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

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Title: Meta-analysis of the efficacy and safety of vamorolone in Duchenne muscular dystrophy.
Authors: Ibrahim, Mohamed Said (AUTHOR), Abdelwahab, Omar Ahmed (AUTHOR), Elawfi, Bashaer (AUTHOR), Ali, Fatmaelzahraa Yasser (AUTHOR), Amro, Sarah (AUTHOR), Mohammed, Shrouk F. (AUTHOR), Shaheen, Nour (AUTHOR), Negida, Ahmed (AUTHOR), Arndt, Martin (AUTHOR), Hijazi, Mido Max (AUTHOR), Schaefer, Jochen (AUTHOR), Siepmann, Timo (AUTHOR)
Source: Neurological Sciences. May2025, Vol. 46 Issue 5, p2249-2262. 14p.
Subjects: Duchenne muscular dystrophy, Fixed effects model, Neuromuscular diseases, Weight gain, Insulin resistance
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. [ABSTRACT FROM AUTHOR]
Copyright of Neurological Sciences is the property of Springer Nature and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)
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  Data: Meta-analysis of the efficacy and safety of vamorolone in Duchenne muscular dystrophy.
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  Data: <searchLink fieldCode="AR" term="%22Ibrahim%2C+Mohamed+Said%22">Ibrahim, Mohamed Said</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Abdelwahab%2C+Omar+Ahmed%22">Abdelwahab, Omar Ahmed</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Elawfi%2C+Bashaer%22">Elawfi, Bashaer</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Ali%2C+Fatmaelzahraa+Yasser%22">Ali, Fatmaelzahraa Yasser</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Amro%2C+Sarah%22">Amro, Sarah</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Mohammed%2C+Shrouk+F%2E%22">Mohammed, Shrouk F.</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Shaheen%2C+Nour%22">Shaheen, Nour</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Negida%2C+Ahmed%22">Negida, Ahmed</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Arndt%2C+Martin%22">Arndt, Martin</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Hijazi%2C+Mido+Max%22">Hijazi, Mido Max</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Schaefer%2C+Jochen%22">Schaefer, Jochen</searchLink> (AUTHOR)<br /><searchLink fieldCode="AR" term="%22Siepmann%2C+Timo%22">Siepmann, Timo</searchLink> (AUTHOR)
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  Data: <searchLink fieldCode="JN" term="%22Neurological+Sciences%22">Neurological Sciences</searchLink>. May2025, Vol. 46 Issue 5, p2249-2262. 14p.
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  Data: 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. [ABSTRACT FROM AUTHOR]
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  Data: <i>Copyright of Neurological Sciences is the property of Springer Nature and its content may not be copied or emailed to multiple sites without the copyright holder's express written permission. Additionally, content may not be used with any artificial intelligence tools or machine learning technologies. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract.</i> (Copyright applies to all Abstracts.)
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