Effects of megestrol acetate on weight gain, body composition, and pulmonary function in patients with cystic fibrosis.
OBJECTIVE
Malnutrition is a negative prognostic indicator in patients with cystic fibrosis (CF) and may accentuate pulmonary decline. We tested whether megestrol acetate would have beneficial effects on growth in patients with CF and pancreatic insufficiency.


METHODS
We performed a randomized, double-blind, placebo controlled study. All patients were taking replacement enzymes to compensate for pancreatic insufficiency. Patients (n = 17) were randomly assigned to receive either megestrol acetate or placebo.


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.


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.
Eubanks
Valerie
V
Department of Pediatrics, Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, USA.
Koppersmith
Nicki
N
Wooldridge
Nancy
N
Clancy
J P
JP
Lyrene
Raymond
R
Arani
Ramin B
RB
Lee
Jeanette
J
Moldawer
Lyle
L
Atchison
Joy
J
Sorscher
Eric J
EJ
Makris
Christopher M
CM
D000293:Adolescent; D000328:Adult; D000367:Age Factors; D001823:Body Composition / Q000187:drug effects*; D015519:Bone Density / Q000187:drug effects; D002648:Child; D002669:Child Welfare; D003550:Cystic Fibrosis / Q000188:drug therapy*; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D006854:Hydrocortisone / Q000097:blood; D007328:Insulin / Q000097:blood; D008168:Lung / Q000187:drug effects* / Q000502:physiology*; D008297:Male; D019290:Megestrol Acetate / Q000627:therapeutic use*; D013997:Time Factors; D016896:Treatment Outcome; D015430:Weight Gain / Q000187:drug effects*
D007328:Insulin; D019290:Megestrol Acetate; D006854:Hydrocortisone