Treatment with neridronate in children and adolescents with osteogenesis imperfecta: Data from open-label, not controlled, three-year Italian study

Introduction The present study assessed the long-term efficacy and safety of intravenous (i.v.) neridronate in children and adolescents affected by osteogenesis imperfecta (OI). Methods 55 young patients (mean age 12.6 ± 3.9 years) affected by OI were included in the study. Neridronate was administered by i.v. infusion at a dose of 2 mg/kg (maximum dose of 100 mg) at intervals of three-months for three years. Dual X-ray absorptiometry of the lumbar spine, hip and ultradistal and proximal radius were evaluated every 6 months. Blood calcium, phosphate, albumin, fasting urinary calcium/creatinine ratio were obtained at baseline and every 3 months. Serum bone turnover markers total and bone alkaline phosphatase were performed every 12 months in a proportion of patients. Results Mean lumbar spine and total hip bone mineral density (BMD) and bone mineral content significantly increased from baseline compared to all subsequent time points (p < 0.001). Mean ultradistal radius BMD significantly increased from month 18 (p = 0.026). Levels of bone turnover markers significantly decreased from baseline to all post-baseline observation time points. There was no statistically significant effect on fracture risk (p = 0.185), although a significant reduction was observed in the mean number of fractures occurring during treatment compared to pre-treatment values. The most frequent adverse events were arthralgia, fever, joint sprain. An acute phase reaction was reported in 26 (22.8%) patients. None of the reported serious adverse events was considered as treatment-related. Conclusion Long-term i.v. neridronate treatment has positive effects on BMD, bone turnover markers and fracture risk with a good safety profile.