英夫利昔单抗与环孢素治疗激素耐药性急性重度溃疡性结肠炎：来自 CONSTRUCT 实用试验数据的基于模型的成本-效用分析。
Infliximab versus ciclosporin in steroid resistant acute severe ulcerative colitis: a model-based cost-utility analysis of data from CONSTRUCT pragmatic trial.
机构信息
Department of Public Health, College of Health Sciences, QU-Health, Qatar University, PO Box 2713, Doha, Qatar.
Marie Curie Palliative Care Research Centre, Cardiff University, 8th Floor Neuadd Meirionnydd, Heath Park Way, Cardiff, CF14 4YS, UK.
出版信息
BMC Health Serv Res. 2023 Mar 8;23(1):226. doi: 10.1186/s12913-023-09233-w.
BACKGROUND
There is limited evidence in the literature on the long-term effectiveness and cost-effectiveness of treatments for Acute Severe Ulcerative Colitis (ASUC). The study aimed to perform decision analytic model-based long-term cost-utility analysis (CUA) of infliximab versus ciclosporin for steroid-resistant ASUC investigated in CONSTRUCT pragmatic trial.
METHODS
A decision tree (DT) model was developed using two-year health effect, resource use and costs data from CONSTRUCT trial to estimate relative cost-effectiveness of two competing drugs from the United Kingdom (UK) National Health Services (NHS) perspective. Using short-term trial data, a Markov model (MM) was then developed and evaluated over further 18 years. Both DT and MM were combined to investigate cost-effectiveness of infliximab versus ciclosporin for ASUC patients over 20-year time horizon, with a rigorous multiple deterministic and probabilistic sensitivity analyses to address uncertainty in results.
RESULTS
The decision tree mirrored trial-based results. Beyond 2-year trial follow-up, Markov model predicted a decrease in colectomy rate, but it remained slightly higher for ciclosporin. NHS costs and quality adjusted life years (QALYs) over base-case 20 year time horizon were £26,793 and 9.816 for ciclosporin and £34,185 and 9.106 for infliximab, suggesting ciclosporin dominates infliximab. Ciclosporin had 95% probability of being cost-effective at a willingness-to-pay (WTP) threshold value up to £20,000.
CONCLUSION
Using data from a pragmatic RCT, the cost-effectiveness models produced incremental net health benefit in favour of ciclosporin relative to infliximab. Results from long-term modelling indicated that ciclosporin remains dominant compared with infliximab for the treatment of NHS ASUC patients, however, these need to be interpreted cautiously.
TRIAL REGISTRATION
CONSTRUCT Trial registration number ISRCTN22663589; EudraCT number: 2008- 001968-36 (Date 27/08/2008).
背景
在急性重度溃疡性结肠炎（ASUC）的治疗长期有效性和成本效益方面，文献中的证据有限。本研究旨在对 CONSTRUCT 实用试验中研究的英夫利昔单抗与环孢素治疗类固醇耐药性 ASUC 进行基于决策分析模型的长期成本效用分析（CUA）。
方法
使用 CONSTRUCT 试验的两年健康效果、资源利用和成本数据，为英国国家医疗服务体系（NHS）开发决策树（DT）模型，以估计两种竞争药物的相对成本效益。然后，使用短期试验数据，开发并评估了进一步 18 年的马尔可夫模型（MM）。将 DT 和 MM 结合起来，在 20 年的时间范围内调查英夫利昔单抗与环孢素治疗 ASUC 患者的成本效益，通过严格的多次确定性和概率敏感性分析来解决结果中的不确定性。
结果
决策树反映了试验结果。在 2 年试验随访期之后，马尔可夫模型预测结肠切除术的比率下降，但环孢素的比率仍然略高。在基本案例 20 年时间范围内，NHS 成本和质量调整生命年（QALYs）分别为环孢素 26793 英镑和 9.816，英夫利昔单抗 34185 英镑和 9.106，表明环孢素优于英夫利昔单抗。在 20000 英镑的意愿支付（WTP）阈值下，环孢素 95%的概率具有成本效益。
结论
使用来自实用 RCT 的数据，成本效益模型产生了有利于环孢素相对于英夫利昔单抗的增量净健康效益。长期建模的结果表明，与英夫利昔单抗相比，环孢素在治疗 NHS ASUC 患者方面仍然具有优势，但需要谨慎解释。
试验注册
CONSTRUCT 试验注册号 ISRCTN22663589；EudraCT 编号：2008-001968-36（2008 年 8 月 27 日）。
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