The effect of intravenous pamidronate treatment for type i osteogenesis imperfecta patients have on their bone mineral density, fracture rate, and mobility

Abstract

Objective: Osteogenesis imperfecta (OI) is a genetic disorder caused by the mutation in encoding collagen type I, and characterised by fragile bone and reduced bone mineral density. The purpose of this study was to analyse the effects siklik intravenous pamidronate treatment has on the bone mineral density and the fracture rateand mobility. Patients and Methods: The sample group of this study comprises of 7 patients (6 male (85.7%), 1 women (14.2%) with type I OI, who received intravenous pamidronate, between August 2004 and October 2010. The intravenous dose of pamidronate disodium (Aredia, Novartis) was 0.75 mg/kg of the body weight, administered as a single dose every 6 months, over 8 hours in a saline solution. Bone mineral density was measured before and after the treatment using a dual energy X-ray absorptiometer at vertebral bodies from L1 to L4. Bone mineral density (g/cm(2)), T scores and Z scores, number of fractures, and modified Bleck scores were recorded. Results: The median age of patients at the time of treatment was 15 years (min: max=13: 18), the median BMI was 17.86 (min: max=14.29: 20.68) kg/m(2), the median follow-up period was 36 (min: max=12: 72), and the median number of doses received was 5 (min: max=1: 20). The median bone mineral content was increased from 0.368 (min: max=0.23-0.59) to 0.628 (min: max=0.53: 0.88) (p=0.02), the median T score was increased from -6.6 (min: max=-7.50: -1.90) to -4.2 (min-max=-5.10:-1.90) (p=0.043), the median Z score was increased from -4.91 (min: max=-7.7: -3.2) to -3.9 (p=0.144), and the median modified Bleck score was increased from 6 (min: max=2: 9) to 9 (min: max=: -7.7: -3.2) (p=0.043). The median numbers of fracture decreased from 0.40 (min: max=0.15: 0.76) to 0 (min: max: 0: 1) per year (p= 0.540). Conclusion: Intravenous pamidronate in patients with OI increased bone mineral density and patient's mobility, and improved the fracture rate. Pamidronate is a safe and effective treatment method in patients with OI.