[Clinical study of oral alendronate in the treatment of pediatric osteogenesis imperfecta].

OBJECTIVE

To explore the clinical efficacy and safety of oral alendronate in children with osteogenesis imperfecta (OI).

METHODS

Eleven OI children were recruited from August 2008 to April 2011 at Shanghai Institute of Pediatric Research to receive alendronate for a duration of (1.7 ± 0.3) years. The growth, fracture incidence, physical activity, the quality of daily life and safety parameters were evaluated.

RESULTS

All patients obtained marked improvement. The rates of bone fractures decreased more remarkably than that at pre-treatment (0 - 1.2 fractures per year vs 0.5 - 5.0 fractures per year, medium 0 vs 1.40 fractures per year) (P = 0.003). Their levels of physical activities improved significantly (median level from 4 to 3, P = 0.004). There was significant post-treatment improvement in the self-care activity scores (median score from 43 to 73, P = 0.003). The bone density of lumbar vertebrae, long bones and metaphysis improved at post-treatment. The radiographic examinations revealed the thickness of bone cortex. The change in height did not show any significant difference. No change was found in the serum levels of calcium, phosphorus, parathyroid hormone, alkaline phosphate or other biochemical markers. No adverse reaction occurred throughout treatment.

CONCLUSION

Oral alendronate treatment reduces the incidence of bone fracture and improves physical activity and life quality in OI children, and as a well-tolerated regimen, it is both safe and effective in clinical practice.