OXANDROLONE THERAPY IN CONSTITUTIONALLY DELAYED GROWTH AND PUBERTY

Background. Male adolescents with constitutional delay of growth and p uberty may have significant psychosocial difficulties related to their sexual immaturity and short stature. The purpose of this study was to test the hypothesis that 1 year of oxandrolone therapy would increase growth velocity and thereby improve psychosocial functioning in boys with constitutional delay of growth and pubertal development. Methods. Forty boys (ages 11 to 14.7 years) with delayed pubertal development and short stature were recruited from the pediatric endocrine clinics of 14 medical centers. The boys were randomized using a block design s tratified for age to receive either oxandrolone (0.1 mg/kg daily for 1 year) or an identical-appearing placebo tablet, using a double-masked design. Results. Growth velocity in the oxandrolone-treated boys was significantly greater than in the control boys (9.5 vs 6.8 cm/y). Like wise, the mean height SD score increased 0.41 in the oxandrolone group , whereas it decreased 0.03 in the control group. Those in the oxandro lone group gained 2.4 kg more than those in the placebo group. Mean pr edicted adult heights did not change in either group. The mean rates o f pubertal progression were equivalent in both groups. Self-image (Pie rs-Harris Self Concept Scale) and social competence (Child Behavior Pr ofile) were normal at baseline in both groups and did not change signi ficantly over the course of the study in either group. No complication s of oxandrolone therapy were identified. Conclusions. This randomized , placebo-controlled trial demonstrates that low-dose oxandrolone can increase both height and weight velocity in boys with delayed puberty safely. Under the conditions of this study, however, the increased gro wth velocity in the oxandrolone-treated boys was not associated with a greater improvement in psychosocial status compared with the control boys.