title: Patient-reported outcomes in randomised controlled trials of prostate cancer: methodological quality and impact on clinical decision making.
abstract: BACKGROUND
  Patient-reported outcomes (PRO) data from randomised controlled trials (RCTs) are increasingly used to inform patient-centred care as well as clinical and health policy decisions.
  
  
  OBJECTIVE
  The main objective of this study was to investigate the methodological quality of PRO assessment in RCTs of prostate cancer (PCa) and to estimate the likely impact of these studies on clinical decision making.
  
  
  METHODS
  A systematic literature search of studies was undertaken on main electronic databases to retrieve articles published between January 2004 and March 2012. RCTs were evaluated on a predetermined extraction form, including (1) basic trial demographics and clinical and PRO characteristics; (2) level of PRO reporting based on the recently published recommendations by the International Society for Quality of Life Research; and (3) bias, assessed using the Cochrane Risk of Bias tool. Studies were systematically analysed to evaluate their relevance for supporting clinical decision making.
  
  
  RESULTS
  Sixty-five RCTs enrolling a total of 22 071 patients were evaluated, with 31 (48%) in patients with nonmetastatic disease. When a PRO difference between treatments was found, it related in most cases to symptoms only (n=29, 58%). Although the extent of missing data was generally documented (72% of RCTs), few reported details on statistical handling of this data (18%) and reasons for dropout (35%). Improvements in key methodological aspects over time were found. Thirteen (20%) RCTs were judged as likely to be robust in informing clinical decision making. Higher-quality PRO studies were generally associated with those RCTs that had higher internal validity.
  
  
  CONCLUSIONS
  Including PRO in RCTs of PCa patients is critical for better evaluating the treatment effectiveness of new therapeutic approaches. Marked improvements in PRO quality reporting over time were found, and it is estimated that at least one-fifth of PRO RCTs have provided sufficient details to allow health policy makers and physicians to make critical appraisals of results.
  
  
  RESULTS
  In this report, we have investigated the methodological quality of PCa trials that have included a PRO assessment. We conclude that including PRO is critical to better evaluating the treatment effectiveness of new therapeutic approaches from the patient's perspective. Also, at least one-fifth of PRO RCTs in PCa have provided sufficient details to allow health policy makers and physicians to make a critical appraisal of results.
authors.affiliation: Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), Rome, Italy. Electronic address: f.efficace@gimema.it.
authors.forename: Fabio
authors.initials: F
authors.lastname: Efficace
authors.affiliation: Department of Surgery, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors.forename: Michael
authors.initials: M
authors.lastname: Feuerstein
authors.affiliation: Institute of Applied Health Sciences, University of Aberdeen, Aberdeen, United Kingdom.
authors.forename: Peter
authors.initials: P
authors.lastname: Fayers
authors.affiliation: Data Center and Health Outcomes Research Unit, Italian Group for Adult Hematologic Diseases (GIMEMA), Rome, Italy.
authors.forename: Valentina
authors.initials: V
authors.lastname: Cafaro
authors.affiliation: Department of Surgery, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors.forename: James
authors.initials: J
authors.lastname: Eastham
authors.affiliation: Department of Surgery, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
authors.forename: Andrea
authors.initials: A
authors.lastname: Pusic
authors.affiliation: Centre for Surgical Research, University of Bristol and Division of Surgery, University Hospitals Bristol NHS Foundation Trust, Bristol, United Kingdom.
authors.forename: Jane
authors.initials: J
authors.lastname: Blazeby
mesh_terms: D003657:Decision Making; D006801:Humans; D008297:Male; D063868:Patient Outcome Assessment; D011050:Policy Making; D011471:Prostatic Neoplasms / Q000628:therapy*; D011788:Quality of Life; D016032:Randomized Controlled Trials as Topic; D012107:Research Design / Q000592:standards*
keywords: Clinical decision making; Clinical trials; Patient-reported outcomes; Prostate cancer; Quality of life