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

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.