A two-year study reports that boys with Duchenne muscular dystrophy (DMD) that are on a long-term regime of glucocorticoids (GCs) are more likely to develop spinal fractures, have poorer growth and gain more body fat than those on intermittent use.
However, the hypothesis suggested that taking oral GCs every day tends to prolong their ability to walk. The researchers stress that children with DMD should be routinely screened for bone health and vertebral fractures using suitable imaging techniques.
Treatment with GCs usually starts between ages four and six, when the motor capacities of these patients peak and stop improving.
One study, based on eight years of data covering 79 DMD patients, reported that 37 of these boys who never were treated with Emflaza also never developed any spinal fractures, while these fractures were found in treated patients.
The untreated group also had poorer cardiac function and more severe scoliosis, among other findings.
Researchers conducted a retrospective study to compare growth, body mass, bone mineral density. Vertebral fractures, and walking ability in DMD children on a daily or intermittent GCs regime.
Compared to at the start of the study, the boys on daily GCs showed a significantly shorter stature and their height gradually decreased over two and a half years. By contrast, the stature of boys receiving intermittent GCs remained unchanged.
In spite of their reduced growth, boys on daily GCs increased their body mass index to significantly higher levels than those on an intermittent regimen, suggesting these children gained more fat mass.
40 per cent of boys on daily GC use developed vertebral fractures compared to eight per cent on intermittent GC treatment.
No significant differences were seen in the number of long bone fractures, which were rare.
There was a trend for daily GC treatment to prolong a patient’s ability to walk, the researchers reported. At the final assessment at two years of follow-up, 10 boys on the intermittent regimen and five on the daily use had lost this ability.
“In conclusion, there was a trend for more boys on daily GCs to remain ambulant [able to walk] but at the cost of more VFs [vertebral fractures], greater adiposity and markedly diminished growth,” the researchers wrote.