Moriarty James P, Borah Bijan J, Foote Robert L, Pulido Jose S, Shah Nilay D
Robert D. and Patricia E. Kern Center for the Science of Health Care Delivery, Mayo Clinic, Rochester, Minnesota, United States of America.
Robert D. and Patricia E. Kern Center for the Science of Health Care Delivery, Mayo Clinic, Rochester, Minnesota, United States of America; Division of Health Care Policy and Research, Mayo Clinic, Rochester, Minnesota, United States of America.
PLoS One. 2015 May 18;10(5):e0127814. doi: 10.1371/journal.pone.0127814. eCollection 2015.
Proton beam therapy is a commonly accepted treatment for intraocular melanomas, but the literature is lacking in descriptions of patient preferences of clinical outcomes and economic impact. In addition, no economic evaluations have been published regarding the incremental cost-effectiveness of proton beam therapy compared with enucleation or plaque brachytherapy, typical alternative treatments. We, therefore, conducted a cost-utility analysis of these three approaches for the treatment of intraocular melanomas.
A Markov model was constructed. Model parameters were identified from the published literature and publicly available data sources. Cost-effectiveness of each treatment was calculated in 2011 US Dollars per quality-adjusted life-year. Incremental cost-effectiveness ratios were calculated assuming enucleation as reference. One-way sensitivity analyses were conducted on all model parameters. A decision threshold of $50,000/quality-adjusted life-year was used to determine cost-effectiveness.
Enucleation had the lowest costs and quality-adjusted life-years, and plaque brachytherapy had the highest costs and quality-adjusted life-years. Compared with enucleation, the base-case incremental cost-effectiveness ratios for plaque brachytherapy and proton beam therapy were $77,500/quality-adjusted life-year and $106,100/quality-adjusted life-year, respectively. Results were highly sensitive to multiple parameters. All three treatments were considered optimal, and even dominant, depending on the values used for sensitive parameters.
Base-case analysis results suggest enucleation to be optimal. However, the optimal choice was not robust to sensitivity analyses and, depending on the assumption, both plaque brachytherapy and proton beam therapy could be considered cost-effective. Future clinical studies should focus on generating further evidence with the greatest parameter uncertainty to inform future cost-effectiveness analyses.
质子束疗法是眼内黑色素瘤普遍认可的一种治疗方法，但文献中缺乏对患者对临床结局偏好及经济影响的描述。此外，与典型的替代治疗方法眼球摘除术或敷贴近距离放疗相比，尚未有关于质子束疗法增量成本效益的经济评估发表。因此，我们对这三种眼内黑色素瘤治疗方法进行了成本效用分析。
构建了一个马尔可夫模型。模型参数从已发表的文献和公开可用的数据来源中确定。每种治疗方法的成本效益以每质量调整生命年的2011年美元计算。假设以眼球摘除术为参照计算增量成本效益比。对所有模型参数进行了单因素敏感性分析。使用每质量调整生命年50,000美元的决策阈值来确定成本效益。
眼球摘除术成本和质量调整生命年最低，敷贴近距离放疗成本和质量调整生命年最高。与眼球摘除术相比，敷贴近距离放疗和质子束疗法的基础病例增量成本效益比分别为每质量调整生命年77,500美元和每质量调整生命年106,100美元。结果对多个参数高度敏感。根据敏感参数所采用的值，所有三种治疗方法都被认为是最优的，甚至是占优的。
基础病例分析结果表明眼球摘除术是最优的。然而，最优选择对敏感性分析并不稳健，根据假设，敷贴近距离放疗和质子束疗法都可被认为具有成本效益。未来的临床研究应聚焦于在最大参数不确定性的情况下生成更多证据，以为未来的成本效益分析提供信息。
