Oxandrolone therapy in skeletal dysplasia.

Three patients with short stature and different forms of skeletal dysplasia were treated with oxandrolone, 1.25 mg/day. All patients had a normal growth hormone response (10 ng/ml) to the insulin-induced hypoglycemia test (ITT). After one year of follow-up, it was noted that the pretreatment growth rate which was 2.5 cm/yr in the patient with spondyloepiphyseal dysplasia congenita (chronological age, 13 4/12 yr) had increased to 6.7 cm/yr after one year of treatment, while the pretreatment growth rate of the patient with hypochrondroplasia (chronological age, 12 7/12 yr) which was recorded at 2 cm/yr had risen to 5.3 cm/yr. The patient with multiple epiphyseal dysplasia (chronological age, 9 5/12 yr) had a pretreatment growth rate of 1.5 cm/yr which had risen to 8 cm/yr after the same period of treatment. An increase was noted in the height standard deviation score for chronological age and in the height standard deviation score for bone age in all patients. After one year of therapy, all patients were observed to still be in the prepubertal stage. Oxandrolone therapy seems to be useful in the treatment of short stature seen in skeletal dysplasia. However, a more lengthy study is needed in order to assess the efficacy of treatment with regard to adult height prognosis and to determine the optimal dosing required.