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Published on: May 24, 2016
Oral bisphosphonate treatment in patients with Duchenne muscular dystrophy on long term glucocorticoid therapy
Cuixia Tian1, Brenda L Wong1, Lindsey Hornung2
1Division of Neurology, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH 45229, USA.; Department of Pediatrics, University of Cincinnati College of Medicine, 2600 Clifton Ave, Cincinnati, OH 45220, USA.
Abstract:
Osteoporosis is a major problem in patients with Duchenne muscular dystrophy (DMD), due to glucocorticoid therapy and muscle weakness. Evidence on which to base optimal prevention and treatment strategies, including bisphosphonate use, in DMD are limited. Our objective was to describe bone health outcomes of oral alendronate treatment in patients with DMD and glucocorticoid-induced osteoporosis. We retrospectively studied 54 patients treated between 2005 and 2017, and assessed changes in dual-energy x-ray absorptiometry (DXA) whole body and lumbar spine bone mineral density and content, and lateral distal femur bone mineral density. We also examined vertebral fracture development in a subset with serial spine radiographs. Pre-alendronate DXA Z-score trajectories decreased progressively. Over three years post-alendronate initiation, Z-score trajectories improved (p<0.01) at most sites compared with pre-alendronate trajectories. Height-adjusted Z-score trajectories for lumbar spine bone mineral density (p = 0.01) and whole body bone mineral content (p = 0.0004) also improved. The positive trajectories did not seem to be sustained long term in those treated up to 6 years. Radiographic vertebral findings in 43 patients appeared stable. In conclusion, oral bisphosphonate therapy using alendronate was associated with improvement of DXA bone health indices during the first three years of treatment, and may help mitigate progression of osteoporosis in glucocorticoid-treated patients with DMD.
Insights
Oral alendronate improved bone density in Duchenne muscular dystrophy (DMD) patients with osteoporosis. While initial gains were seen within three years, long-term benefits of bisphosphonate therapy require further investigation.
Area of Science:
- Bone Metabolism and Musculoskeletal Disorders
- Pediatric Endocrinology
- Pharmacology
Background:
- Osteoporosis is a significant complication in Duchenne muscular dystrophy (DMD) patients, often exacerbated by glucocorticoid therapy and muscle weakness.
- Limited evidence exists for optimal osteoporosis prevention and treatment strategies, including bisphosphonate use, in the DMD population.
Purpose of the Study:
- To evaluate bone health outcomes in DMD patients with glucocorticoid-induced osteoporosis treated with oral alendronate.
- To assess the impact of alendronate on bone mineral density (BMD) and vertebral fracture incidence.
Main Methods:
- Retrospective study of 54 DMD patients treated with oral alendronate between 2005 and 2017.
- Assessment of dual-energy x-ray absorptiometry (DXA) for whole body, lumbar spine BMD, and lateral distal femur BMD.
- Evaluation of serial spine radiographs for vertebral fracture development in a subset of patients.
Main Results:
- Pre-alendronate DXA Z-score trajectories showed progressive decline.
- Over three years, Z-score trajectories improved significantly at most sites post-alendronate initiation (p<0.01).
- Height-adjusted Z-scores for lumbar spine BMD and whole body bone mineral content also improved (p=0.01 and p=0.0004, respectively).
- Sustained long-term benefits were not evident in patients treated up to 6 years.
- Vertebral fracture findings remained stable in 43 patients.
Conclusions:
- Oral bisphosphonate therapy with alendronate is associated with improved DXA bone health indices within the first three years of treatment in DMD patients.
- Alendronate may help mitigate the progression of osteoporosis in DMD patients receiving glucocorticoid therapy.
- Further research is needed to confirm long-term efficacy and optimal management strategies.
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