Efecto de la terapia con alendronato en niños con osteogénesis imperfecta.(Ing.)

Bone Disease Centre, Charles University Hospital, Pilsen, Czech Republic

Abstract

Objective. – To evaluate the effect of orally administered alendronate in children with osteogenesis imperfecta.

Methods. – Thirty children (16 girls and 14 boys; mean age at baseline 10.7 ± 6.0 years; range 4–16 years) with osteogenesis imperfecta type I (n = 22), III (n = 2), or IV (n = 6) were treated with alendronate (5 mg/day in patients aged 4–10 years and 10 mg/day in children >10 years of age) for 3 years.

Results. – After 1 year of alendronate therapy we observed a significant increase in areal and volumetric bone mineral density Z-scores (from –2.03 ± 1.51 to –1.04 ± 1.20, and from –1.91 ± 1.38 to –1.33 ± 1.30, respectively, P < 0.001), together with a significant drop in fracture rate (from 3.77 ± 1.57 to 0.13 ± 0.57, P < 0.000001), relief of chronic pain (from 3.83 ± 1.44 days of pain/week to 0.73 ± 0.77, P < 0.000001) and improvement in ambulation/mobility (P < 0.00002). After additional 2 years of therapy there were no further significant changes in these parameters, however the improvement was still remarkable in comparison to the pretreatment values (P < 0.003, P < 0.004, P < 0.000001, P < 0.000001 and P < 0.00001, respectively). A significant drop in markers of bone turnover (urinary deoxypyridinoline and serum osteocalcin) occurred after 3 years of therapy (P < 0.003 and 0.004, respectively). No adverse reactions were observed throughout the treatment.

Conclusions. – Alendronate has positively influenced quality of life in paediatric patients with osteogenesis imperfecta. Bisphosphonate therapy should be used only in the context of a well-defined protocol.