Tomiska, M., Tomiskova, M., Salajka, F., Adam, Z., & Vorlicek, J. (2003). Palliative treatment of cancer anorexia with oral suspension of megestrol acetate. Neoplasma, 50, 227–233.

To evaluate the effect of different doses of oral suspension of megestrol acetate (MA) on appetite, quality of life, body weight, and anthropometric measures in patients with advanced cancer

Researchers evaluated a fixed dose of MA (480 mg) versus escalating doses of MA. Patients were evaluated at baseline, two, four, and eight weeks.

• The study included 22 patients with advanced, nonhormonally dependent cancer who were experiencing anorexia-related weight loss (at least 5% of usual body weight).
• Patients had a life expectancy of more than three months, and a World Health Organization performance status of two or less.
• Median patient age was 59 years.
• A total of 19 patients were evaluated (2 died, 1 withdrew): 15 men and 4 women.
• Patients were diagnosed with lung cancer (nine), gastrointestinal cancer (six), renal cancer (two), mesothelioma (one), and non-Hodgkin lymphoma (one).
• At eight weeks, 12 patients were evaluated (eight patients died, one withdrew from study, and one stopped because of side effects).

The study was a randomized, multicenter, blinded, two-arm trial.

• Visual analog scale using 0–100 mm to measure appetite (patient self-report)
• Body weight
• Anthropometric measures of mid-arm circumference and four skinfold thickness
• Hand grip strength
• Serum measure of albumin, prealbumin, c-reactive protein, and cortisol
• European Organization for Research and Treatment Cancer Core Quality of Life questionnaire (EORTC QLQ-C30, version 1.0)

Twelve patients were evaluated at eight weeks (eight patients died, one withdrew from study, and one stopped because of side effects). Therefore, the patients were evaluated as a single group regardless of whether they were receiving a fixed dose or an escalating dose of MA. There was statistically significant improvement in appetite over baseline in all patients at two weeks (p = 0.0001), four weeks (p = < 0.01), and eight weeks (p = 0.022). Overall quality of life improved in the majority of patients at two and eight weeks, but it was not statistically significant. There was no statistically significant improvement in body weight or other measures. In terms of side effects, 4 of 19 patients experienced clinically significant edema, resulting in 1 patient withdrawing from the study.

Original aims of the study could not be met because of the small sample size, so no conclusions can be drawn about the effectiveness of oral MA suspension or optimal or superior dose of MA.

• The study had a very small sample size, with significant attrition (36%).
• The study had no placebo group; all patients were assessed compared to their baseline measures.