BISPHOSPHONATE THERAPY FOR CHILDHOOD OSTEOPOROSIS

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Children with OI who do not qualify for protocol 837 ( Bisphosphonate Therapy for Osteogenesis Imperfecta) as a result of their age or medical history and children with osteoporotic disease other than OI ( idiopathic juvenile osteoporosis, steroid induced osteoporosis) will be treated with bisphosphonates to increase their bone mineral density. We plan to study these children in a non-randomized fashion. They will receive IV or oral bisphosphonate therapy. IV therapy will be done with pamidronate, 1.5-3 mg/kg (depending on age) divided over 3 days every 2-4 months. Oral treatment will be done with alendronate as < 1 mg/kg, from a minimum dose of 10 mg to a maximum of 40mg daily. Regardless of treatment arm, children will return to clinic every 4 months. Those receiving IV therapy will be admitted for 2 1/2 days; those on oral will have a day visit. Our primary efficacy outcome variable will be BMD, measured at 4-monthly intervals. Secondary outcome will include the following: fracture incidence; calcium biochemistry and biomarkers of bone formation and resorption; anthropometric measurements; characterization of dental anatomy and dental effects of therapy ( for children with OI or other conditions involving dentinogenesis imperfecta); systematic assessment of quality of life and pain measures; evaluation of gross motor function (for children with OI or other gross motor impairment); and safety of treatment. By studying these children in this manner, we will collect preliminary data that will enable us to design future studies of the treatment of childhood osteoporosis.