Decision Analytic Modeling for Global Clinical Trial Planning: A Case for HIV-Positive Patients at High Risk for Mycobacterium tuberculosis Sepsis in Uganda.
Decision Analytic Modeling for Global Clinical Trial Planning: A Case for HIV-Positive Patients at High Risk for Mycobacterium tuberculosis Sepsis in Uganda.
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DOI：
10.3390/ijerph20065041
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发表时间：
2023-03-13
影响因子：
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通讯作者：
Moore, Christopher C
中科院分区：
文献类型：
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作者：
Keim-Malpass, Jessica;Heysell, Scott K;Thomas, Tania A;Lobo, Jennifer M;Mpagama, Stellah G;Muzoora, Conrad;Moore, Christopher C
脓毒症是撒哈拉以南非洲人类免疫缺陷病毒（HIV）感染者死亡的一个重要原因。在一项大型多国临床试验开始前的规划阶段，研究在标准护理抗生素基础上立即添加抗结核治疗对艾滋病毒感染者败血症的疗效，我们使用决策分析来评估临床试验设计的成本和潜在的健康结局影响，该临床试验设计基于初步数据和流行病学参数估计。本分析的目的是强调这种方法作为一个案例，决策分析可以估计拟议临床试验设计的成本效益。在这种情况下，我们使用三种不同的结核病诊断方法，即尿液TB-LAM，痰expert - mtb /RIF，以及LAM/Xpert的组合，估计了即时经验性抗结核（TB）治疗与诊断依赖标准治疗的影响。我们构建了决策分析模型，比较了三种诊断方法的两种治疗策略。与所有三种依赖诊断的标准治疗模式相比，即时经验治疗显示出良好的成本效益。在我们的方法学案例范例中，建议的随机临床试验干预在决策模拟框架中显示出最有利的结果。应用决策分析和经济评价的原则可以对研究设计和临床试验计划产生重大影响。
Sepsis is a significant cause of mortality among people living with human immunodeficiency virus (HIV) in sub-Saharan Africa. In the planning period prior to the start of a large multi-country clinical trial studying the efficacy of the immediate empiric addition of anti-tuberculosis therapy to standard-of-care antibiotics for sepsis in people living with HIV, we used decision analysis to assess the costs and potential health outcome impacts of the clinical trial design based on preliminary data and epidemiological parameter estimates. The purpose of this analysis was to highlight this approach as a case example where decision analysis can estimate the cost effectiveness of a proposed clinical trial design. In this case, we estimated the impact of immediate empiric anti-tuberculosis (TB) therapy versus the diagnosis-dependent standard of care using three different TB diagnostics: urine TB-LAM, sputum Xpert-MTB/RIF, and the combination of LAM/Xpert. We constructed decision analytic models comparing the two treatment strategies for each of the three diagnostic approaches. Immediate empiric-therapy demonstrated favorable cost-effectiveness compared with all three diagnosis-dependent standard of care models. In our methodological case exemplar, the proposed randomized clinical trial intervention demonstrated the most favorable outcome within this decision simulation framework. Applying the principles of decision analysis and economic evaluation can have significant impacts on study design and clinical trial planning.