Gustave Roussy, Université Paris-Saclay, Service de Biostatistique et d'Epidémiologie, Villejuif, France; Université Paris-Saclay, Paris-Sud University, Villejuif, France.
Department of Medical Oncology, Centre Oscar Lambret, Lille, France.
Value Health. 2021 May;24(5):676-682. doi: 10.1016/j.jval.2020.11.017. Epub 2021 Mar 26.
Continuous chemotherapy has been used to treat patients with metastatic esophageal squamous cell carcinoma (mESCC), despite weak evidence supporting a clinical benefit, associated side effects for the patients, and unjustified medical costs. In the French setting, we conducted a cost-utility analysis alongside the randomized E-DIS trial (NCT01248299), which compared first-line fluorouracil/platinum-based chemotherapy continuation (CT-CONT) to CT discontinuation (CT-DISC) in progressive-free patients after an initial 6-week treatment phase.
A partitioned survival analysis was performed using patient-level data collected during the trial for survival outcomes, quality of life (EQ-5D-3L), and medical costs. The mean quality-adjusted life-years (QALYs) and medical costs were estimated over an 18-month period to assess the incremental net monetary benefit and incremental cost-effectiveness ratio. Uncertainty was handled using the nonparametric bootstrap and univariate analysis. Sixty-seven patients with mESCC were randomized and included in the cost-utility analysis.
On average, CT-CONT slightly decreased the number of QALYs (-0.038) and increased the cost per patient (+ €1177). At a willingness-to-pay threshold of €50 000/QALY, the incremental net monetary benefit was negative (-€3077 [95% confidence interval: -6564; 4359]), and the incremental cost-effectiveness ratio was -30 958€/QALY (CT-CONT dominated). The probability of the CT-CONT treatment option being cost-effective at a willingness-to-pay threshold of €50 000/QALY, compared to CT-DISC, was 29%.
CT-DISC may be considered as an alternative therapeutic option to CT-CONT in patients with mESCC who have stable disease after an initial chemotherapy treatment phase. A continuous chemotherapy could indeed reduce the number of QALYs because of the disutility associated with the continuous treatment.
转移性食管鳞癌(mESCC)患者采用持续化疗,尽管该方案的临床获益、患者相关副作用和不合理的医疗费用等方面的证据有限。本研究在法国开展了一项 E-DIS 试验(NCT01248299)的成本-效用分析,该试验比较了初始 6 周治疗阶段后无进展患者中氟尿嘧啶/铂类化疗持续(CT-CONT)与化疗终止(CT-DISC)的一线治疗。
采用基于患者水平数据的分割生存分析,对试验中收集的生存结局、生活质量(EQ-5D-3L)和医疗费用数据进行分析。18 个月时,评估平均质量调整生命年(QALYs)和医疗成本,以评估增量净货币收益和增量成本效益比。使用非参数自举法和单变量分析处理不确定性。67 例 mESCC 患者被随机分组并纳入成本-效用分析。
平均而言,CT-CONT 略微降低了 QALYs(-0.038)并增加了每位患者的成本(+1177 欧元)。在支付意愿阈值为 50000 欧元/QALY 时,增量净货币收益为负值(-3077 欧元[95%置信区间:-6564;4359]),增量成本效益比为-30958 欧元/QALY(CT-CONT 占优)。与 CT-DISC 相比,在支付意愿阈值为 50000 欧元/QALY 时,CT-CONT 治疗方案具有成本效益的概率为 29%。
对于初始化疗治疗阶段后疾病稳定的 mESCC 患者,CT-DISC 可能是 CT-CONT 的替代治疗选择。持续化疗可能确实会降低 QALYs,因为连续治疗会带来不适。