Analysis of the Efficacy and Safety of Pamidronate Disodium in the Treatment of Osteogenic Imperfecta in Children

OBJECTIVE To retrospectively analyze the efficacy and safety of pamidronate disodium(APD) in the treatment of osteogenic imperfecta(OI) in children. METHODS Children who first used APD at the Children's Hospital Affiliated to Chongqing Medical University from January 1, 2014 to June 30, 2023 were selected as the research subjects. The growth and development status, improvement of bone metabolism and biochemical indicators, changes in bone density(BMD) and fractures, and occurrence of adverse drug reactions(ADRs) before and after treatment were compared. RESULTS A total of 14 pediatric patients were included, with a median age of 5.16 years. All children, regardless of the duration of treatment(1, 2 years, 3 years or more), showed significant improvements in height, body mass, and lumbar BMD compared to before treatment, while the average number of fractures per year decreased significantly(P<0.05). After 1 year of treatment, the alkaline phosphatase significantly increased(P=0.024). After 2 years of treatment, the total 25-hydroxyvitamin D₃(T-25OHD₃) significantly improved(P=0.014). After 3 years of treatment, the Z-value of height significantly improved(P=0.036). The most common ADR were fever, skeletal muscle pain, asymptomatic hypocalcemia and hypophosphatemia. CONCLUSION Pediatric patients with OI have good tolerance to APD treatment, with increased lumbar BMD and BMD Z values, reduced fracture rates, and improved growth and development.