Background: Due to a large variety in treatment outcomes reported in therapeutic trials and lacking patient-relevant outcomes, it is hard to adequately compare and improve current therapies for patients with capillary malformations (CMs). The Core Outcome Set for Capillary Malformations (COSCAM) project aims to develop a core outcome set (COS) for use in future CM trials, in which we will first develop a core outcome (sub)domain set (CDS). Here, we describe the methods for the development of a CDS and present the results of the first development stage.
Methods: The COSCAM project is carried out according to the recommendations of the Cochrane Skin Core OUtcomes Set INitiative (CS-COUSIN) and the Core Outcome Measures in Effectiveness Trials (COMET) initiative. During the first stage, we identified all potentially relevant outcome subdomains based on a systematic review, two focus group sessions and input from patient representatives of Dutch patient organizations and the COSCAM-founding group. In stage two, we will present the subdomains in a three-round e-Delphi study and online consensus meeting, in which CM patients, parents/caregivers and CM experts worldwide rate the importance of the proposed subdomains, hereby finalizing the core outcome (sub)domains of the CDS.
Results: A total of 67 potential outcome subdomains were included; sixteen were previously used in the literature, 20 were proposed by Dutch patients and their parents/caregivers (n = 13) in focus group sessions and 38 were suggested by the experts of the COSCAM-founding group. Seven were excluded because of overlap.
Conclusion: The final CDS may serve as a minimum standard in future CM trials, thereby facilitating adequate comparison of treatment outcomes. After this CDS development, we will select appropriate outcome measurement instruments to measure the core outcome subdomains.
This Core Outcome Set for Capillary Malformations (COS-CAM) project first aims to reach international consensus on a CDS as part of a COS, in order to unify outcome use and reporting for CMs worldwide. Here, we describe the methods to develop a CDS and present the results of the first development stage
ContributorsG B Langbroek, A Wolkerstorfer, S E R Horbach, P I Spuls, K M Kelly, S J Robertson, M I van Raath, F Al-Niaimi, T Kono, P Boixeda, H J Laubach, A M Badawi, A Troilius Rubin, M Haedersdal, W Manuskiatti, C M A M van der Horst, D T Ubbink, COSCAM study group
Disease Category: Skin
Disease Name: Capillary malformation
Age Range: Unknown
Sex: Either
Nature of Intervention:
- Consumers (caregivers)
- Consumers (patients)
- Patient/ support group representatives
- COS for clinical trials or clinical research
- Focus group(s)
- Systematic review
In this first stage of the COS development, a systematic review (literature search from 2005 to May 2020) was conducted. Next, two small online focus group sessions, including nine Dutch CM patients and/or their parents/caregivers, were organized to collect patient-reported outcome subdomains. The focus group sessions were conducted in May 2020.