Comparing intermittent and daily prednisone in duchenne muscular dystrophy: a systematic review and meta-analysis

Duchenne muscular dystrophy (DMD) is an X-linked disorder caused by DMD gene mutations, leading to muscle wasting due to dystrophin deficiency. Current treatment with corticosteroids like prednisone shows benefits but lacks clarity on optimal dosing regimens. This systematic review and meta-analysis aim to determine the efficacy and safety of daily versus intermittent prednisone dosing in DMD management.

We conducted a systematic search of PubMed, Google Scholar, Embase, and Scopus databases to identify studies comparing daily versus intermittent prednisone in DMD treatment. The study protocol was registered with PROSPERO (CRD42024549050).

After the systematic search, 6 trials were included in the pooled analysis. Intermittent prednisone was associated with a higher prevalence of cushingoid appearance (RR: 1.72; 95% CI: 1.17 to 2.51; P = 0.005), excessive hair growth (RR: 1.56; 95% CI: 1.08 to 2.24; P = 0.02), and hypertension (RR: 3.42; 95% CI: 1.87 to 6.25; P < 0.0001). In contrast, there were no statistically significant differences between daily versus intermittent prednisone in terms of forced vital capacity (FVC), blood pressure, loss of ambulation, weight changes, weight gain, bone fracture, behavioral changes, and DEXA lumbar spine Z-scores.

No significant differences in efficacy outcomes regimens were observed. However, intermittent prednisone was associated with a higher prevalence of certain adverse effects, such as cushingoid appearance, excessive hair growth, and hypertension. These findings provide valuable insights for clinicians when choosing treatment strategies and highlight the need for personalized approaches to minimize side effects while maintaining efficacy.