Background: Severe osteogenesis imperfecta (OI) is a disorder characterized by osteoporosis, frequent fractures, progressive deformity and short stature. We determine the effect of oxandrolone on predicted adult height (PAH), fracture incidence and bone mineral density in teenage boys with OI.
Methods: In a prospective, double-blind, randomized, placebo-controlled clinical trial, 31 boys (12.116.6 years old) who were genetically proved to have OI with an annual fracture rate more than 3 in spite of receiving cyclic pamidronate were treated with oxandrolone (2.5 mg/day), or placebo, at an outpatient pediatric endocrine clinic in Tehran for 2 years.
Results: Oxandrolone differed from placebo in significantly increasing PAH (P < 0.01), and the height standard deviation score (P < 0.01) and bone mineral density (P < 0.005) and reducing fracture incidence compared to placebo.
Conclusion: This first randomized controlled clinical trial in male teenagers with severe OI shows that oxandrolone increases PAH, height standard deviation score and bone mineral density and reduces fracture incidence.
27 - 29 Sep 2018
European Society for Paediatric Endocrinology