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Intavenous pamidronate treatment to infants with severe osteogenesis imperfecta
  1. Eva Åström (eva.astrom{at}karolinska.se)
  1. Karolinska Institute, department of Women and Child Health, Sweden
    1. Håkan Jorulf
    1. Karolinska Institute, department of Women and Child Health, Sweden
      1. Stefan Söderhäll
      1. Karolinska Institute, department of Women and Child Health, Sweden

        Abstract

        Objective: Children with severe form of Osteogenesis Imperfecta (OI) have in several studies been treated with intravenous pamidronate, but there are only few reports of the effect of early treatment. The aim of this study was to evaluate the effect of treatment started in infancy.

        Methods: In a prospective observational study, with a historic control group, disodium pamidronate (APD) was given as monthly intravenous infusions to eleven children with OI aged 3-13 (median 3.6) months, who had severe OI with congenital bowing of the femora and vertebral compression fractures.

        Results: During treatment for between 3 and 6 (median 4.5) years DXA measurements of the lumbar spine showed a gradual increase in bone density. Bone metabolism parameters in serum (ALP, osteocalcin, P1CP, 1CTP) and in urine (deoxypyridinoline) all indicated a decrease in bone turnover. An improvement of mobility was seen and at the latest recording, at the age of 3.3- 6.5 (median 4.8) years, they all had walking ability. Vertebral remodelling was seen, with increased vertebral height, and no one developed scoliosis, kyphosis or basilar impression. All children required femoral intramedullar rods for fractures and five needed tibial rodding for extreme curvatures which prevented functional standing and walking. No adverse effects were seen on growth, fracture healing or blood chemistry.

        Conclusions: APD is an efficient symptomatic treatment for infants with severe OI, but additional orthopaedic surgery is often needed. Early treatment may prevent scoliosis and basilar impression. Long term follow up is important.

        • bisphosphonate
        • children
        • infant
        • osteogenesis imperfecta
        • treatment

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