Pharmacological and non-pharmacological therapies for prevention and treatment of osteoporosis in Duchenne Muscular Dystrophy: A systematic review

Background

Long term glucocorticoid treatment in Duchenne Muscular Dystrophy (DMD) is associated with a high incidence of fragility fractures. This systematic review aims to assess the current evidence for pharmacological and non-pharmacological treatment for osteoporosis in children and adults with DMD.

Methods

Three online databases (Embase, Medline, Cochrane Library) were searched for studies that evaluated interventions for treatment or prevention of osteoporosis in DMD. Included studies had to report changes in bone mineral density (BMD) or bone mineral content (BMC) Z-scores or fracture incidence.

Results

Nineteen studies were identified, including twelve that evaluated bisphosphonate, three evaluated testosterone (2 studies of the same patient group), one evaluated vitamin D/calcium, one teriparatide, and two evaluated vibration therapy. Only two randomised-controlled trials were found, one of intravenous bisphosphonate and one of vibration therapy. Changes in lumbar spine BMD ranged from −0.3 to +1.3 in studies of bisphosphonate and − 0.2 to 0.0 with vibration therapy, whereas this was +0.38 with testosterone and + 0.9 with vitamin D/calcium. There was limited information on impact on fracture in all studies. None of the pharmacological studies involved a fracture naïve group at baseline. In addition, none addressed a group of individuals over 18 years at baseline.

Conclusion

This systematic review provides evidence for the effectiveness of bisphosphonate therapy in improving bone density in children and adolescents with DMD. However, there is less information on the impact on fracture. The review did not find studies exclusively in those over 18 years old with DMD and limited information on non-bisphosphonate pharmacological agents.