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.
Bibliographical noteFunding Information:
Funding/Support: This study was supported by departmental research funds from the Department of Dermatology, Northwestern University; Merz Center for Quality and Outcomes Research in Dermatologic Surgery; and the IMPROVED (Measurement of Priority Outcome Variables in Dermatologic Surgery) Group.
reported serving as an investigator for and receiving research funding from Sanofi and Regeneron; being a consultant for Sanofi, RTI Health Solutions, Eczema Society of Canada, and Canadian Agency for Drugs and Technology in Health; and receiving honoraria from Prime Inc, Spire Learning, CME Outfitters, Eczema Society of Canada, and the Canadian Dermatology Association; his institution has received educational grants from Sanofi and AbbVie. Dr Etzkorn reported receiving grants from the Dermatology Foundation during the conduct of the study. Dr Harwood reported personal fees from Sanofi and Roche outside the submitted work. Dr Lee reported receiving personal fees from UptoDate outside the submitted work. Dr Schmitt reported receiving grants from Novartis, Pfizer, ALK, and Sanofi and personal fees from Novartis and Sanofi outside the submitted work. Dr Alam reported receiving personal fees from Pulse Biosciences outside the submitted work. No other disclosures were reported.
PubMed: MeSH publication types
- Journal Article
- Research Support, Non-U.S. Gov't
- Systematic Review