TY - JOUR

T1 - A core outcome domain set for clinical research on capillary malformations (the COSCAM project)

T2 - an e-Delphi process and consensus meeting*

AU - Langbroek, Ginger Beau

AU - Wolkerstorfer, Albert

AU - Horbach, Sophie E.R.

AU - Spuls, Phyllis I.

AU - Kelly, Kristen M.

AU - Robertson, Susan J.

AU - van Raath, M. Ingmar

AU - Al-Niaimi, Firas

AU - Kono, Taro

AU - Boixeda, Pablo

AU - Laubach, Hans J.

AU - Badawi, Ashraf M.

AU - Rubin, Agneta Troilius

AU - Haedersdal, Merete

AU - Manuskiatti, Woraphong

AU - van der Horst, Chantal M.A.M.

AU - Ubbink, D. T.

AU - COSCAM study group

N1 - Funding Information: A.W. reports a lecture honorarium to the institution from Candela. P.I.S. has performed consultancies in the past for Sanofi and AbbVie (unpaid), has received departmental independent research grants for the TREAT NL registry, for which she is Chief Investigator, from pharma companies since December 2019, and is involved in performing clinical trials with many pharmaceutical companies that manufacture drugs used for the treatment of conditions such as psoriasis and atopic dermatitis, for which financial compensation is paid to the department or hospital. K.M.K. reports grants from Biophotas, personal fees from IQVIA, personal fees from FDZJ, grants from Orlucent, nonfinancial support from Sciton, and grants and nonfinancial support from Michaelson Diagnostics, outside the submitted work; and is secretary for the American Society for Laser Medicine and Surgery. S.J.R. reports personal fees from Candela (Syneron Medical HK Ltd), outside the submitted work. P.B. reports consulting fees, payment for lectures and nonfinancial support from Cynosure. H.J.L. reports payment or honoraria for lectures, presentations, speakers’ bureaus or educational events. A.T.R. is chair of the Multidisciplinary Vascular Anomaly Group at Skåne University Hospital, Malmö, Sweden. M.H. reports grants from LEO Pharma, nonfinancial support from Cherry Imaging, nonfinancial support from Cynosure‐Hologic, grants and nonfinancial support from Lutronic, grants and nonfinancial support from Mirai Medical, nonfinancial support from Perfaction Technologies, nonfinancial support from miraDry–Sientra, and grants and nonfinancial support from Venus Concept, outside the submitted work. The other authors declare they have no conflicts of interest. Funding Information: First, we thank all of the stakeholders who contributed to this study. We also would like to thank the following (inter)national patient organizations that provided help with patient recruitment: the Dutch HEVAS Association, the Dutch Wijnvlek-Sturge Weber Association, the VASCAPA Association, the Sturge Weber Foundation UK and USA, the Sturge Weber Association Germany, Spain and Italy, and the Birthmark Support Group UK. We also owe our gratitude to the patients who provided feedback on the first Delphi survey and participated in the focus group sessions. Lastly, many thanks to the CS-COUSIN methods working group (Kim Thomas, Jochen Schmitt, Jamie Kirkham, Toni Lange, Murad Alam, Christian Apfelbacher, Joanne Chalmers, Jan Kottner and Phyllis Spuls), Anne Fledderus for providing methodological support, and Timothy van Asbeck for his linguistic advice.

PY - 2022

Y1 - 2022

N2 - 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. What is already known about this topic? Besides physical and functional sequelae, capillary malformations (CMs) often cause emotional and social burden. The lack of uniform outcome measures obstructs proper evaluation and comparison of treatment strategies. As a result, there is limited evidence on the best available treatment options. The development of a core outcome set (COS) may improve standardized reporting of trial outcomes. What does this study add? A core outcome domain set (CDS), as part of a COS, was developed for clinical research on CMs. International consensus was reached on the recommended core outcome subdomains to be measured in CM trials: 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. This CDS enables the next step in the development of a COS, namely to reach consensus on the core outcome measurement instruments to score the core outcome subdomains. What are the clinical implications of this work? The obtained CDS will facilitate standardized reporting of treatment outcomes, thereby enabling proper comparison of treatment results. This comparison is likely to provide more reliable information for patients about the best available treatment options.

AB - 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. What is already known about this topic? Besides physical and functional sequelae, capillary malformations (CMs) often cause emotional and social burden. The lack of uniform outcome measures obstructs proper evaluation and comparison of treatment strategies. As a result, there is limited evidence on the best available treatment options. The development of a core outcome set (COS) may improve standardized reporting of trial outcomes. What does this study add? A core outcome domain set (CDS), as part of a COS, was developed for clinical research on CMs. International consensus was reached on the recommended core outcome subdomains to be measured in CM trials: 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. This CDS enables the next step in the development of a COS, namely to reach consensus on the core outcome measurement instruments to score the core outcome subdomains. What are the clinical implications of this work? The obtained CDS will facilitate standardized reporting of treatment outcomes, thereby enabling proper comparison of treatment results. This comparison is likely to provide more reliable information for patients about the best available treatment options.

U2 - 10.1111/bjd.21723

DO - 10.1111/bjd.21723

M3 - Journal article

C2 - 35762296

AN - SCOPUS:85135196558

VL - 187

SP - 730

EP - 742

JO - British Journal of Dermatology

JF - British Journal of Dermatology

SN - 0007-0963

IS - 5

ER -