NCIC Clinical Trials Group experience of employing patient-reported outcomes in clinical trials: an illustrative study in a palliative setting

摘要

In this article we briefly review the experience of the National Cancer Institute of Canada (NCIC)nClinical Trials Group (CTG) with respect to the assessment of patient reported outcomes innclinical trials, and illustrate issues important to assessing symptom palliation in clinical trials ofncancer therapy. We highlight a standard approach taken by the NCIC CTG, and illustrate hownthis approach may be applied to the complex problem of symptom control analysis in patientsnwith locally advanced NSCLC. We further illustrate how variations in this analysis yield differentnapparent rates of palliation. Apparent rates of palliation critically depended on the outcomenmeasures used: single symptom response across patients (5–32%, depending on the symptomnof interest), symptom response in specific symptomatic patients (37–100%), symptom controln(45–82%), index symptom response (60%), proportion of patients experiencing improvementnin all symptoms (21%), or health-related quality of life (HRQoL) improvement (23%, global).nRates also varied substantively depending on which cohort of patients was considered relevantnto each analysis (i.e., was included in the respective denominator). Substantive discordance innpatients’ apparent palliation was seen when HRQoL data were compared with symptom diaryndata. Appropriate and valid descriptions of palliative outcomes in clinical trials are complexnundertakings. We conclude that several measures are required for a textured clinical descriptionnof outcome, and recommend reporting palliation according to individual symptom responsenrates and HRQoL response rates, in order to address each construct of palliation success.