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, 9met 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.
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, 9met 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.
| Original language | English |
|---|---|
| Pages (from-to) | 326-333 |
| Number of pages | 8 |
| Journal | JAMA dermatology |
| Volume | 156 |
| Issue number | 3 |
| DOIs | |
| Publication status | Published - 15 Jan 2020 |
