Prednisone Linked to Impaired Growth in Muscular Dystrophy

Study Finds Blood Biomarkers Tied to Prednisone Use in DMD

An international research team led by Binghamton University identified a set of blood proteins that may explain why prednisone, a corticosteroid used to treat Duchenne muscular dystrophy (DMD), can impair childhood growth and bone health.

The findings come from a double-blind clinical trial in boys aged 4 to 7 with DMD, comparing prednisone, vamorolone, and placebo over two 24-week periods.

What the Clinical Trial Showed

During the first 24 weeks, participants received either placebo, prednisone at 0.75 mg/kg/day, or vamorolone at 6 mg/kg/day. In the second 24 weeks, those on placebo or prednisone switched to vamorolone.

Prednisone significantly reduced markers of bone formation, bone turnover, and growth-plate activity, while vamorolone and placebo did not show these changes. When prednisone was stopped and replaced with vamorolone, the suppressed biomarkers returned toward pretreatment levels.

Key Biomarkers and Their Significance

The study tracked four standard bone formation markers: alkaline phosphatase (ALP), osteocalcin, procollagen type I N-terminal propeptide (P1NP), and collagen type I C-terminal telopeptide (CTX1). All declined during prednisone treatment but remained stable with vamorolone or placebo.

A broader protein screen found 10 additional proteins reduced by prednisone, many linked to bone and cartilage health. These proteins are important for skeletal development and bone density.

Practical Implications for Metabolic Health Management

For families and clinicians managing DMD, these findings clarify how prednisone may slow growth by affecting bone and cartilage proteins, offering a potential explanation for observed side effects.

The study suggests that vamorolone may be less likely to impair growth or bone health, as it did not produce the same biomarker changes. Both drugs improved motor outcomes, but only prednisone showed the adverse biomarker pattern.

Limitations and Considerations

The trial included only boys aged 4 to 7 with Duchenne muscular dystrophy, so findings may not apply to other ages or conditions.

The study measured blood biomarkers rather than direct clinical outcomes like final height or fracture risk. The reversibility of biomarker changes after switching drugs suggests a drug effect, but longer-term impacts need further study.