Roth Joshua A, Carlson Josh J
Department of Pharmacy, University of Washington, Seattle, WA 98195, USA.
J Gastrointest Cancer. 2012 Jun;43(2):215-23. doi: 10.1007/s12029-010-9242-0.
The ABC-02 trial demonstrated a statistically significant survival benefit associated with the addition of cisplatin to gemcitabine in the palliative treatment of advanced biliary tract cancer (BTC). Based on the ABC-02 findings, this analysis seeks to evaluate the cost-utility of adding cisplatin to standard gemcitabine therapy from a U.S. societal perspective.
A decision-analytic model was developed to estimate direct medical costs, patient time costs, and quality-adjusted life-years (QALYs) for two treatment strategies: (1) gemcitabine + cisplatin, (2) gemcitabine monotherapy. Model parameters were derived from the pivotal trial of gemcitabine + cisplatin in advanced BTC, published literature, and government sources. The model included trial-based adverse events and costs related to drug treatment, routine follow-up, adverse events, and post-progression care. The model results were examined using one-way and probabilistic sensitivity analyses (PSA).
Total QALYs for the gemcitabine + cisplatin and gemcitabine monotherapy strategies were 0.751 and 0.561, respectively. Total costs were $44,885 and $33,653 respectively. Relative to gemcitabine monotherapy, gemcitabine + cisplatin had an incremental cost-effectiveness ratio (ICER) of $59,480 per QALY gained. One-way sensitivity analyses found results to be sensitive to progression-free survival, overall survival, pre and post-progression health state utility values, and the cost of post-progression care. In the PSA, gemcitabine monotherapy had the highest probability of being cost-effective until a willingness-to-pay of $60,000, after which the gemcitabine + cisplatin strategy had the highest probability.
The results of this analysis suggest that in advanced BTC, the cisplatin + gemcitabine regimen is a cost-effective treatment alternative to gemcitabine monotherapy by currently accepted standards of willingness to pay.
ABC - 02试验表明，在晚期胆管癌（BTC）的姑息治疗中，吉西他滨联合顺铂治疗具有统计学上显著的生存获益。基于ABC - 02的研究结果，本分析旨在从美国社会角度评估在标准吉西他滨治疗中添加顺铂的成本效益。
建立了一个决策分析模型，以估计两种治疗策略的直接医疗成本、患者时间成本和质量调整生命年（QALY）：（1）吉西他滨 + 顺铂，（2）吉西他滨单药治疗。模型参数来源于吉西他滨 + 顺铂治疗晚期BTC的关键试验、已发表文献和政府资料。该模型包括基于试验的不良事件以及与药物治疗、常规随访、不良事件和疾病进展后护理相关的成本。使用单因素和概率敏感性分析（PSA）对模型结果进行检验。
吉西他滨 + 顺铂和吉西他滨单药治疗策略的总QALY分别为0.751和0.561。总成本分别为44,885美元和33,653美元。相对于吉西他滨单药治疗，吉西他滨 + 顺铂每获得一个QALY的增量成本效益比（ICER）为59,480美元。单因素敏感性分析发现，结果对无进展生存期、总生存期、疾病进展前后的健康状态效用值以及疾病进展后护理成本敏感。在PSA中，在支付意愿达到60,000美元之前，吉西他滨单药治疗具有成本效益的概率最高，之后吉西他滨 + 顺铂策略具有最高概率。
本分析结果表明，在晚期BTC中，按照目前公认的支付意愿标准，顺铂 + 吉西他滨方案是吉西他滨单药治疗的一种具有成本效益的替代治疗方案。
