title: A core outcome set for localised prostate cancer effectiveness trials.
abstract: To develop a core outcome set (COS) applicable for effectiveness trials of all interventions for localised prostate cancer. Many treatments exist for localised prostate cancer, although it is unclear which offers the optimal therapeutic ratio; which is confounded by inconsistencies in the selection, definition, measurement and reporting of outcomes in clinical trials.
  
  
  
  A list of 79 outcomes was derived from a systematic review of published localised prostate cancer effectiveness studies and semi-structured interviews with 15 patients with prostate cancer patients. A two-stage consensus process involving 118 patients and 56 international healthcare professionals (HCPs; cancer specialist nurses, urological surgeons and oncologists) was undertaken, consisting of a three-round Delphi survey followed by a face-to-face consensus panel meeting of 13 HCPs and eight patients.
  
  
  
  The final COS included 19 outcomes. In all, 12 apply to all interventions: death from prostate cancer, death from any cause, local disease recurrence, distant disease recurrence/metastases, disease progression, need for salvage therapy, overall quality of life, stress urinary incontinence, urinary function, bowel function, faecal incontinence, and sexual function. Seven were intervention-specific: perioperative deaths (surgery), positive surgical margin (surgery), thromboembolic disease (surgery), bothersome or symptomatic urethral or anastomotic stricture (surgery), need for curative treatment (active surveillance), treatment failure (ablative therapy), and side-effects of hormonal therapy (hormone therapy). The UK-centric participants may limit the generalisability to other countries, but trialists should reason why the COS would not be applicable. The default position should not be that a COS developed in one country will automatically not be applicable elsewhere.
  
  
  
  We have established a COS for trials of effectiveness in localised prostate cancer, applicable across all interventions that should be measured in all localised prostate cancer effectiveness trials.
authors.affiliation: Academic Urology Unit, University of Aberdeen, Aberdeen, UK.
authors.forename: Steven
authors.identifier: 0000-0002-2691-8421
authors.initials: S
authors.lastname: MacLennan
authors.affiliation: Health Services Research Unit, University of Aberdeen, Aberdeen, UK.
authors.forename: Paula R
authors.initials: PR
authors.lastname: Williamson
authors.affiliation: Department of Biostatistics, University of Liverpool, Liverpool, UK.
authors.forename: Hanneke
authors.initials: H
authors.lastname: Bekema
authors.affiliation: Department of Anesthesiology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands.
authors.forename: Marion
authors.initials: M
authors.lastname: Campbell
authors.affiliation: Department of Anesthesiology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands.
authors.forename: Craig
authors.initials: C
authors.lastname: Ramsay
authors.affiliation: Academic Urology Unit, University of Aberdeen, Aberdeen, UK.|Department of Urology, Aberdeen Royal Infirmary, Aberdeen, UK.
authors.forename: James
authors.initials: J
authors.lastname: N'Dow
authors.affiliation: Academic Urology Unit, University of Aberdeen, Aberdeen, UK.
authors.forename: Sara
authors.initials: S
authors.lastname: MacLennan
authors.affiliation: Health Economics Group, Institute of Health and Society, University of Newcastle, Newcastle, UK.
authors.forename: Luke
authors.initials: L
authors.lastname: Vale
authors.affiliation: Department of Urology, University of Minnesota, Minneapolis, MN, USA.|Minneapolis VA Health Care System, Minneapolis, MN, USA.
authors.forename: Philipp
authors.initials: P
authors.lastname: Dahm
authors.affiliation: Department of Urology, University Hospital, St. Etienne, France.
authors.forename: Nicolas
authors.initials: N
authors.lastname: Mottet
authors.affiliation: Academic Urology Unit, University of Aberdeen, Aberdeen, UK.|Department of Urology, Aberdeen Royal Infirmary, Aberdeen, UK.
authors.forename: Thomas
authors.initials: T
authors.lastname: Lam
mesh_terms: D002985:Clinical Protocols; D002986:Clinical Trials as Topic / Q000379:methods*; D057186:Comparative Effectiveness Research; D032921:Consensus; D003697:Delphi Technique; D018450:Disease Progression; D018572:Disease-Free Survival; D006801:Humans; D008297:Male; D008875:Middle Aged; D009364:Neoplasm Recurrence, Local; D011183:Postoperative Complications / Q000401:mortality / Q000628:therapy; D011468:Prostatectomy; D011471:Prostatic Neoplasms / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D011788:Quality of Life; D016896:Treatment Outcome
keywords: Delphi survey; clinical trials; consensus group meeting; consensus process; core outcome set; localised prostate cancer