Goldberg, R.M., Loprinzi, C.L., Mailliard, J.A., O'Fallon, J.R., Krook, J.E., Ghosh, C., . . . Shanahan, T.G. (1995). Pentoxifylline for treatment of cancer anorexia and cachexia? A randomized, double-blind, placebo-controlled trial. Journal of Clinical Oncology, 13, 2856–2859.

Patients were randomized, in double-blind fashion, to pentoxifylline 400 mg by mouth three times daily or placebo three times daily. If patients lost 5% of their on-study weight, the bind was broken and patients could cross over to the pentoxifylline arm.

• N = 70 patients (randomized, 35 in each arm)
• ELIGIBILITY CRITERIA: Older than 18 years of age, Eastern Cooperative Oncology Group (ECOG) performance status 0–2, life expectancy greater than three months, weight loss greater than five pounds over two months, estimated calorie intake of less than 20 kcal/kg/d
• EXCLUSION CRITERIA: Parenteral or tube feeding; edema or ascites; use of corticosteroids, androgens, progestational agents, or hydrazine sulfate within one month; mechanical gastrointestinal (GI) obstruction; more than five episodes of emesis per week; primary or metastatic brain tumors; hemoptysis, GI hemorrhage, or Sr. Cr more than two times normal
• Patients were stratified by
  • Primary tumor site
  • Amount of weight loss
  • Use of chemotherapy (CDDP versus non-CDDP)
  • Sex, ECOG performance status, and MD estimate of survival.

Patients were randomized in a double-blind fashion.

• Objective evidence of weight change (excluded if patient had edema or ascites)
• Questionnaires to evaluate appetite and perceived food intake

No significant difference was seen between cohorts in weight gain (P = .43). According to the questionnaires, there was no evidence that pentoxifylline is more effective than placebo in enhancing appetite. Toxicity data were similar for both groups for nausea and vomiting, fluid retention, and abdominal pain. The study was closed after the planned interim analysis, and it was concluded that pentoxifylline is not an effective treatment for cancer anorexia and cachexia.

The 95% confidence interval for the difference in the percentage of weight gain for pentoxifylline minus placebo ranged from a loss of 3.3% to a gain of 1.9%; this confidence interval suggests that the percentage of weight gain for pentoxifylline-treated patients is about the same for patients receiving placebo.

• Heterogeneous group of patients with cancers arising from a variety of primary sites