ABSTRACT
Objective: To ascertain clinical outcome following cyclical intravenous pamidronate therapy in a group of infants and young children with type III osteogenesis imperfecta (OI). Design, setting and method: A prospective observational study was carried out on infants and young children referred to Ward 9 Lady Ridgeway Hospital (LRH) for assessment and treatment of OI from January 2005 to February 2007. All were treated with cyclical high dose intravenous pamidronate. Data collected included clinical history, treatment history, dose and frequency of treatment, adverse effects of the drug and outcome of treatment. Results: Thirteen patients with clinical presentations compatible with type III OI were referred to Ward 9 LRH during the study period. Ten had fractures during fetal life and three presented with fractures during infancy. Age at commencement of the drug varied from 3 weeks to 42 months (median 10 months). The patients were followed up for periods ranging from 3 to 24 months. Four out of the 13 patients developed new fractures during follow up. Conclusion: Intravenous high dose cyclical pamidronate is a useful form of therapy to prevent recurrent fractures in infants and young children with type III OI.