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Oxandrolone Improves Height Velocity and BMI in Patients with Cystic Fibrosis

Todd Varness1, Erin E Seffrood, Ellen L Connor

  • 1Department of Pediatrics, University of Wisconsin School of Medicine and Public Health, H4/4 Clinical Science Center, 600 Highland Avenue, Madison, WI 53792-4108, USA.

International Journal of Pediatric Endocrinology
|February 11, 2010
PubMed

Insights

Oxandrolone significantly improved height velocity and BMI z score in pediatric cystic fibrosis (CF) patients. This anabolic steroid showed potential for enhancing growth and nutritional status in CF, with no reported adverse events.

Area of Science:

  • Pediatric Endocrinology
  • Pulmonology
  • Nutritional Science

Background:

  • Cystic Fibrosis (CF) is a genetic disorder affecting multiple organs, often leading to malnutrition and growth failure.
  • Optimizing nutritional status and linear growth is crucial for improving outcomes in pediatric CF patients.
  • Anabolic steroids are sometimes considered to address growth deficits in chronic diseases.

Observation:

  • A retrospective review of 5 pediatric CF patients (ages 8.5-14.5 years) treated with oxandrolone (2.5 mg daily for 8-38 months) was conducted.
  • Key metrics including height z score, height velocity (HV), BMI z score, weight velocity (WV), and pulmonary function were analyzed.
  • Data were compared pre- and post-oxandrolone treatment.

Findings:

  • Oxandrolone treatment led to a statistically significant improvement in height velocity (HV) (P < .01) and BMI z score (P = .02).
  • Beneficial trends were observed for height z score and weight velocity (WV), though they did not reach statistical significance.
  • No adverse events were reported during the study period.

Implications:

  • Oxandrolone may be an effective therapeutic option for improving linear growth and nutritional status in pediatric CF patients.
  • Further large-scale studies are warranted to confirm the efficacy, safety, and cost-effectiveness of oxandrolone in CF.
  • This finding could inform treatment strategies aimed at enhancing growth and overall health in children with cystic fibrosis.

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