Beneficial effect of long term intravenous bisphosphonate treatment of osteogenesis imperfecta

E Aström, S Söderhäll, E Aström, S Söderhäll

Abstract

Aim: To find an effective symptomatic treatment for osteogenesis imperfecta (OI).

Methods: In a prospective observational study disodium pamidronate (APD) was given as monthly intravenous infusions to 28 children and adolescents (aged 0.6-18 years) with severe OI or a milder form of the disease, but with spinal compression fractures.

Results: During treatment for 2-9 years, dual energy x ray absorptiometry measurements of the total body and of the lumbar spine showed a gradual increase in bone density. All bone metabolism variables in serum (alkaline phosphatase, osteocalcin, procollagen 1 C-terminal peptide, collagen 1 teleopeptide) and urine (deoxypyridinoline) indicated that there was a decrease in bone turnover. All patients experienced beneficial effects and the younger patients reported a major improvement in wellbeing, pain, and mobility without significant side effects. Vertebral remodelling was also seen.

Conclusions: APD seems to be an efficient symptomatic treatment for children and adolescents with OI.

Figures

Figure 1
Bone density (DXA, L1–L4) in g/cm2 during APD treatment. Data for individual patients are plotted against lunar mean, 5th, and 95th confidence interval curves for healthy children

Figure 2
Bone density (DXA, total body) in g/cm2 during APD treatment. Data for individual patients are plotted against lunar mean, 5th, and 95th confidence interval curves for healthy children

Figure 3
Radiographs (lateral view) of the lumbar spine of an 1.1 year old girl with osteogenesis imperfecta type IV (patient 2). (A) Before start of treatment. (B) After two years treatment. A considerable increase of mineralisation and vertebral height can be seen, with almost complete regeneration of the vertebral height around L1

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Source: PubMed

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