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

What was studied

This review pooled safety data on drugs used in Duchenne muscular dystrophy. It estimated how often adverse drug reactions occur. It used givinostat as a shared reference. The team searched PubMed, Embase and ClinicalTrials.gov.

What they found

Rates of reactions differed across drugs. Prednisone and prednisolone had the highest rates of serious reactions. Some rates were higher than the product labels list. Deflazacort showed more erythema and pollakiuria. Vamorolone showed more Cushingoid features and appetite change. For deflazacort and prednisone, some reactions grew with dose and time.

What it means for patients

The team says precise estimates can help doctors weigh safety and benefit. They say it can help address family worries. They say it may also help families keep to treatment.

Why it is not certain yet

The comparison with givinostat was exploratory. It used pooled rates, not head-to-head trials. The abstract does not give trial sizes. It does not give trial lengths. Details of the methods are brief. This text uses the abstract and nothing else. Not every fact is in the abstract. Some of the work is not described there.

Source

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

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