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Alendronate treatment in osteogenesis imperfecta.
Ercan Madenci1, Kutluhan Yilmaz, Mustafa Yilmaz
1Departments of Physical Therapy and Rehabilitation, Gaziantep University, Medical Faculty, 27100 Gaziantep, Turkey. drmadenci@yahoo.com
Summary
Alendronate therapy significantly increased bone density in children with Osteogenesis Imperfecta (OI). This potent bisphosphonate showed promise as a safe oral treatment option for improving bone health in OI patients.
Area of Science:
- Pediatric Endocrinology
- Bone Metabolism
- Pharmacology
Background:
- Osteogenesis Imperfecta (OI) is a genetic disorder causing brittle bones due to collagen defects.
- Current treatments like pamidronate are effective but alendronate offers greater potency.
- Evaluating novel bisphosphonate therapies is crucial for managing OI.
Purpose of the Study:
- To assess the efficacy and safety of oral alendronate in pediatric patients with Osteogenesis Imperfecta.
- To compare alendronate's effects on bone mineral density and clinical outcomes against existing therapies.
Main Methods:
- A 2-year prospective study involving three children (ages 3-7) with OI types III and IV.
- Alendronate administered orally at 0.3-0.56 mg/kg/day.
- Evaluated outcomes included fracture rates, ambulation, growth, and bone mineral density (DXA).
Main Results:
- Significant improvements in bone mineral density (BMD) observed in lumbar spine (47.8%-106.6%) and forearm (24%-51.4%).
- Lumbar spine BMD z-scores improved from -5.26 to -3.1.
- Fracture rates showed no significant change, but ambulation improved in one severely affected patient. No adverse effects were noted.
Conclusions:
- Alendronate is a safe and well-tolerated oral bisphosphonate for children with OI.
- It effectively increases bone density, supporting further research into potent bisphosphonates for OI.
- This study pioneers the use of forearm BMD measurement in OI assessment.