Treatment with Zoledronate in Children with Osteogenesis Imperfecta. Efficacy, safety and experiences of patients.

Keemink, Y.S. (2014) Treatment with Zoledronate in Children with Osteogenesis Imperfecta. Efficacy, safety and experiences of patients. thesis, Medicine.

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Abstract

Background; Osteogenesis imperfecta (OI) is a rare genetic disorder of collagen manifesting as increased susceptibility for fractures and bone deformities. Other organs containing collagen can also be affected. Up to now, bisphosphonates (BPs) have turned out as the best treatment option to increase bone density and reduce fracture incidence. The most recent added BP is intravenous zoledronate (ZOL). Objective: To investigate the efficacy and safety of treatment with ZOL as well as the experiences and perceptions of children with OI about ZOL. Methods: Retrospective data-analysis of all 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. To identify the patients’ experiences and perceptions of ZOL, all participants and / or their parents were interviewed using a semi-structured questionnaire. Results: Altogether, 25 children (1.2-18.8 years of age) were treated and included in this study. Follow-up data was 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 made a positive clinical difference and the reported quality of life (QOL) increased significantly (p<0.01). Conclusion: This retrospective analysis to ZOL treatment in Dutch children with OI showed a significant improvement in bone density and biochemical measurements, however no statistically significant changes in vertebral morphology. No severe side effects were documented and patients self-reported QOL increased. Further studies are needed to assess a possible superiority to other BPs.

Item Type:	Thesis (Thesis)
Supervisor name:	Nijholt, Dr. I.M.
Supervisor name:	Dijk, Drs. A.T.H. van and Brussel, Dr. M. v. and Wilhelmina Children’s Hospital and Universitary Medical Centre Utrecht
Faculty:	Medical Sciences
Date Deposited:	25 Jun 2020 11:03
Last Modified:	25 Jun 2020 11:03
URI:	https://umcg.studenttheses.ub.rug.nl/id/eprint/2357

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