克罗恩病是一种终生的疾病,可以影响胃肠道的任何部分。一些患有克罗恩病的人可能比其他人有更高的严重病程风险,并且能够确定患者的风险水平可能导致个性化管理。
为了评估预后测试的准确性,根据严重病程后的风险对诊断为克罗恩病的患者进行分层的两种工具的临床影响和成本效益。
数据源MEDLINE,EMBASE,搜索了Cochrane系统评价数据库和Cochrane对照试验中央登记册,以告知有关预后准确性的系统评价,预后工具的临床影响,和经济评估。为经济评估审查提供信息的其他数据来源是NHS经济评估数据库,效果评论摘要数据库和卫生技术评估数据库。
从开始到2019年6月,对电子数据库进行了系统评价,以评估IBDX®的预后准确性和临床影响(克罗恩病预后测试;GlycomindsLtd,Lod,以色列)生物标志物分层工具和PredictSURE-IBD™(PredictImmuneLtd,剑桥,英国)工具。报告克罗恩病治疗成本效益的研究的系统评价从开始到2019年7月。两名审稿人独立同意纳入研究,评估纳入研究的质量,并验证从研究中提取的数据。研究中的临床和方法学异质性排除了预后准确性数据的综合。开发了一种从头经济模型,以比较两种治疗方法的成本和后果-“自上而下”和“逐步”策略,在接受克罗恩病严重病程的高风险人群中,加强标准护理。该模型包括决策树和马尔可夫队列模型。
16种出版物,包括8项原始研究(n=1478),被认为与预后准确性的审查有关。还审查了营销预后工具的公司提供的文件。无符合IBDX生物标志物分层工具敏感性或特异性合格标准的研究报告,而一项研究提供了敏感性的估计,PredictSURE-IBD工具的特异性和阴性预测值。所有确定的研究都是观察性的,被认为对工具的有效性提供了微弱的证据。由于这两种工具的数据很少,在基本病例分析中,假设PredictSURE-IBD的准确度为100%.在情景分析中,IBDX的准确性假设为100%,测试的成本是分析之间的唯一区别。对成本效益的增量分析表明,自上而下(通过在模型中使用PredictSURE-IBD)比提高(通过模型的标准护理部门)成本更高,并且产生的质量调整生命年数更少。
尽管对文献进行了广泛的系统搜索,对于生物标志物分层工具IBDX和PredictSURE-IBD的预后准确性,未发现确凿的证据.
尽管模型表明标准护理在测试中占主导地位,缺乏两种检测预后准确性的证据,以及自上而下和阶梯式治疗方法获益的不确定性,意味着这些结果应被解释为指示性的,而不是确定性的.
本研究注册为PROSPEROCRD42019138737。
该项目由美国国家卫生研究所(NIHR)证据综合计划资助,并将在《卫生技术评估》(第一卷)中全文发表。25号23.有关更多项目信息,请参阅NIHR期刊库网站。
克罗恩病是消化系统的一部分发炎(肿胀)的病症。任何年龄的人都可能患上克罗恩病。这是一种终生无法治愈的疾病。在英国,克罗恩病影响约1每650人。消化系统的任何部分都可能受到影响,疾病的严重程度因人而异。症状来来去去,有时根本没有症状。克罗恩病的常见症状是腹泻,胃痛和粪便中的血。给予治疗以减轻或控制症状,并试图阻止炎症复发。一些患有克罗恩病的人比其他人更有可能有更多的复发,并发展可能需要手术的克罗恩病并发症。该项目研究了两种工具在识别可能出现并发症或需要手术的克罗恩病患者方面的效果。识别那些有更高的机会经历克罗恩病并发症的人可以帮助他们和他们的医生选择他们的治疗方法,目的是减少复发次数和长期手术风险。此外,审查评估了这些工具是否物有所值。我们发现有限的证据表明,这些工具在识别更有可能发生克罗恩病并发症的人方面有多好。缺乏有关工具的证据意味着成本效益分析只能评估此时在临床实践中给予的治疗或更有可能发生并发症的人的更强化治疗的资金价值。分析发现,对于发生克罗恩病并发症的机会较高的人,当前的标准护理比强化治疗提供了更多的金钱价值。
Crohn\'s disease is a lifelong condition that can affect any segment of the gastrointestinal tract. Some people with Crohn\'s disease may be at higher risk of following a severe course of disease than others and being able to identify the level of risk a patient has could lead to personalised management.
To assess the prognostic test accuracy, clinical impact and cost-effectiveness of two tools for the stratification of people with a diagnosis of Crohn\'s disease by risk of following a severe course of disease.
The data sources MEDLINE, EMBASE, the Cochrane Database of Systematic Reviews and the Cochrane Central Register of Controlled Trials were searched to inform the systematic reviews on prognostic accuracy, clinical impact of the prognostic tools, and economic evaluations. Additional data sources to inform the
review of economic evaluations were NHS Economic Evaluation Database, Database of Abstracts of Reviews of Effects and the Health Technology Assessment Database.
Systematic reviews of electronic databases were carried out from inception to June 2019 for studies assessing the prognostic accuracy and clinical impact of the IBDX® (Crohn\'s disease Prognosis Test; Glycominds Ltd, Lod, Israel) biomarker stratification tool and the PredictSURE-IBD™ (PredictImmune Ltd, Cambridge, UK) tool. Systematic reviews of studies reporting on the cost-effectiveness of treatments for Crohn\'s disease were run from inception to July 2019. Two reviewers independently agreed on studies for inclusion, assessed the quality of included studies and validated the data extracted from studies. Clinical and methodological heterogeneity across studies precluded the synthesis of data for prognostic accuracy. A de novo economic model was developed to compare the costs and consequences of two treatment approaches - the \'top-down\' and \'step-up\' strategies, with step-up considered standard care - in people at high risk of following a severe course of Crohn\'s disease. The model comprised a decision tree and a Markov cohort model.
Sixteen publications, including eight original studies (n = 1478), were deemed relevant to the
review of prognostic accuracy. Documents supplied by the companies marketing the prognostic tools were also reviewed. No study meeting the eligibility criteria reported on the sensitivity or specificity of the IBDX biomarker stratification tool, whereas one study provided estimates of sensitivity, specificity and negative predictive value for the PredictSURE-IBD tool. All identified studies were observational and were considered to provide weak evidence on the effectiveness of the tools. Owing to the paucity of data on the two tools, in the base-case analysis the accuracy of PredictSURE-IBD was assumed to be 100%. Accuracy of IBDX was assumed to be 100% in a scenario analysis, with the cost of the tests being the only difference between the analyses. The incremental analysis of cost-effectiveness demonstrated that top-down (via the use of PredictSURE-IBD in the model) is more expensive and generates fewer quality-adjusted life-years than step-up (via the standard care arm of the model).
Despite extensive systematic searches of the literature, no robust evidence was identified of the prognostic accuracy of the biomarker stratification tools IBDX and PredictSURE-IBD.
Although the model indicates that standard care dominates the tests, the lack of evidence of prognostic accuracy of the two tests and the uncertainty around the benefits of the top-down and step-up treatment approaches mean that the results should be interpreted as indicative rather than definitive.
This study is registered as PROSPERO CRD42019138737.
This project was funded by the National Institute for Health Research (NIHR) Evidence Synthesis programme and will be published in full in Health Technology Assessment; Vol. 25, No. 23. See the NIHR Journals Library website for further project information.
Crohn’s disease is a condition in which parts of the digestive system become inflamed (swollen). People of any age can develop Crohn’s disease. It is a lifelong condition for which there is no cure. In the UK, Crohn’s disease affects about 1 in every 650 people. Any part of the digestive system can be affected, and the severity of the disease can vary from person to person. Symptoms come and go, and there can be times when there are no symptoms at all. Common symptoms of Crohn’s disease are diarrhoea, stomach-ache and blood in faeces. Treatment is given to reduce or control symptoms and to try to stop inflammation from coming back. Some people with Crohn’s disease are more likely than others to have more relapses and to develop complications of Crohn’s disease that might require surgery. This project looked at how well two tools worked at identifying people with Crohn’s disease who might develop complications or need surgery. Identifying those who have a higher chance of experiencing complications of Crohn’s disease could help them and their doctor to choose their treatment, with the goal of reducing the number of relapses and the risk of surgery in the longer term. In addition, the
review assessed whether or not the tools offered value for money. We found limited evidence of how well the tools worked in identifying people who were more likely to develop complications of Crohn’s disease. The lack of evidence on the tools meant that the cost-effectiveness analysis could only assess the value for money of the treatment that is given in clinical practice at this time or of more intensive treatments for people who are more likely to develop complications. The analysis found that current standard care offers more value for money than intensive treatments for people with a higher chance of developing complications of Crohn’s disease.