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Oxandrolone therapy in skeletal dysplasia
1Department of Pediatrics, Dokuz Eylül University Faculty of Medicine, Izmir.
Abstract:
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.
Insights
Oxandrolone treatment improved growth rates in three children with skeletal dysplasia and short stature. Further studies are needed to confirm long-term efficacy and optimal dosing for adult height.
Area of Science:
- Pediatric Endocrinology
- Genetics and Developmental Biology
Background:
- Short stature associated with skeletal dysplasia presents a significant clinical challenge.
- Growth hormone response is typically normal in these patients, suggesting alternative therapeutic targets.
Observation:
- Three pediatric patients with distinct skeletal dysplasias (spondyloepiphyseal dysplasia congenita, hypochondroplasia, multiple epiphyseal dysplasia) presented with short stature.
- All patients exhibited normal growth hormone (GH) response to insulin-induced hypoglycemia testing (ITT).
Findings:
- Daily low-dose oxandrolone (1.25 mg/day) administration for one year significantly increased growth velocity in all patients.
- Growth rates increased from 1.5-2.5 cm/yr to 5.3-8.0 cm/yr.
- Height standard deviation scores for both chronological and bone age improved, with patients remaining prepubertal.
Implications:
- Low-dose oxandrolone therapy shows promise as a treatment for short stature in children with skeletal dysplasia.
- Longitudinal studies are essential to evaluate the impact on adult height prognosis and establish optimal dosing strategies.