Aim The aim for this study is to investigate the methodological quality and potential impact on clinical decision making of patient reported outcome (PRO) assessment in randomised controlled trials (RCTs) in the gynaecological cancer sites.
Methods A systematic review identified RCTs published between January 2004 and June 2012. Relevant studies were evaluated using a pre-determined extraction form which included: (1) Trial demographics and clinical and PRO characteristics; (2) level of PRO reporting and (3) bias, assessed using the Cochrane Risk of Bias tool. All studies were additionally analysed in relation to their relevance in supporting clinical decision making.
Results Fifty RCTs enrolling 24,991 patients were identified. In eight RCTs (16%) a PRO was the primary end-point. Twenty-one studies (42%) were carried out in a multi-national context. Where statistically significant PRO differences between treatments were found, it related in most cases to both symptoms and domains other than symptoms (n = 17, 57%). The majority of studies (n = 42, 84%) did not mention the mode of administration nor the methods of collecting PRO data. Statistical approaches for dealing with missing data were only explicitly mentioned in nine RCTs (18%). Sixteen RCTs (32%) were considered to be of high-quality and thus able to inform clinical decision making. Higher-quality PRO studies were generally associated with RCTs that were at a low risk of bias.
Conclusion This study showed that RCTs with PROs were generally well designed and conducted. In a third the information was very informative to fully understand the pros and cons of PROs treatment decision-making.
This paper stems from a larger project (i.e. Patient Reported Outcome Measurements Over Time In ONcology-PROMOTION Project) funded by a research
grant from the European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Group. Also, additional support for the conduct of the study
was provided by the Italian Group for Adult Hemato logic Diseases (GIMEMA). JMB is supported by the MRC ConDuCT Hub for Trials Methodology Research. We also acknowledge Alessandro Perreca and Salvatore Soldati, from the GIMEMA, for their
contribution to data management.
- Clinical decision-making
- Clinical trials
- Gynaecologic cancer
- Patient-reported outcomes
- Quality of life