Few Studies Explore How to Improve Duchenne MD Patients’ Bone Health, Analysis Finds

Bone fragility is a serious consequence of long-term corticosteroid treatment — the main option for Duchenne muscular dystrophy (DMD) — but an analysis found that there are no high-quality studies on how to improve patients’ bone health.

The analysis, “Interventions to prevent and treat corticosteroid-induced osteoporosis and prevent osteoporotic fractures in Duchenne muscular dystrophy (Review),” was published by the Cochrane Library.

Long-term corticosteroid use causes bones to lose many of the minerals they need for strength. As a result, they become brittle and can break easily.

For their analysis, researchers at Queen’s University Belfast searched for published studies of ways to improve DMD patients’ bone mineral density. The quasi-randomized (not truly randomized) or randomized, controlled trials could include children, adolescents, or adults.

Of the 18 potential studies the researchers initially identified, only two were eligible for their analysis. The researchers were unable to assess the trials’ quality because only abstracts were available.

The first study included 13 participants and compared risedronate (available as Actonel and Atelvia) to no treatment. The second study assessed the effect of whole-body vibration on 21 patients. The vibration method was compared to the effects of a placebo device.

Both trials reported an improvement in children who received treatment, and no improvement in the control (placebo or no treatment) groups. But the abstracts did not provide details comparing the groups, so researchers could not draw conclusions about either treatment’s effectiveness.

Neither study provided information about adverse events.

The research team also found two ongoing, randomized clinical trials. One examined whole-body vibration, and the other compared zoledronic acid (available under brand name Zometa in the U.S.) to vitamin D and calcium in Duchenne children and adolescents.

The lack of further information prevented the team from performing a so-called meta-analysis, in which data from various studies is pooled and analyzed.

“Findings from two ongoing relevant studies and two trials, for which only abstracts are available, will be important in future updates of this review,” the authors wrote.