Rochau Ursula, Schwarzer Ruth, Jahn Beate, Sroczynski Gaby, Kluibenschaedl Martina, Wolf Dominik, Radich Jerald, Brixner Diana, Gastl Guenther, Siebert Uwe
Division of Public Health Decision Modelling, Health Technology Assessment and Health Economics, ONCOTYROL-Center for Personalized Cancer Medicine, Innsbruck, Austria,
Appl Health Econ Health Policy. 2014 Apr;12(2):103-15. doi: 10.1007/s40258-013-0071-8.
Several tyrosine kinase inhibitors (TKIs) are approved for the treatment of chronic myeloid leukemia (CML). Decision-analytic modeling can help to extrapolate data from short-term clinical trials and also consider quality of life when evaluating different treatment strategies.
Our goal was to describe and analyze the structural and methodological approaches of published decision-analytic models for various treatment strategies in CML and to derive recommendations for the development of future CML models.
We performed a systematic literature search in electronic databases (MEDLINE/PreMEDLINE, EconLit, EMBASE, NHS EED, and Tuft's CEA Registry) to identify published studies evaluating CML treatment strategies using mathematical models. The search was updated in August 2013.
The models were required to compare different treatment strategies in relation to relevant clinical and patient-relevant health outcomes [e.g., life-years gained, quality-adjusted life-years] over a defined time horizon and population.
We used standardized forms for data extraction, description of study design, methodological framework, and data sources for each model.
We identified 18 different decision-analytic modeling studies. Of these, 17 included economic evaluations. Modeling approaches included decision trees, Markov cohort models, state-transition models with individual (Monte Carlo) simulations, and mathematical equations. Analytic time horizons ranged from 2 years to a lifetime. Treatment strategies compared included bone marrow or stem cell transplantation, conventional chemotherapy, interferon-α, and TKIs. Only one model evaluated a second-generation TKI. Most models did not report a model validation. All models conducted deterministic sensitivity analyses and four reported a probabilistic sensitivity analysis.
Articles that were not published in English or German were not included in this review. Our literature search was restricted to published full-text articles in certain databases. Therefore, publications that met our inclusion criteria but were published in different databases, different languages, or as abstracts only may have been missed.
While several well-designed models of CML treatment strategies exist, there remains a need for the assessment of the long-term efficacy and cost effectiveness of novel treatment options such as second-generation TKIs. Additionally, these models should be validated using independent data.
几种酪氨酸激酶抑制剂（TKIs）已被批准用于治疗慢性粒细胞白血病（CML）。决策分析模型有助于从短期临床试验中推断数据，并在评估不同治疗策略时考虑生活质量。
我们的目标是描述和分析已发表的关于CML各种治疗策略的决策分析模型的结构和方法，并为未来CML模型的开发提出建议。
我们在电子数据库（MEDLINE/PreMEDLINE、EconLit、EMBASE、NHS EED和塔夫茨成本效果分析注册库）中进行了系统的文献检索，以识别使用数学模型评估CML治疗策略的已发表研究。检索于2013年8月更新。
要求模型在规定的时间范围和人群内，比较不同治疗策略在相关临床和患者相关健康结局[如获得的生命年、质量调整生命年]方面的差异。
我们使用标准化表格对每个模型的数据提取、研究设计描述、方法框架和数据来源进行整理。
我们识别出18项不同的决策分析模型研究。其中，17项包括经济评估。建模方法包括决策树、马尔可夫队列模型、带有个体（蒙特卡洛）模拟的状态转换模型和数学方程。分析时间范围从2年到终身。比较的治疗策略包括骨髓或干细胞移植、传统化疗、α干扰素和TKIs。只有一个模型评估了第二代TKI。大多数模型未报告模型验证情况。所有模型都进行了确定性敏感性分析，四项报告了概率敏感性分析。
未发表在英文或德文的文章未纳入本综述。我们的文献检索仅限于某些数据库中已发表的全文文章。因此，可能遗漏了符合我们纳入标准但发表在不同数据库、不同语言或仅作为摘要发表的文献。
虽然存在几种设计良好的CML治疗策略模型，但仍需要评估新型治疗方案（如第二代TKIs）的长期疗效和成本效益。此外，这些模型应使用独立数据进行验证。
