Prednisone, not vamorolone, suppresses novel serum bone and cartilage biomarkers associated with growth failure in children with Duchenne muscular dystrophy.

What was studied

This study looked at blood markers of bone and cartilage change in children. The children had Duchenne muscular dystrophy and were 4 to under 7 years old. Blood samples came from a double-blind trial of vamorolone. In the first 24 weeks, children took placebo, prednisone, or vamorolone. In the next 24 weeks, the placebo and vamorolone groups all took vamorolone.

What they found

Bone markers ALP, osteocalcin, P1NP, and CTX1 fell with prednisone. They did not change with vamorolone or placebo. A protein screen found 10 proteins that fell with prednisone only. Genes for these proteins are known to cause bone or cartilage disorders. After the move from prednisone to vamorolone, all 10 markers went back to baseline.

What it means for patients

The authors say the 10 markers likely reflect cell death in cartilage and bone cells. They link this to usual corticosteroids. They say vamorolone may spare these cells. This fits with normal growth in children treated with vamorolone.

Why it is not certain yet

The abstract does not say how many children took part. The idea about cell death draws on animal studies. The authors use words like "likely" and "may". The study measured blood markers, not growth itself.

Source

Prednisone, not vamorolone, suppresses novel serum bone and cartilage biomarkers associated with growth failure in children with Duchenne muscular dystrophy.

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