Kardinal, C.G., Loprinzi, C.L., Schaid, D.J., Hass, A.C., Dose, A.M., Athmann, L.M., . . . Schray, M.F. (1990). A controlled trial of cyproheptadine in cancer patients with anorexia and/or cachexia. Cancer, 65(12), 2657–2662.

Intervention Characteristics/Basic Study Process

  • Cyproheptadine 8 mg by mouth three times daily versus placebo
  • Patients stratified for
    • Primary disease site: lung, gastrointestinal, or other
    • Chemotherapy: CDDP or non-CDDP
    • Amount of RT: whole or upper abdomen

Sample Characteristics

  • N = 293 evaluable patients (143 cyproheptadine, 150 placebo)
  • KEY DISEASE CHARACTERISTICS: The three groups entered had the following key disease characteristics.
  1. Advanced malignant disease with no definite planned cytotoxic therapy with weight loss of five pounds over two months and intake less than 20 calories/kg
  2. Same as 1 but patients receiving chemo or immunotherapy
  3. Same as 1 but receiving upper abdominal or whole abdominal RT with planned dose greater than 2500 cGy
  • EXCLUSION CRITERIA: Tube feeding or parenteral nutrition; life expectancy less than six weeks; ascites, pedal edema; mechanical obstruction; brain tumors; glaucoma; urinary hesitancy; concomitant treatment with steroids, progestational agents, and MAOI inhibitors

 

Study Design

  • Randomized, placebo-controlled, double-blinded clinical trial

Measurement Instruments/Methods

  • Percentage of weight change from baseline values (Patients’ weights were monitored monthly by HCP, and patients were asked to weigh themselves weekly at home.)
  • Patient appetite questionnaire
  • Food intake questionnaire
  • Perceived drug benefits and toxicities (e.g., lethargy, sedation, nausea, dry mouth)

Results

  • Appetite improved
    • 64% of cyproheptadine
    • 50% of placebo
    • P = 0.02
  • Average maximum weight gain
    • Cyproheptadine: 0.9 pounds
    • Placebo: 0.5 pounds
    • P = 0.78
  • Average weight loss
    • Cyproheptadine: 4.5 pounds
    • Placebo: 4.9 pounds
    • P = 0.72
  • Toxicities: Lethargy, dizziness, dry mouth

Conclusions

Cyproheptadine in patients with advanced malignant disease showed mild appetite enhancement but no significant weight enhancing effect.

Limitations

  • Patients remained in the study for a short time. The median time in the study was 34 days with cyproheptadine and 39 days with placebo. Only 25% of the patients entered still were participating in it after three months. 
  • The intervention was done late in the disease trajectory.