EFFECTS OF LONG-TERM ALENDRONATE TREATMENT ON A LARGE SAMPLE OF PEDIATRIC PATIENTS WITH OSTEOGENESIS IMPERFECTA

Objective: Osteogenesis imperfecta (01) is a group of inherited diseases characterized by reduced hone mass, recurrent hone fractures, and progressive hone deformities. Here, we evaluate the efficacy and safety of long-term treatment with alendronate in a large sample of Chinese children and adolescents with OI. Methods: In this prospective study, a total of 91 children and adolescents with OI were included. The patients received 3 years' treatment with 70 mg alendronate weekly and 500 mg calcium daily. During the treatment, fracture incidence, hone mineral density (BMD), and serum levels of the hone turnover biomarkers (alkaline phosphatase [AI 2] and cross linked C-telopeptide of type I collagen [beta-CTX]) were evaluated. Linear growth speed and parameters of safety were also measured. Results: After 3 years of treatment, the mean annual fracture incidence decreased from 1.2 +/- 0.8 to 0.2 +/- 0.3 (P<.01.). BMD at the lumbar spine and femoral neck significantly increased by 74.6% and 39.5 with their BMD Z-score increasing from -3.0 to 0.1 and from -4.2 to -1.3, respectively (both P<.01 vs. baseline). In addition, serum ALP and beta-CTX levels decreased by 35.6% and 44.3%, respectively (both P<.05 vs. baseline). Height significantly increased, but without an obvious increase in its Z-score. Patient tolerance of alendronate was good. Conclusion: Three years' treatment with alendronate was demonstrated for the first time to significantly reduce fracture incidence, increase lumbar spine and femoral neck HMI), and decrease hone turnover biomarkers in Chinese children and adolescents with OI.