Gordon JN et al. (2005) Thalidomide in the treatment of cancer cachexia: a randomised placebo controlled trial. Gut 54: 540–545

Profound metabolic disturbances resulting in severe muscle and fat wasting (cachexia) are significant in patients with advanced cancer. Several proinflammatory cytokines have been implicated in development of cachexia and anorexia; modulation of their effects might improve outcome in these patients. As thalidomide has been shown to downregulate the production of proinflammatory cytokines and improve cachexia associated with other conditions, its effect was investigated in this open-label, randomized, placebo-controlled trial in patients with advanced inoperable pancreatic cancer.

Of 50 randomized patients, 33 were evaluated at 4 weeks and 20 at 8 weeks. After this point, too few patients remained in the study for comparison to be statistically relevant—disease had progressed in 9 patients, 11 patients had died and a further 10 patients withdrew for other reasons. Patients receiving thalidomide (200 mg daily) lost significantly less weight and lean muscle mass at both time points than patients receiving placebo. Quality-of-life scores and overall survival duration did not differ significantly between the active and placebo groups; weight loss correlated with change in physical function. Thalidomide was generally well tolerated and adverse effects were comparable between groups, except for constipation (more common with thalidomide) and insomnia (less common with thalidomide).

This study was limited by its small evaluable sample size but indicates a role for thalidomide in maintaining body mass in patients with inoperable pancreatic cancer. It is not yet clear whether maintenance of mass would translate into survival benefit.