开发一个Netherton综合征的核心成果集:一个国际多利益相关者e-Delphi共识研究研究
Anouk E M Nouwen1, Aviël Ragamin1, Maria J Knol2
1Department of Dermatology, Erasmus University Medical Center, Rotterdam, The Netherlands.
概括
尼瑟顿综合征 (NS) 患者需要一种标准化的方法来衡量治疗成功. 这项研究开发了10个领域的21个结果的核心结果集 (COS),以改善NS的临床护理和研究.
科学领域:
- 皮肤病学和遗传学
- 罕见疾病研究 罕见疾病研究
- 临床试验方法论 临床试验方法论
背景情况:
- 尼瑟顿综合征 (NS) 是一种严重的遗传疾病,由于SPINK5基因变异,影响皮肤成熟.
- NS会导致显著的皮肤屏障损伤,需要有效的治疗策略.
- 目前的治疗结果报告缺乏标准化,阻碍了指导方针的制定.
研究的目的:
- 建立一个基于共识的Netherton综合征的核心结果集 (COS).
- 在NS临床护理和研究中标准化结果测量.
- 促进对NS的基于证据的治疗指南的制定.
主要方法:
- 在文献中搜索并对潜在结果进行分类.
- 专家小组讨论在第二届国际尼瑟顿大会.
- 与41个国际利益相关者进行了两轮的e-Delphi调查.
- 在线共识会议以最终确定COS.
主要成果:
- 建立了一个核心成果集 (COS),包括10个领域的21个成果.
- 领域包括皮肤,感觉,治疗副作用,活力,情绪功能,身体发展,营养,感染,过敏和评估结果.
- COS反映了超越皮肤表现的NS的多方面的影响.
结论:
- 对21个结果的共识为NS研究和临床实践提供了标准化的框架.
- COS强调需要采用多学科方法来管理NS.
- 实施COS将使研究结果可以比较,并指导未来的治疗建议.
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