Patient-reported outcomes in randomised controlled trials of colorectal cancer: an analysis determining the availability of robust data to inform clinical decision-making

Abstract

Purpose: Randomised controlled trials (RCTs) are the most robust study design measuring outcomes of colorectal cancer (CRC) treatments, but to influence clinical practice trial design and reporting of patient-reported outcomes (PROs) must be of high quality. Objectives of this study were as follows: to examine the quality of PRO reporting in RCTs of CRC treatment; to assess the availability of robust data to inform clinical decision-making; and to investigate whether quality of reporting improved over time.

Methods: A systematic review from January 2004-February 2012 identified RCTs of CRC treatment describing PROs. Relevant abstracts were screened and manuscripts obtained. Methodological quality was assessed using International Society for Quality of Life Research-patient-reported outcome reporting standards. Changes in reporting quality over time were established by comparison with previous data, and risk of bias was assessed with the Cochrane risk of bias tool.

Results: Sixty-six RCTs were identified, seven studies (10 %) reported survival benefit favouring the experimental treatment, 35 trials (53 %) identified differences in PROs between treatment groups, and the clinical significance of these differences was discussed in 19 studies (29 %). The most commonly reported treatment type was chemotherapy (n = 45; 68 %). Improvements over time in key methodological issues including the documentation of missing data and the discussion of the clinical significance of PROs were found. Thirteen trials (20 %) had high-quality reporting.

Conclusions: Whilst improvements in PRO quality reporting over time were found, several recent studies still fail to robustly inform clinical practice. Quality of PRO reporting must continue to improve to maximise the clinical impact of PRO findings.

Keywords: Cancer; Colorectal; Health-related quality of life; Patient-reported outcomes; Randomised; Trails.

Fig. 1

Schematic breakdown of literature search results of colorectal randomised controlled trials (preferred reporting items for systematic reviews and meta-analysis). PRO patient-reported outcomes

Fig. 2

Risk bar chart showing proportion of studies with low, unclear or high risk of bias across all RCTs (n = 66) by quality of PRO reporting

Fig. 3

Descriptive comparison of level of reporting on selected key PRO issues in RCTs of colorectal describe both clinical and patient-reported outcomes between 1980 and 2003 (yellow bars) (Efficace et al. 2003) and 2004 and 2012 (blue bars)