Bone Abstracts

Background: In pediatric osteogenesis imperfecta (OI), bisphosphonates (BPs) are considered the best treatment option to increase bone density and reduce fracture rate. Pamidronate (PAM) is regarded as standard care for moderate to severe OI. The most recent added BP is intravenous zoledronate (ZOL). ZOL has practical advances over PAM, however research on its efficacy and safety is scarce.

Objectives: To investigate the efficacy and safety of treatment with ZOL inDutch children.

Methods: Retrospective data-analysis of Dutch children with OI who received ZOL between 2009–2014. Bone densitometry scans (DXA), lumbar spine radiographs, biochemical measurements, fracture rate and side effects were documented to assess efficacy and safety. Results at baseline were compared to those after one year and thereafter. In addition, patients’ experiences with ZOL were identified using a semi-structured interview.

Results: 25 children (1.2–18.8 years of age) were treated and included. Follow-up data were available for 1 to 3.5 years of treatment. Zoledronate treatment was associated with a significant increase of bone density on DXA scans (P<0.01) and decrease of fracture rate (P<0.01). Vertebral height increased significantly in all patients (P=0.02) but there were no significant changes in vertebral index and discus coefficient. Furthermore, there was a significant decrease in total serum alkaline phosphatase (P=0.03). Infusion related adverse events, mainly flu like symptoms, were reported by 36% of the participants. There were no clinical symptoms of hypocalcaemia following infusion. The majority of patients felt that ZOL had made a positive clinical difference and the reported quality of life (QOL) increased significantly (P<0.01).

Conclusion: This retrospective analysis of Dutch children with OI confirms the efficacy and safety of treatment with ZOL. No severe side effects were documented and patients self-reported QOL increased. Should Zoledronate be applied as the first line treatment in pediatric OI?

Disclosure: The authors declared no competing interests.