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Cyclic pamidronate infusion improves bone mineralisation and reduces fracture incidence in osteogenesis imperfecta
1Department of Paediatrics, National University of Singapore, National University Hospital, Singapore. paeleeys@nus.edu.sg
Insights
Pamidronate treatment significantly reduced fractures and improved bone mineral density in children with osteogenesis imperfecta. This bisphosphonate therapy is effective and safe for managing this bone disorder.
Area of Science:
- Pediatric Endocrinology
- Bone Metabolism
- Pharmacology
Background:
- Osteogenesis imperfecta (OI) is a genetic disorder characterized by fragile bones and increased fracture risk.
- Current treatments aim to improve bone strength and reduce fractures, but efficacy varies.
Purpose of the Study:
- To evaluate the efficacy and safety of pamidronate in improving bone mineralization and reducing fracture incidence in children with OI.
- To assess the impact of pamidronate on bone turnover markers.
Main Methods:
- A prospective open study involving six children with OI.
- Intravenous pamidronate administered at 1.5 mg/kg bi-monthly for 12-23 months.
- Fracture incidence, bone mineral density (BMD), urine N-telopeptide, and serum alkaline phosphatase (ALP) were measured.
Main Results:
- Fracture incidence decreased significantly from a median of 3 to 0 fractures per year (P<0.05).
- Lumbar spine areal BMD z-scores improved significantly, and volumetric BMD increased (P<0.05).
- Bone turnover markers (urine N-telopeptide and serum ALP) decreased, indicating reduced bone resorption and turnover (P<0.05).
Conclusions:
- One year of cyclical pamidronate treatment is effective and safe for improving bone mineralization in children with osteogenesis imperfecta.
- Pamidronate therapy leads to a significant reduction in fracture incidence and improves bone density in OI patients.
Unlabelled:
A prospective open study was performed to determine the efficacy and safety of pamidronate in improving bone mineralisation and reducing fracture incidence in osteogenesis imperfecta (OI). Intravenous pamidronate was administered at 1.5 mg/kg bi-monthly to six children with OI, over 12-23 months. The number of fractures decreased from median of 3 (range 1-12) to 0 fractures/year (range 0-4) (P<0.05). After 12 months of treatment, there was significant improvement in areal bone mineral density (BMD) z-scores of the lumbar spine from median of -2.40 (range -3.20 to -1.67) to -1.90 (range -2.38 to -0.91) (P<0.05) and in the volumetric BMD which increased from median of 0.095 to 0.146 g/cm3 (P<0.05). Urine N-telopeptide levels (bone resorption marker) decreased from a median of 461.5 bone collagen equivalent/creatinine (BCE/Cr) (range 129-721 BCE/Cr) to 223.5 BCE/Cr (range 107-312 BCE/Cr) (P<0.05) and serum alkaline phosphatase (ALP) (bone formation marker) from a median of 230.0 U/l (range 148-305 U/l) to 133.5 U/l (range 79-233 U/l) (P<0.05), reflecting reduced bone turnover. This may represent a net reduction in bone resorption and provides a biochemical explanation for the increase in bone mineralisation. Height standard deviation scores were not affected and there were no significant adverse effects.
Conclusion:
1 year cyclical pamidronate is effective and safe in improving bone mineralisation and reducing fracture incidence in osteogenesis imperfecta.