Research Group of Egypt, Negida Academy, Arlington, USA
      name:Faculty of Medicine, Alexandria University, Alexandria, Egypt
      name:Medical Research Group of Egypt, Negida Academy, Arlington, USA
      name:Faculty of Medicine, Alexandria University, Alexandria, Egypt
      name:Medical Research Group of Egypt, Negida Academy, Arlington, USA
      name:Faculty of Medicine, Zagazig University, Zagazig, Egypt
      name:School of Pharmacy and Biomedical Sciences, University of Portsmouth, Portsmount, UK
      name:Department of Global Health and Social Medicine, Harvard Medical School, Boston, USA
      name:Department of Neurology, Medical Faculty and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany
      name:Department of Neurosurgery, Medical Faculty and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany
      name:Department of Neurology, Medical Faculty and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany
      name:Dresden International University, Division of Medicine, Dresden, Germany
      name:Department of Neurology, Medical Faculty and University Hospital Carl Gustav Carus, TUD Dresden University of Technology, Dresden, Germany

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