title: A randomized, double-blind, placebo-controlled clinical trial of megestrol acetate as an appetite stimulant in children with weight loss due to cancer and/or cancer therapy.
abstract: BACKGROUND
  Megestrol acetate (MA) is an appetite stimulant with efficacy in promoting weight gain in adults with cancer-associated anorexia-cachexia. Studies documenting MA efficacy in children, however, are limited. We present the first randomized, double-blind, placebo-controlled clinical trial of MA versus placebo in children with cancer and weight loss.
  
  
  METHODS
  Subjects <18 years of age with weight loss (minimum 5% from highest previous weight; or %ideal body weight <90%) due to cancer and/or cancer therapy were randomized to either MA (7.5 mg/kg/day) or placebo for a planned study duration of 90 days. Primary outcome was the difference between groups in mean percent weight change from beginning to end of the study period. Secondary outcomes included effects on anthropometrics, body composition, need for tube feeding or parenteral nutrition, and toxicities.
  
  
  RESULTS
  Twenty-six patients were randomly assigned (13 MA, 13 placebo). The MA group experienced a mean weight gain of +19.7% compared to a mean weight loss of -1.2% in the placebo group, for a difference of +20.9% (95%CI: +11.3% to +30.5%, P = 0.003) in favor of MA over placebo. MA subjects experienced significant increases in weight for age z-scores, body mass index z-scores, and mid upper arm circumference compared to placebo. DXA scanning suggested disproportionate increases in fat accrual. Adrenal suppression was the main toxicity of MA.
  
  
  CONCLUSIONS
  In children with high-risk malignancies, MA resulted in significant increases in mean percent weight change compared to placebo. Further studies of MA should be pursued to better delineate the effect on nutritional status.
authors.affiliation: Pediatric Hematology-Oncology-BMT, CancerCare Manitoba, University of Manitoba, Winnipeg, Canada.
authors.forename: Geoff D E
authors.initials: GD
authors.lastname: Cuvelier
authors.forename: Tina J
authors.initials: TJ
authors.lastname: Baker
authors.forename: Elaine F
authors.initials: EF
authors.lastname: Peddie
authors.forename: Linda M
authors.initials: LM
authors.lastname: Casey
authors.forename: Pascal J
authors.initials: PJ
authors.lastname: Lambert
authors.forename: Dianne S
authors.initials: DS
authors.lastname: Distefano
authors.forename: Marlene G
authors.initials: MG
authors.lastname: Wardle
authors.forename: Beth A
authors.initials: BA
authors.lastname: Mychajlunow
authors.forename: Marcel A
authors.initials: MA
authors.lastname: Romanick
authors.forename: David B
authors.initials: DB
authors.lastname: Dix
authors.forename: Beverly A
authors.initials: BA
authors.lastname: Wilson
mesh_terms: D000293:Adolescent; D000328:Adult; D001066:Appetite / Q000187:drug effects*; D019167:Appetite Stimulants / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D004311:Double-Blind Method; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D008297:Male; D019290:Megestrol Acetate / Q000627:therapeutic use*; D009367:Neoplasm Staging; D009369:Neoplasms / Q000150:complications* / Q000628:therapy; D009748:Nutrition Disorders / Q000175:diagnosis / Q000188:drug therapy* / Q000209:etiology; D011379:Prognosis; D011788:Quality of Life; D015431:Weight Loss / Q000187:drug effects*; D055815:Young Adult
chemical_list: D019167:Appetite Stimulants; D019290:Megestrol Acetate
keywords: appetite stimulant; megestrol acetate; pediatric cancer; randomized trial; weight loss