Using patient-reported outcomes to describe the patient experience on phase I clinical trials

Abstract

Background: Symptoms are common among patients enrolled in phase I trials. We assessed the validity of Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) items in relation to previously validated assessments of quality of life and psychological distress. We used data from a randomized trial testing a palliative care support intervention for patients enrolled on phase I trials. Methods: Patients (n ¼ 479) were accrued to the parent study prior to initiating a phase I clinical trial with data collected at baseline, 4, and 12 weeks. We determined the correlation of PRO-CTCAE with distress level, Functional Assessment of Cancer Therapy - General (FACT-G) total, and subscale domain scores. Results: Patients were predominantly female (56.8%) and older than age 60 years, and 30.7% were from minority populations. The correlation coefficient for distress level for all PRO-CTCAE items was small to moderate (Pearson r ¼ 0.33-0.46). Pearson correlation coefficient for FACT-G total was moderate (r ¼ -0.45 to -0.69). Stronger associations were noted for mood items of the PRO-CTCAE only (with distress level, r ¼ 0.55-0.6; with FACT-G, r ¼ -0.54 to -0.6). PRO-CTCAE symptom interference scores had the strongest correlation with distress level (Pearson r ¼ 0.46) and FACT-G total (Pearson r ¼ -0.69). Correlations between PRO-CTCAE items and corresponding FACT-G (total and subscales) and distress levels reached statistical significance for all items (P