Endocrine Abstracts

Background: Glucocorticoid treatment is commonly used for Duchenne Muscular Dystrophy (DMD) in young people, but 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, with the goal of guiding future management strategies.

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

Results: Seventeen studies were identified, including eleven on bisphosphonate treatment, two on testosterone therapy, one on vitamin D/calcium, one on teriparatide treatment, and two on vibration therapy. Only two randomised-controlled trials were found, one for bisphosphonate (zoledronic acid) with 24-month follow-up and one for vibration therapy with 14-month follow-up. Among the bisphosphonate studies, four investigated intravenous administration, five oral administration, one intramuscular administration, and one both intravenous and oral administration. Changes in lumbar spine BMD Z-scores ranged from −0.3 to +1.3 with bisphosphonate treatment, from +0.13 to +0.38 with testosterone therapy, +0.9 with vitamin D/calcium, and from −0.2 to 0.0 with vibration therapy. The zoledronic acid trial showed a significant difference of +1.4 standard deviations in lumbar spine BMD Z-scores between the treatment and no treatment groups. Limited information was available regarding the impact of treatment on fracture incidence, although data from the randomised trial of intravenous zoledronic acid showed a lower rate of vertebral fractures in the treatment group (15%) compared to the no treatment group (24%). None of the studies involved a population without prior fractures, and none addressed individuals over 18 years.

Conclusion: This systematic review provides emerging evidence for the effectiveness of bisphosphonate therapy in improving bone density in children and adolescents with DMD. However, there is limited information regarding fracture reduction. The review did not find studies involving individuals without prior fractures or those over 18 years old with DMD. Developing new clinical guidance for osteoporosis in children and adults with DMD will require expert consensus in conjunction with published evidence.