Zoledronate for Osteogenesis imperfecta: evaluation of safety profile in children.

BACKGROUND

The advent of bisphosphonates (BPs) has revolutionised the outcome of Osteogenesis imperfecta (OI) in the last few years. There has always been a safety concern regarding zoledronate's use due to a paucity of studies. The current study is a retrospective evaluation of children with OI on the short- and long-term side effects of zoledronate and the frequency of fractures per year after the drug was introduced.

METHODS

A total of 26 children diagnosed with OI, with a median age of 84 (45-121) months were enrolled in the study. They received cyclical zoledronate for a median duration of 36 (11-61) months at quarterly intervals between January 2008 and December 2014. Safety evaluation involved assessment of its short- and long-term effects in addition to the frequency of fractures after its usage.

RESULTS

One (3%) neonate had symptomatic hypocalcemia 15 days after the infusion. Three children (11%) had acute phase reactions. None had long-term side effects, including osteonecrosis of the jaw, in our 7-year experience. OI of types III and IV (total of 22) had significant reductions in the number of fractures (p<0.05).

CONCLUSIONS

Further long-duration studies are necessary to evaluate the longterm safety of zoledronate.