Quality of patient‐reported outcome reporting across cancer randomized controlled trials according to the CONSORT patient‐reported outcome extension: A pooled analysis of 557 trials

Abstract

Background The main objectives of this study were to identify the number of randomized controlled trials (RCTs) that have included a patient-reported outcome (PRO) endpoint across a wide range of cancer specialties and to evaluate completeness of PRO reporting according to the CONSORT PRO extension. Methods RCTs with a PRO endpoint, conducted across several cancer specialties and published between 2004 and 2013 were considered. Studies were evaluated based on previously defined criteria, including the CONSORT PRO extension and the Cochrane Collaboration's tool for assessing RCT Risk of Bias. Analyses were also conducted by type of PRO endpoint (primary versus secondary) and by cancer disease site. Results A total of 56,696 potentially eligible records were scrutinized and 557 RCTs with a PRO evaluation, enrolling overall 254,677 patients, were identified. PROs were most frequently used in RCTs of breast (N=123), lung (N=85) and colorectal (N=66) cancer. Overall, PROs were secondary endpoint in 421 (76%) RCTs. Four out of six CONSORT PRO items evaluated were documented in less than 50% of the RCTs. Level of reporting was higher in RCTs with PRO as a primary endpoint. Presence of a supplementary report was the only statistically significant factor associated with greater completeness of reporting for both RCTs with PRO as primary (β=0.19; P=0.001) or secondary endpoint (β=0.30; P<0.001). Conclusion Implementation of the CONSORT PRO extension is equally important across all cancer specialties. Its use can also contribute in revealing the robust PRO design of some studies, which might be obscured by poor outcome reporting.

Methodological aspects of health‐related quality of life measurement and analysis in patients with multiple myeloma

“…Diversity in applied PRO methodologies and poor quality of PRO reporting from clinical trials has previously been reported (Brundage et al , 2011a; Lemieux et al , ; Dirven et al , ; Efficace et al , , ; Bylicki et al , ; Hamel et al , ). However, to our knowledge, this is the first review to investigate methodological aspects in PRO measurements and analyses in studies of patients with MM.…”

Preliminary evidence on the uptake, use and benefits of the CONSORT-PRO extension

“…Again, these are knowledge transfer concerns requiring intervention to improve reporting practices and to ensure PRO results are interpreted accurately so they can appropriately inform patient care. Recent reviews confirm that reporting of PRO endpoints remains unsatisfactory overall; particularly regarding the reporting of PRO hypotheses, methodology, missing data, and generalisability of results [19][20][21][22][23][24][25][26]. Failing to report this information is wasteful as it limits the potential for readers to appraise the effect of interventions on patient health status, and the potential for PRO systematic reviews to impact clinical recommendations and health policy [27,28].…”

“…It may also decrease clinicians' confidence in the value of PRO data [29]. These aforementioned reviews [19][20][21][22][23][24] predominately include RCTs published before CONSORT-PRO. We expect that adherence to CONSORT-PRO will improve with time, as awareness and uptake increases.…”

Propensity score methods and regression adjustment for analysis of nonrandomized studies with health‐related quality of life outcomes

“…The importance of health‐related quality of life (HRQoL) outcomes in medical research is now critical to better inform patient care and to facilitate clinical decision‐making. Cancer clinical trials, for example, now frequently include HRQoL as an outcome (either primary or secondary) along with more traditional clinical or laboratory outcomes . In studies including a HRQoL evaluation, an important aspect is often the estimate of the average treatment effect on the treated (ATT) on patients' self‐reported health status and/or symptoms.…”

“…Cancer clinical trials, for example, now frequently include HRQoL as an outcome (either primary or secondary) along with more traditional clinical or laboratory outcomes. 1 In studies including a HRQoL evaluation, an important aspect is often the estimate of the average treatment effect on the treated (ATT) on patients' self-reported health status and/or symptoms. ATT is the expected difference between observed outcomes of treated subjects and those they would experience had they not been treated (unobserved).…”