SynthesisBMC pharmacology & toxicology2026
Meta-analysis of clinical trials assessing the safety of pharmacological treatments for muscular degeneration in Duchenne muscular dystrophy.
Synthesis in BMC pharmacology & toxicology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionCurrently, 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).
methodsA 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.
resultsThirteen 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).
conclusionPrecise 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.
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