The correlation between patient characteristics and expectations of benefit from Phase I clinical trials.

Background: Patients in Phase I clinical trials sometimes report high expectations regarding the benefit of treatment. The authors examined a range of patient characteristics to determine which factors were associated with greater expectations of benefit from Phase I trials.

Methods: Participants were adult patients with cancer who had been offered participation in Phase I studies and had decided to participate. Patients completed interviewer-administered surveys before initiation of treatment. Physicians assessed Eastern Cooperative Oncology Group performance status for each patient. Statistical analyses (Pearson product moment correlation and t tests) used multiple imputation to account for missing data.

Results: Overall, 593 patients who were offered participation in Phase I trials were contacted, and 328 patients agreed to participate in a study of decision making by cancer patients. Of these, 260 patients (79%) enrolled in a Phase I trial. Patients' expectations regarding the chance that their disease would be controlled with experimental therapy were unrelated to age, gender, living situation, education level, or functional status. Expectations were correlated positively with beliefs about the benefit of standard therapy and the maximum benefit patients may experience from experimental therapy. Greater expectations of benefit were associated with better health-related quality of life, stronger religious faith, optimism, relative health stock, monetary risk seeking, and poorer numeracy.

Conclusions: Expectations expressed as beliefs in personal outcomes may be related more to quality of life and personality variables than to patients' knowledge or functional status. Whether such expectations are accurate reflections of knowledge has important implications for evaluating the informed consent process.