A 3‐year, randomized, double‐blind, placebo‐controlled trial evaluated the effect of oral alendronate on the BMD of 64 adult patients with osteogenesis imperfecta. The mean increases in the lumbar spine BMD were 10.1 ± 9.8% (p < 0.001) and 0.7 ± 5.7% in the alendronate and placebo groups, respectively. Oral alendronate increases BMD in adult patients with osteogenesis imperfecta.

Introduction: This study evaluated the effect of oral alendronate on the BMD of adult patients with osteogenesis imperfecta.

Materials and Methods: We carried out a 3‐year, randomized, double‐blind, placebo‐controlled trial of oral alendronate in 64 adult patients with osteogenesis imperfecta. The primary endpoint was the difference between the groups in the mean percent change in lumbar spine BMD at 3 years. Secondary outcomes included changes in BMD of total hip, vertebral and peripheral fracture incidence, pain, hearing loss, and bone turnover biochemical markers. Patients were treated daily with either placebo or 10 mg alendronate. All received 1 g of calcium and 800 IU of vitamin D daily.

Results: The mean ± SD increases in the lumbar spine BMD were 10.1 ± 9.8% (p < 0.001) and 0.7 ± 5.7% in the alendronate and placebo groups, respectively. Hip BMD increased in the alendronate group by 3.3 ± 0.5% (p = 0.001) and decreased in the placebo group by 0.3 ± 0.6%. The sample size was not sufficient to determine an effect of alendronate on fracture rate. A significant increase of the pain score was noted in the alendronate group (p = 0.04) in the intent‐to‐treat analysis but not in the per protocol analysis. There was no change in hearing in either group. Bone resorption and formation biochemical markers were significantly decreased in the alendronate group (p < 0.001). There were no differences in severe adverse effects between the groups, but there was an increase in nonsevere upper gastrointestinal effects in the alendronate group (p = 0.003).

Conclusions: Oral alendronate increases BMD and increase nonsevere gastrointestinal adverse effects but does not modify the hearing loss in adult patients with osteogenesis imperfecta. More studies are needed to evaluate an effect on the fracture rate.