Methodological Issues Regarding Cancer Anorexia/Cachexia Trials

This chapter reviews information regarding conducting and analyzing trials designed to test potential agents for alleviating cancer-related anorexia/cachexia. The information was derived from experience generated by the North Central Cancer Treatment Group (NCCTG) in conducting a series of ten trials which, to date, have entered over 2000 patients. As with any research endeavor, the study of anorexia/cachexia is difficult and fraught with potential pitfalls. The discussion here may aid in the standardization of design and analytic approaches for anorexia/cachexia trials so that a meta-analytic process, formal or otherwise, may be facilitated. The overriding principle has been to keep the process as simple as possible due to the many potentially concomitant confounding influences. If the treatments applied to anorexia/cachexia do not produce profound results in the presence of substantial systemic noise, then they are not likely to be seen as efficacious in the community clinical environment beyond the clinical trials setting.