The use of bisphosphonates in children with osteogenesis imperfecta

Children with osteogenesis imperfecta (OI) suffer recurrent fractures resulting in pain, deformity and disability. There is no accepted medical therapy for the condition other than symptomatic pain relief, and surgical correction of the deformities. Recent experience with the bisphosphonate group of drugs suggests, however, that anti-resorptive therapy may reduce fracture frequency, increase bone density, promote remodeling of previously crush-fractured vertebrae, reduce chronic pain, and improve mobility in both children and infants. The prospects for preventing disability and deformity, and perhaps improving growth in affected children are good. Current studies are focusing on the evaluation of the efficacy of oral drug therapy, and protocols are under development to evaluate new, more efficient molecules. Until gene therapy becomes a reality, the use of bisphosphonates appears to be the most efficient way of altering the natural course of severe OI, and improving the quality of life of patients.