背景:原发性硬化性胆管炎(PSC)是一种进行性免疫介导的肝病,没有药物治疗可以减缓疾病进展。然而,新疗法的前景令人鼓舞,几项创新的临床试验正在进行中。尽管取得了这些进步,研究的结果存在相当大的异质性,对于衡量什么结果缺乏共识,何时测量以及如何测量。此外,近年来,PSC治疗目标发生了范式转变,从基于生物化学的终点转移到肝纤维化的组织学评估,基于影像学的生物标志物和患者报告的结局指标。大量新的介入试验和不断发展的终点为参与评估新疗法的所有利益相关者提供了机会。为此,有必要通过开发核心结果集(COS)来协调临床试验中使用的措施.
方法:PSC特异性COS的合成将分四个阶段进行。最初,将进行系统的文献综述,以确定以前在PSC试验中使用的结果,其次是与关键利益相关者进行的半结构化定性访谈。后者可能包括患者,临床医生,研究人员,制药行业代表、医疗保健支付者和监管机构,确定更多重要的结果。使用文献综述和利益相关者访谈产生的结果,将进行国际两轮Delphi调查,以优先考虑纳入COS的结果。最后,将召开一次共识会议,批准COS,并传播研究结果,供未来PSC试验应用.
背景:本研究已获得东米德兰兹-莱斯特中央研究伦理委员会(Ref:24/EM/0126)的伦理批准。这项研究的COS将广泛传播,包括在同行评审的期刊上发表。国际会议,通过患者支持小组进行推广,并在有效性试验的核心结果衡量(COMET)数据库中提供。
背景:1239。
BACKGROUND: Primary sclerosing cholangitis (PSC) is a progressive immune-mediated liver disease, for which no medical therapy has been shown to slow disease progression. However, the horizon for new therapies is encouraging, with several innovative clinical trials in progress. Despite these advancements, there is considerable heterogeneity in the outcomes studied, with lack of consensus as to what outcomes to measure, when to measure and how to measure. Furthermore, there has been a paradigm shift in PSC treatment targets over recent years, moving from biochemistry-based endpoints to histological assessment of liver fibrosis, imaging-based biomarkers and patient-reported outcome measures. The abundance of new interventional trials and evolving endpoints pose opportunities for all stakeholders involved in evaluating novel therapies. To this effect, there is a need to harmonise measures used in clinical trials through the development of a core outcome set (COS).
METHODS: Synthesis of a PSC-specific COS will be conducted in four stages. Initially, a systematic literature review will be performed to identify outcomes previously used in PSC trials, followed by semistructured qualitative interviews conducted with key stakeholders. The latter may include patients, clinicians, researchers, pharmaceutical industry representatives and healthcare payers and regulatory agencies, to identify additional outcomes of importance. Using the outcomes generated from the literature review and stakeholder interviews, an international two-round Delphi survey will be conducted to prioritise outcomes for inclusion in the COS. Finally, a consensus meeting will be convened to ratify the COS and disseminate findings for application in future PSC trials.
BACKGROUND: Ethical approval has been granted by the East Midlands-Leicester Central Research Ethics Committee (Ref: 24/EM/0126) for this study. The COS from this study will be widely disseminated including publication in peer-reviewed journals, international conferences, promotion through patient-support groups and made available on the Core Outcomes Measurement in Effectiveness Trials (COMET) database.
BACKGROUND: 1239.