Development of a core outcome domain set for clinical research on capillary malformations (the COSCAM project)

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  • G. 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
  • Hædersdal, Merete
  • W. Manuskiatti
  • C. M.A.M. van der Horst
  • D. T. Ubbink
  • the COSCAM study group

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.

TidsskriftJournal of the European Academy of Dermatology and Venereology
Udgave nummer9
Sider (fra-til)1888-1895
Antal sider8
StatusUdgivet - 2021

Bibliografisk note

Funding Information:
Dr. Haedersdal reports grants from Leo Pharma, grants from Procter and Gamble, non‐financial support from Cherry Imaging, non‐financial support from Cynosure‐Hologic, grants and non‐financial support from Lutronic, grants and non‐financial support from Mirai Medical, grants and non‐financial support from Novoxel, non‐financial support from Perfaction Technologies, outside the submitted work. Dr. Kelly reports grants and other from Michaelson Diagnostics, personal fees and other from Sciton, personal fees from IQVIA, other from Candela, outside the submitted work; and Board member of the American Society for Lasers in Surgery and Medicine. Dr. Robertson reports personal fees from Candela, outside the submitted work. Prof. dr. Spuls has done consultancies in the past for Sanofi 111017 and AbbVie 041217 (unpaid), received a departmental independent research grant for TREAT NL registry Pharma since December 2019 for the TREAT NL registry, is involved in performing clinical trials with many pharmaceutical industries that manufacture drugs used for the treatment of, e.g. psoriasis and atopic dermatitis, for which financial compensation is paid to the department/hospital and, is Chief Investigator (CI) of the systemic and phototherapy atopic eczema registry (TREAT NL) for adults and children and one of the main investigator of the SECURE‐AD registry. The other authors have nothing to declare.

Funding Information:
We thank the Dutch CM patients who participated in the focus group sessions, the patient representatives of the Dutch Wijnvlek-Sturge Weber Association and the HEVAS association for providing feedback on the list with potentially important outcome subdomains, and the international steering committee for providing feedback on the study protocol and for recruiting stakeholders in stage two of the CDS development.

Publisher Copyright:
© 2021 The Authors. Journal of the European Academy of Dermatology and Venereology published by John Wiley & Sons Ltd on behalf of European Academy of Dermatology and Venereology.

ID: 302045450