Efficacy and Safety of Glucocorticoids in the Treatment of Progressive Muscular Dystrophy in Children: A Systematic Review and Meta-Analysis

    November 2021 in “ Translational pediatrics ”
    Ru‐Ping Liang, Yanan Wang, Yan Mei
    TLDR Glucocorticoids for progressive muscular dystrophy in children don't improve muscle strength or function but do increase certain side effects.
    The systematic review and meta-analysis of 5 articles involving 217 patient cases evaluated the efficacy and safety of glucocorticoids in treating progressive muscular dystrophy (PMD) in children. The study found that glucocorticoids can prolong the independent walking time of PMD patients for 2–5 years or longer, improve cardiopulmonary function, and reduce the risk of cardiomyopathy. However, glucocorticoids also significantly increased the risk of acne, excessive hair growth, emotional dysphoria, and other adverse effects. The study also highlighted the need for calcium, potassium, and vitamin D supplementation with glucocorticoids, and warned against abrupt discontinuation of the treatment. The study had limitations including small sample sizes, lack of data for children under 6, and differences in the types and doses of glucocorticoids used in each study. Further research is needed on patients with low age PMD and PMD patients who cannot walk independently.
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