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Consensus Statement
January 15, 2020

Core Outcome Set for Actinic Keratosis Clinical Trials

Author Affiliations
  • 1University of Cincinnati College of Medicine, Cincinnati, Ohio
  • 2Department of Dermatology, Feinberg School of Medicine, Northwestern University, Chicago, Illinois
  • 3Division of Dermatology, College of Medicine Tucson, University of Arizona, Tucson, Arizona
  • 4Department of Dermatology, West Virginia University, Morgantown
  • 5Department of Dermatology, St Louis University, St Louis, Missouri
  • 6Department of Dermatology, Perelman School of Medicine, University of Pennsylvania, Philadelphia
  • 7Division of Cardiology, Feinberg School of Medicine, Northwestern University, Chicago, Illinois
  • 8Division of Dermatology, Department of Medicine, University of Toronto, Toronto, Ontario, Canada
  • 9Division of Dermatology, Department of Medicine, Women's College Research Institute, Women's College Hospital, Toronto, Ontario, Canada
  • 10Private practice, Media, Pennsylvania
  • 11Department of Dermatology, Royal London Hospital, Barts Health National Health Service Trust, London, United Kingdom
  • 12Blizard Institute, Barts and the London School of Medicine and Dentistry, Centre for Cutaneous Research and Cell Biology, Queen Mary University of London, London, United Kingdom
  • 13Division of Plastic and Reconstructive Surgery, Feinberg School of Medicine, Northwestern University, Chicago, Illinois
  • 14Department of Dermatology, Memorial Sloan Kettering Cancer Center, New York City, New York
  • 15Department of Ophthalmology, Feinberg School of Medicine, Northwestern University, Chicago, Illinois
  • 16Department of Dermatology, Churchill Hospital, Oxford, United Kingdom
  • 17Department of Dermatology, University of Minnesota, Minneapolis
  • 18Department of Radiation Oncology, Feinberg School of Medicine, Northwestern University, Chicago, Illinois
  • 19Department of Otolaryngology–Head and Neck Surgery, Feinberg School of Medicine, Northwestern University, Chicago, Illinois
  • 20Department of Dermatology & Skin Science, University of British Columbia, Vancouver, British Columbia, Canada
  • 21Center for Evidence-Based Healthcare, Carl Gustav Carus, TU Dresden, Dresden, Germany
  • 22Department of Biostatistics, University of Liverpool, Liverpool, United Kingdom
  • 23Department of Dermatology, Perelman Center for Advanced Medicine, Hospital of the University of Pennsylvania, Philadelphia
  • 24Division of Dermatologic Surgery, Department of Dermatology, Hospital of the University of Pennsylvania, Philadelphia
  • 25Department of Dermatology, Penn State Health, Milton S. Hershey Medical Center, Hershey, Pennsylvania
  • 26Department of Surgery, Feinberg School of Medicine, Northwestern University, Chicago, Illinois
JAMA Dermatol. 2020;156(3):326-333. doi:10.1001/jamadermatol.2019.4212
Key Points

Question  What are the most important outcomes to report in clinical trials on actinic keratosis?

Findings  In this survey study including physician and patient stakeholders (33 in round 1 and 29 in round 2), a consensus was reached regarding a core set of 6 of 137 outcomes and domains of actinic keratosis: complete clearance of actinic keratoses, percentage of actinic keratoses cleared, severity of adverse events, patient perspective on effectiveness, patient-reported future treatment preference, and rate of recurrence.

Meaning  In studies of treatment of actinic keratosis, the recommended core outcomes should be reported as a minimum to facilitate comparison of results across studies.

Abstract

Importance  Although various treatments have been found in clinical trials to be effective in treating actinic keratosis (AK), researchers often report different outcomes. Heterogeneous outcome reporting precludes the comparison of results across studies and impedes the synthesis of treatment effectiveness in systematic reviews.

Objective  To establish an international core outcome set for all clinical studies on AK treatment using systematic literature review and a Delphi consensus process.

Evidence Review  Survey study with a formal consensus process. The keywords actinic keratosis and treatment were searched in PubMed, Embase, CINAHL, and the Cochrane Library to identify English-language studies investigating AK treatments published between January 1, 1980, and July 13, 2015. Physician and patient stakeholders were nominated to participate in Delphi surveys by the Measurement of Priority Outcome Variables in Dermatologic Surgery Steering Committee members. All participants from the first round were invited to participate in the second round. Outcomes reported in randomized controlled clinical trials on AK treatment were rated via web-based e-Delphi consensus surveys. Stakeholders were asked to assess the relative importance of each outcome in 2 Delphi survey rounds. Outcomes were provisionally included, pending the final consensus conference, if at least 70% of patient or physician stakeholders rated the outcome as critically important in 1 or both Delphi rounds and the outcome received a mean score of 7.5 from either stakeholder group. Data analysis was performed from November 5, 2018, to February 27, 2019.

Findings  A total of 516 outcomes were identified by reviewing the literature and surveying key stakeholder groups. After deduplication and combination of similar outcomes, 137 of the 516 outcomes were included in the Delphi surveys. Twenty-one physicians and 12 patients participated in round 1 of the eDelphi survey, with 17 physicians (81%) retained and 12 patients (100%) retained in round 2. Of the 137 candidate outcomes, 9 met a priori Delphi consensus criteria, and 6 were included in the final outcomes set after a consensus meeting: complete clearance of AKs, percentage of AKs cleared, severity of adverse events, patient perspective on effectiveness, patient-reported future treatment preference, and recurrence rate. It was recommended that treatment response be assessed at 2 to 4 months and recurrence at 6 to 12 months, with the AK rate of progression to cutaneous squamous cell carcinoma reported whenever long-term follow-up was possible.

Conclusions and Relevance  Consensus was reached regarding a core outcome set for AK trials. Further research may help determine the specific outcome measures used to assess each of these outcomes.

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