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Pamidronate treatment of children with moderate-to-severe osteogenesis imperfecta: a note of caution
M Alharbi1, G Pinto, G Finidori
1Department of Pediatric Endocrinology, Necker Enfants Malades Teaching Hospital, Paris, France.
Background/Aims:
Bisphosphonates have been reported to decrease fractures related to osteogenesis imperfecta (OI). We assessed the efficacy and long-term safety of pamidronate therapy in patients with moderate-to-severe OI.
Methods:
We conducted an open-label uncontrolled study in 14 boys and 13 girls whose mean age was 6.8 years at baseline. Intravenous pamidronate, 1 mg/kg/day, was given for 3 consecutive days every 4 months for 2-6 years, with physical therapy and orthopedic surgery as appropriate. Mobility score, fracture rate, height, bone mineral density (BMD) and bone healing were evaluated throughout follow-up.
Results:
In 24 (89%) patients, the fracture rate decreased to
Conclusion:
Pamidronate with physiotherapy and orthopedic management improved outcomes without delaying fracture healing in 19 (70%) of 27 patients. Delayed fracture healing occurred in 8/27 patients. Pamidronate should be reserved for severe OI with multiple fractures and/or flattened vertebras.
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