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Introduction <p>Currently, there is a lack of meta-analysis providing precise incidence estimates of adverse drug reactions (ADRs) and associated risk differences (RDs) linked to pharmacological treatments for muscle degeneration in Duchenne Muscular Dystrophy (DMD).</p> Methods <p>A systematic search of PubMed, Embase, and ClinicalTrials.gov (January 1, 2000–June 5, 2026) identified clinical trials evaluating safety of pharmacological treatments for muscle degeneration in DMD. A random-effects meta-analysis estimated ADR incidence, and RDs versus a common reference (givinostat) were derived from the pooled incidences rather than from conventional head-to-head treatment contrasts.</p> Results <p>Thirteen clinical trials were included. Heterogeneity in ADR incidence across treatments (<i>p</i> &lt; 0.05) was identified. Prednisone and prednisolone showed the highest proportions of serious ADRs. Discrepancies were found compared to Summary of Product Characteristics (SmPC): deflazacort showed higher rates of erythema (+ 1.5%) and pollakiuria (+ 4.8%), while vamorolone showed increased Cushingoid features and appetite (+ 7.0%). Subgroup analyses showed dose- and duration-dependent ADRs for deflazacort and prednisone. A reference-anchored exploratory comparison of pooled incidences showed lower gastrointestinal and hematological ADR risks with corticosteroid monotherapy than with givinostat plus corticosteroids. Deflazacort showed lower risks of diarrhea (RD: − 0.3860), decreased appetite (RD: − 0.3160), and decreased platelet count (RD: − 0.4110).</p> Conclusion <p>Precise ADR estimates can support decision-making in DMD care, helping clinicians balance safety and efficacy, address family concerns, and promote adherence by contextualizing treatment risks.</p>

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Meta-analysis of clinical trials assessing the safety of pharmacological treatments for muscular degeneration in Duchenne muscular dystrophy

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年6月30日

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  • Gerard Ompad,
  • Petra Maria Saura,
  • Nicole Sonne Heckmann,
  • Andrea Rossi,
  • Elena Olmastroni,
  • Manuela Casula,
  • Maurizio Sessa

摘要

Introduction

Currently, there is a lack of meta-analysis providing precise incidence estimates of adverse drug reactions (ADRs) and associated risk differences (RDs) linked to pharmacological treatments for muscle degeneration in Duchenne Muscular Dystrophy (DMD).

Methods

A systematic search of PubMed, Embase, and ClinicalTrials.gov (January 1, 2000–June 5, 2026) identified clinical trials evaluating safety of pharmacological treatments for muscle degeneration in DMD. A random-effects meta-analysis estimated ADR incidence, and RDs versus a common reference (givinostat) were derived from the pooled incidences rather than from conventional head-to-head treatment contrasts.

Results

Thirteen clinical trials were included. Heterogeneity in ADR incidence across treatments (p < 0.05) was identified. Prednisone and prednisolone showed the highest proportions of serious ADRs. Discrepancies were found compared to Summary of Product Characteristics (SmPC): deflazacort showed higher rates of erythema (+ 1.5%) and pollakiuria (+ 4.8%), while vamorolone showed increased Cushingoid features and appetite (+ 7.0%). Subgroup analyses showed dose- and duration-dependent ADRs for deflazacort and prednisone. A reference-anchored exploratory comparison of pooled incidences showed lower gastrointestinal and hematological ADR risks with corticosteroid monotherapy than with givinostat plus corticosteroids. Deflazacort showed lower risks of diarrhea (RD: − 0.3860), decreased appetite (RD: − 0.3160), and decreased platelet count (RD: − 0.4110).

Conclusion

Precise ADR estimates can support decision-making in DMD care, helping clinicians balance safety and efficacy, address family concerns, and promote adherence by contextualizing treatment risks.