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Effects of Bisphosphonates on Osteoporosis Induced by Duchenne Muscular Dystrophy: A Prospective Study
Institution:2. Department of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing, China.;2. School of Medicine of Chung Shan Medical University;3. Institute of Medicine of Chung Shan Medical University;4. Department of Medical Research, Chung Shan Medical University Hospital, Taichung, Taiwan.;1. Service of Clinical Pharmacology, Lausanne University Hospital (CHUV), Bugnon 17, 1011 Lausanne, Switzerland;2. Center for Molecular Diseases, Division of Genetic Medicine, Lausanne University Hospital (CHUV), Pierre-Decker 2, 1011 Lausanne, Switzerland;3. Paediatric Neurology and Neurorehabilitation Unit, Lausanne University Hospital (CHUV), Pierre-Decker 5, 1011 Lausanne, Switzerland;4. Pediatric Neurology, University Children''s Hospital Basel, UKBB, and Inselspital Bern, Switzerland;5. Institute of Social and Preventive Medicine, University of Bern, Switzerland;1. Division of Human Development and Disability, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, 1600 Clifton Rd, Atlanta, GA 30333, USA;2. Department of Epidemiology, College of Public Health, The University of Iowa, 145 N Riverside Dr, S416 CPHB, Iowa City, IA 52242, USA;3. New York State Department of Health, Empire State Plaza, Corning Tower, Albany, NY 12237, USA;4. University at Buffalo, The State University of New York, 219 Bryant Street, Buffalo, NY 14222, USA;5. Children''s Hospital Colorado, 13123 East 16th Avenue, Aurora, CO 80045, USA;6. College of Medicine, University of Arizona, Tucson, AZ 85724, USA
Abstract:Objective: Duchenne muscular dystrophy (DMD) is a severe X-linked progressive neuromuscular disease that brings a significantly increased risk of osteoporosis and bone fractures. We prospectively evaluated the effects of oral and intravenous bisphosphonates on the bones of children with DMD.Methods: This study included a total of 52 children with DMD. They were divided into zoledronic acid (ZOL), alendronate (ALN), and control groups according to bone mineral density (BMD) and history of fragility fractures. For 2 years, all patients took calcium, vitamin D, and calcitriol. Meanwhile, 17 patients received infusions of ZOL, and 18 patients received ALN. BMD, serum levels of alkaline phosphatase (ALP) and the cross-linked C-telopeptide of type I collagen (β-CTX) were evaluated.Results: After 24 months of treatment, the percentage changes in lumbar spine BMD were 23.2 ± 9.7% and 23.6 ± 8.8% in the ZOL and ALN groups (all P<.01 vs. baseline). The increases did not differ between the ZOL and ALN groups, but were significantly larger than those of the control group (P<.01). Serum β-CTX and ALP levels, respectively, were decreased by 44.4 ± 18.0% and 31.9 ± 26.7% in the ZOL group and by 36.0 ± 20.3% and 25.8 ± 14.4% in the ALN group (all P<.01 vs. baseline).Conclusion: Zoledronic acid and alendronate had similar protective effects to increase bone mineral density and reduce bone resorption in children with DMD, which were superior to treatment of calcium, vitamin D, and calcitriol.Abbreviations: 25OHD = 25 hydroxyvitamin D; ALN = alendro-nate; ALP = alkaline phosphatase; ALT = alanine aminotransferase; BMD = bone mineral density; BP = bisphosphonate; Ca = calcium; β-CTX = cross-linked C-telopeptide of type I collagen; DMD = Duchenne muscular dystrophy; FN = femoral neck; GC = glucocorticoid; LS = lumbar spine; ZOL = zoledronic acid
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