Patient-reported outcomes in randomised controlled trials of gynaecological cancers: investigating methodological quality and impact on clinical decision-making.
OBJECTIVE
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.


CONCLUSIONS
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.
Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), Rome, Italy. Electronic address: f.efficace@gimema.it.
Fabio
F
Efficace
Academic Medical Center, University of Amsterdam, Department of Medical Psychology, Amsterdam, The Netherlands.
Marc
M
Jacobs
Department of Surgery, Memorial Sloan Kettering Cancer Center, New York, USA.
Andrea
A
Pusic
Department of Obstetrics and Gynecology, Medical University of Graz, Graz, Austria.
Elfriede
E
Greimel
Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), Rome, Italy.
Alfonso
A
Piciocchi
Division of Psychosocial Research & Epidemiology, The Netherlands Cancer Institute, Amsterdam, The Netherlands.
Jacobien M
JM
Kieffer
Leeds Institute of Cancer Studies and Pathology, University of Leeds, Leeds, UK.
Alexandra
A
Gilbert
Institute of Applied Health Sciences, University of Aberdeen, Aberdeen, UK.
Peter
P
Fayers
Centre for Surgical Research, University of Bristol and University Hospitals NHS Foundation Trust, Bristol, UK.
Jane
J
Blazeby
D003657:Decision Making; D005260:Female; D005833:Genital Neoplasms, Female / Q000628:therapy*; D006801:Humans; D063868:Patient Outcome Assessment; D016032:Randomized Controlled Trials as Topic / Q000379:methods* / Q000592:standards*; D057566:Self Report; D016896:Treatment Outcome
Clinical decision-making; Clinical trials; Gynaecologic cancer; Patient-reported outcomes; Quality of life