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Short-Term Megestrol Helps Cystic Fibrosis Children Gain Weight

Malnourished paediatric cystic fibrosis patients show significant weight gain and improved pulmonary function after short-term therapy with megestrol acetate.

Patients prescribed megestrol reached 100 percent of their ideal body weight within three months of therapy initiation. Their weight gain included both fat and fat-free mass, researchers from the University of Alabama at Birmingham and the University of Florida at Gainesville report.

Malnutrition is a negative prognostic indicator in cystic fibrosis patients and can also accentuate pulmonary decline.

Investigators tested whether megestrol would benefit growth in 17 patients with cystic fibrosis and pancreatic insufficiency. Participants were all taking replacement enzymes to compensate for the pancreatic insufficiency. The children were randomly assigned to megestrol or placebo in a double-blind study.

Those receiving megestrol had a significant increase in weight-for-age z scores. They also had significantly improved pulmonary function, measured by forced vital capacity and forced expiratory volume in one second.

The investigators point out that they observed reversible adrenal suppression in a majority of patients receiving megestrol. They suggest the study provides a foundation for multi-year, longitudinal trials in a larger number of patients.
Journal of Pediatrics, 2002; 140: 439-444. "Effects of megestrol acetate on weight gain, body composition, and pulmonary function in patients with cystic fibrosis"

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