University of Cincinnati College of Medicine, Cincinnati, Ohio.
Department of Dermatology, Feinberg School of Medicine, Northwestern University, Chicago, Illinois.
JAMA Dermatol. 2020 Mar 1;156(3):326-333. doi: 10.1001/jamadermatol.2019.4212.
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.
To establish an international core outcome set for all clinical studies on AK treatment using systematic literature review and a Delphi consensus process.
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.
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.
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.
虽然临床试验中已经发现了各种治疗方法对光化性角化病（AK）有效，但研究人员经常报告不同的结果。结果报告的异质性使得无法在研究之间进行结果比较，并阻碍了系统评价中治疗效果的综合。
使用系统文献回顾和德尔菲共识过程为所有 AK 治疗的临床研究建立国际核心结果集。
一项具有正式共识过程的调查研究。在 PubMed、Embase、CINAHL 和 Cochrane Library 中搜索关键词“光化性角化病”和“治疗”，以确定 1980 年 1 月 1 日至 2015 年 7 月 13 日发表的关于 AK 治疗的英语研究。由测量皮肤病学手术优先结果变量指导委员会成员提名皮肤科医生和患者利益相关者参与德尔菲调查。第一轮的所有参与者都被邀请参加第二轮。通过基于网络的电子德尔菲共识调查对 AK 治疗的随机对照临床试验中报告的结果进行评分。利益相关者被要求在两轮德尔菲调查中评估每个结果的相对重要性。如果至少 70%的患者或医生利益相关者在一轮或两轮德尔菲调查中认为该结果至关重要，并且该结果得到任何利益相关者群体的平均 7.5 分，则该结果将被临时纳入，等待最终的共识会议。数据分析于 2018 年 11 月 5 日至 2019 年 2 月 27 日进行。
通过回顾文献和调查主要利益相关者群体，共确定了 516 个结果。在扣除重复项并合并相似的结果后，137 个结果被纳入德尔菲调查。21 名医生和 12 名患者参加了第一轮电子德尔菲调查，其中 17 名医生（81%）和 12 名患者（100%）保留在第二轮。在 137 个候选结果中，有 9 个符合德尔菲共识标准，并且在共识会议后有 6 个被纳入最终的结果集：AK 的完全清除率、清除 AK 的百分比、不良事件的严重程度、患者对有效性的看法、患者报告的未来治疗偏好和复发率。建议在 2 至 4 个月时评估治疗反应，并在 6 至 12 个月时评估复发情况，只要有可能进行长期随访，就报告 AK 进展为皮肤鳞状细胞癌的发生率。
就 AK 试验的核心结果集达成了共识。进一步的研究可能有助于确定用于评估这些结果的具体衡量标准。
