CFDB - Cystic Fibrosis DataBase

primary studies published RCT

Psychosocial and educational outcomes associated with home- and clinic-based pretest education and cystic fibrosis carrier testing among a population of at-risk relatives.

Study design (if review, criteria of inclusion for studies)

randomized, double-blind, placebo controlled study.

Participants

All patients were taking replacement enzymes to compensate for pancreatic insufficiency. n = 17

Interventions

megestrol acetate or placebo.

Outcome measures

weight-for-age z scores, at and fat-free mass, FEV1, adrenal suppression

Main results

The treatment group had a significant increase in weight-for-age z scores compared with placebo and reached 100% of their ideal body weight within 3 months of initiating therapy. Weight gain included both fat and fat-free mass. Improved pulmonary function (forced vital capacity and forced expiratory volume in 1 second) was noted in the treatment group compared with placebo (P <.04). Reversible adrenal suppression was observed in the majority of patients who received megestrol acetate.

Authors' conclusions

Short-term use of megestrol acetate results in significant weight gain and improved pulmonary function in malnourished subjects with CF. Our study provides a controlled basis for this intervention, identifies important side effects, and provides the foundation for multiyear, longitudinal trials in a larger number of patients with CF.

Keywords: Adolescent; Adult; Appetite Stimulants; Child; Hormones; Hydrocortisone; Hypoglycemic Agents; Insulin; megestrol; non pharmacological intervention - diet; pharmacological_intervention; Steroids; Supplementation; Malnutrition; Nutrition Disorders; Anti-Inflammatory Agents;