ESPE2018 Poster Presentations Bone, Growth Plate & Mineral Metabolism P2 (24 abstracts)
aShahid Beheshti University of Medical Sciences, Tehran, Iran, Islamic Republic of Iran; bPediatric Endocrinology Department, Tehran, Iran, Islamic Republic of Iran; cGenomic Research Center, Tehran, Iran, Islamic Republic of Iran; dLoghman Hakim Clinical Research Development Center, Tehran, Iran, Islamic Republic of Iran; ePediatric Rheumatology Department, Tehran, Iran, Islamic Republic of Iran; fIran University of Medical Sciences, Tehran, Iran, Islamic Republic of Iran; gPediatric Orthopedics Section of Dey Hospital, Tehran, Iran, Islamic Republic of Iran
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.