Objective. To evaluate the effectiveness of
oxandrolone in improving the nutritional status and linear growth of pediatric patients with
cystic fibrosis (CF). Methods. Medical records of patients with CF treated with
oxandrolone were reviewed for height z score, height velocity (HV), BMI z score, weight velocity (WV), Tanner stage, pulmonary function, liver
enzyme levels, and any reported adverse events. Data were compared before (pre-Ox) and after (Ox)
oxandrolone using a paired t-test. Results. 5 subjects (ages 8.5-14.5 years) were treated with
oxandrolone 2.5 mg daily for 8-38 months. After 8-12 months of treatment, there was a statistically significant improvement in HV (pre-Ox = 5.3 +/- 1.4 cm/yr, Ox = 8.3 +/- 1.2 cm/yr, P < .01) and BMI z score (pre-Ox = -0.61 +/- 1.04, Ox = -0.30 +/- 0.86, P = .02). Both height z score (pre-Ox = -1.64 +/- 0.63, Ox = -1.30 +/- 0.49, P = .057) and WV (pre-Ox = 4.2 +/- 3.7 kg/yr, Ox = 6.8 +/- 1.0 kg/yr, P = .072) showed beneficial trends that did not reach statistical significance. No adverse events were reported. Conclusions. In this brief clinical report,
oxandrolone improved the HV and BMI z score in patients with CF. Larger studies are needed to determine if
oxandrolone is an effective, safe, and affordable option to stimulate appetite, improve
weight gain, and promote linear growth in patients with CF.