Placebo-controlled trial of medroxyprogesterone acetate in gastrointestinal malignancies and cachexia

Background: Cancer cachexia is a common and serve medical problem in the management of patients with cancer, and its treatment remains a therapeutic challenge. Medroxyprogesterone acetate (MPA), which has been used for many years for hormonal therapy of breast cancer, has been noted to produce weight gain and increase appetite unrelated to any antitumour effect. To assess its symptomatic therapeutic value in patients suffering from advanced gastrointestinal malignancies and cachexia, the present double-blind placebo-controlled trial was initiated. Methods: Thirty-one patients with advanced gastrointestinal malignancies suffering from anorexia and weight loss were entered into this study and randomised to receive the anabolic steroid medroxyprogesterone acetate (MPA) 500 mg/day or placebo for 3 months. The primary end-points for comparison were body weight, appetite, Karnofsky performance status and quality of life, all of which were serially evaluated in monthly intervals until death. Results: A beneficial effect of MPA was noted in terms of weight as well as quality of life (measured by the functional living index for cancer). In the treatment arm, median body weight increased from 58 kg at study entry to 61 kg after 3 months, as compared to a decrease in median weight from 57.5 kg to 55 kg in the placebo arm. Partial palliative responses in quality of life were documented in 4/10 (40%) evaluable patients treated with MPA versus 2/14 (14%) in those who received placebo. There was no difference between the two treatment groups with regard to appetite (an improvement was indicated by 6 patients in each arm), Karnofsky performance score or overall survival. Conclusion: In summary, our preliminary data seem to support the potential therapeutic value of MPA for symptomatic treatment of cancer cachexia in patients with advanced gastrointestinal malignancies.