Department of Surgery, Amsterdam University Medical Centers, location AMC, University of Amsterdam, Amsterdam, the Netherlands.
Department of Plastic, Reconstructive and Hand Surgery, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, the Netherlands.
Br J Dermatol. 2022 Nov;187(5):730-742. doi: 10.1111/bjd.21723. Epub 2022 Jul 31.
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.
To develop a core outcome domain set (CDS), as part of a core outcome set (COS), for clinical research on CMs.
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.
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.
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.
由于临床试验中缺乏统一的疗效评估指标，有关毛细血管畸形（CMs）最佳治疗方案的证据十分有限。核心结局集（COS）可以标准化报告临床试验结果，便于比较不同治疗方案的疗效。
制定毛细血管畸形临床研究的核心结局域集（CDS），作为核心结局集（COS）的一部分。
根据文献、焦点小组会议以及 COSCAM 工作组的意见，确定了 67 个潜在相关结局子域。这些结局子域在一项在线德尔菲研究中呈现给 CMs 专家（医学专家和相关文献的作者）和（CM）患者（国际患者协会）。在三轮电子德尔菲研究中，参与者反复对这些结局子域的重要性进行了七分制评分。参与者还可以提出其他相关结局子域。共识定义为两组利益相关者均对结局子域的重要性达成≥80%的一致意见。在一次在线共识会议上最终确定了 CDS。
共有来自 45 个国家的 269 名参与者参加了第一轮电子德尔菲研究。其中，106 名是来自 32 个国家的 CMs 专家，主要由皮肤科医生（59%）和整形外科医生（18%）组成。此外，来自 28 个国家的 163 名（CM）患者或其家属参加了研究，其中 58%患有 Sturge-Weber 综合征。在随后的两轮电子德尔菲研究中，分别有 189 名和 148 名参与者参加。经过整个共识过程，11 个结局子域达成了共识：颜色/红色、厚度、可见度、解剖结构变形、青光眼、整体健康相关生活质量、情绪功能、社会功能、干预的耐受性、患者对治疗结果的满意度和复发。
我们建议在未来所有 CMs 治疗的临床试验中使用该 CDS 作为最小报告标准。下一步将选择合适的结局测量工具来评分核心结局子域。
关于这个主题，已经知道了什么？除了身体和功能后遗症外，毛细血管畸形（CMs）常引起情绪和社会负担。缺乏统一的结局指标阻碍了对治疗策略的正确评估和比较。因此，关于最佳治疗方案的证据有限。核心结局集（COS）的制定可能会改善临床试验结果的标准化报告。
本研究增加了什么？制定了毛细血管畸形临床研究的核心结局域集（CDS），作为 COS 的一部分。国际共识达成了推荐的在 CM 临床试验中测量的核心结局子域：颜色/红色、厚度、可见度、解剖结构变形、青光眼、整体健康相关生活质量、情绪功能、社会功能、干预的耐受性、患者对治疗结果的满意度和复发。该 CDS 为下一步开发 COS 奠定了基础，即就评分核心结局子域的核心结局测量工具达成共识。
这对临床有何影响？所获得的 CDS 将促进治疗结果的标准化报告，从而便于比较不同治疗方案的疗效。这种比较可能为患者提供有关最佳治疗方案的更可靠信息。
