Effect of alendronate therapy in children with osteogenesis imperfecta.
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.
Vyskocil
Václav
V
Bone Disease Centre, Charles University Hospital, Pilsen, Czech Republic.
Pikner
Richard
R
Kutílek
Stepán
S
D000284:Administration, Oral; D000293:Adolescent; D019386:Alendronate / Q000008:administration & dosage*; D015519:Bone Density / Q000187:drug effects; D050071:Bone Density Conservation Agents / Q000008:administration & dosage*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D008297:Male; D010013:Osteogenesis Imperfecta / Q000188:drug therapy*; D016896:Treatment Outcome
D050071:Bone Density Conservation Agents; D019386:Alendronate