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*OMIM
*Compound via MeSH
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*Genetics Home Reference
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*Osteogenesis Imperfecta
The Journal of Clinical Endocrinology & Metabolism Vol. 85, No. 5 1846-1850
Copyright © 2000 by The Endocrine Society

Original Studies

Pamidronate Treatment of Severe Osteogenesis Imperfecta in Children under 3 Years of Age1

Horacio Plotkin, Frank Rauch, Nicholas J. Bishop2, Kathleen Montpetit, Joanne Ruck-Gibis, Rose Travers and Francis H. Glorieux

Genetics Unit, Shriners Hospital for Children, and Departments of Surgery and Pediatrics, McGill University, Montréal, Québec, Canada H3G 1A6

Address all correspondence and requests for reprints to: F. H. Glorieux, M.D., Ph.D., Genetics Unit, Shriners Hospital for Children, 1529 Cedar Avenue, Montréal, Québec, Canada H3G 1A6. E-mail: glorieux{at}shriners.mcgill.ca

Severe osteogenesis imperfecta (OI) is a hereditary disorder characterized by increased bone fragility and progressive bone deformity. Cyclical pamidronate infusions improve clinical outcome in children older than 3 yr of age with severe OI. Because earlier treatment may have potential to prevent deformities and improve functional prognosis in young children, we studied nine severely affected OI patients under 2 yr of age (2.3–20.7 months at entry) for a period of 12 months. Pamidronate was administered iv in cycles of 3 consecutive days. Patients received four to eight cycles during the treatment period, with cumulative doses averaging 12.4 mg/kg. Clinical changes were evaluated regularly during treatment, and radiological changes were assessed after 6–12 months of treatment. The control group consisted of six age-matched, severely affected OI patients, who had not received pamidronate treatment. During treatment bone mineral density (BMD) increased between 86–227%. The deviation from normal, as indicated by the z-score, diminished from -6.5 ± 2.1 to -3.0 ± 2.1 (P < 0.001). In the control group the BMD z-score worsened significantly. Vertebral coronal area increased in all treated patients (11.4 ± 3.4 to 14.9 ± 1.8 cm2; P < 0.001), but decreased in the untreated group (P < 0.05). In the treated patients, fracture rate was lower than in control patients (2.6 ± 2.5 vs. 6.3 ± 1.6 fractures/year; P < 0.01). No adverse side-effects were noted, apart from the well known acute phase reaction during the first infusion cycle. Pamidronate treatment in severely affected OI patients under 3 yr of age is safe, increases BMD, and decreases fracture rate.




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