COMET Initiative | A core outcome domain set for clinical research on capillary malformations (the COSCAM project): an e-Delphi process and consensus meeting

A core outcome domain set for clinical research on capillary malformations (the COSCAM project): an e-Delphi process and consensus meeting

Background
There is limited evidence on the best available treatment options for capillary malformations (CMs), mainly due to the absence of uniform outcome measures in trials on therapies. A core outcome set (COS) enables standard reporting of trial outcomes, which facilitates comparison of treatment results.

Objectives
To develop a core outcome domain set (CDS), as part of a core outcome set (COS), for clinical research on CMs.

Methods
Sixty-seven potentially relevant outcome subdomains were recognized based on the literature, focus group sessions, and input from the COSCAM working group. These outcome subdomains were presented in an online Delphi study to CM experts (medical specialists and authors of relevant literature) and (parents of) patients with CM (international patient associations). During three e-Delphi study rounds, the participants repeatedly scored the importance of these outcome subdomains on a seven-point Likert scale. Participants could also propose other relevant outcome subdomains. Consensus was defined as = 80% agreement as to the importance of an outcome subdomain among both stakeholder groups. The CDS was finalized during an online consensus meeting.

Results
In total 269 participants from 45 countries participated in the first e-Delphi study round. Of these, 106 were CM experts from 32 countries, made up predominantly of dermatologists (59%) and plastic surgeons (18%). Moreover, 163 (parents of) patients with CM from 28 countries participated, of whom 58% had Sturge–Weber syndrome. During the two subsequent e-Delphi study rounds, 189 and 148 participants participated, respectively. After the entire consensus process, consensus was reached on 11 outcome subdomains: colour/redness, thickness, noticeability, distortion of anatomical structures, glaucoma, overall health-related quality of life, emotional functioning, social functioning, tolerability of intervention, patient satisfaction with treatment results, and recurrence.

Conclusions
We recommend the CDS to be used as a minimum reporting standard in all future trials of CM therapy. Our next step will be to select suitable outcome measurement instruments to score the core outcome subdomains

Aim

The objective of this study was to finalize the second development stage, in order to reach international consensus on the CDS for clinical research on CMs.

Contributors

Ginger Beau Langbroek, Albert Wolkerstorfer, Sophie E.R. Horbach, Phyllis I. Spuls, Kristen M. Kelly, Susan J. Robertson, M. Ingmar van Raath, Firas Al-Niaimi, Taro Kono, Pablo Boixeda, Hans J. Laubach, Ashraf M. Badawi, Agneta Troilius Rubin,Merete Haedersdal, Woraphong Manuskiatti, Chantal M.A.M. van der Horst, D.T. Ubbink, and on behalf of the COSCAM study group

Publication

Journal: BJD
Volume: 187
Issue: 5
Pages: 730 - 742
Year: 2022
DOI: 10.1111/bjd.21723

Further Study Information

Current Stage: Completed
Date:
Funding source(s): None

Health Area

Disease Category: Heart & circulation

Disease Name: Capillary malformation

Target Population

Age Range: Unknown

Sex: Either

Nature of Intervention: Any

Stakeholders Involved

- Clinical experts
- Consumers (caregivers)
- Consumers (patients)

Study Type

- COS for clinical trials or clinical research

Method(s)

- Consensus meeting
- Delphi process
- Focus group(s)
- Systematic review

The first stage involved a systematic review, focus group sessions, and discussions with the COSCAM founding group. This created an initial list for a 3-round delphi survey. After the delphi survey there was a final online consensus meeting to create the final COS.