Department of Pharmacy, The Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China.
Department of Pharmacy, Zhejiang Cancer Hospital, Hangzhou, China.
BMJ Open. 2020 Feb 12;10(2):e030738. doi: 10.1136/bmjopen-2019-030738.
Cetuximab plus leucovorin, fluorouracil and oxaliplatin (FOLFOX-4) is superior to FOLFOX-4 alone as a first-line treatment for patients with metastatic colorectal cancer with RAS wild-type (RAS wt mCRC), with significantly improved survival benefit by TAILOR, an open-label, randomised, multicentre, phase III trial. Nevertheless, the cost-effectiveness of these two regimens remains uncertain. The following study aims to determine whether cetuximab combined with FOLFOX-4 is a cost-effective regimen for patients with specific RAS wt mCRC in China.
A cost-effectiveness model combined decision tree and Markov model was built to simulate pateints with RAS wt mCRC based on health states of dead, progressive and stable. The health outcomes from the TAILOR trial and utilities from published data were used respectively. Costs were calculated with reference to the Chinese societal perspective. The robustness of the results was evaluated by univariate and probabilistic sensitivity analyses.
The included patients were newly diagnosed Chinese patients with fully RAS wt mCRC.
First-line treatment with either cetuximab plus FOLFOX-4 or FOLFOX-4.
The primary outcomes are costs, quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs).
Baseline analysis disclosed that the QALYs was increased by 0.383 caused by additional cetuximab, while an increase of US$62 947 was observed in relation to FOLFOX-4 chemotherapy. The ICER was US$164 044 per QALY, which exceeded the willingness-to-pay threshold of US$28 106 per QALY.
Despite the survival benefit, cetuximab combined with FOLFOX-4 is not a cost-effective treatment for the first-line regime of patients with RAS wt mCRC in China.
TAILOR trial (NCT01228734); Post-results.
西妥昔单抗联合亚叶酸钙、氟尿嘧啶和奥沙利铂（FOLFOX-4）作为 RAS 野生型（RAS wt mCRC）转移性结直肠癌患者的一线治疗药物优于单独使用 FOLFOX-4，这一结果在 TAILOR 试验中得到证实，这是一项开放标签、随机、多中心、III 期临床试验。然而，这两种方案的成本效益仍然不确定。本研究旨在确定西妥昔单抗联合 FOLFOX-4 是否是中国特定 RAS wt mCRC 患者的一种具有成本效益的治疗方案。
采用决策树和马尔可夫模型相结合的成本效果模型，基于死亡、进展和稳定三种健康状态，模拟 RAS wt mCRC 患者。分别使用 TAILOR 试验的健康结果和已发表数据的效用值。参考中国社会角度计算成本。通过单变量和概率敏感性分析评估结果的稳健性。
纳入的患者为新诊断的中国 RAS wt mCRC 患者。
一线治疗采用西妥昔单抗联合 FOLFOX-4 或 FOLFOX-4。
主要结果是成本、质量调整生命年（QALYs）和增量成本效果比（ICERs）。
基线分析显示，西妥昔单抗的额外使用导致 QALYs 增加了 0.383，而 FOLFOX-4 化疗则增加了 62947 美元。ICER 为每 QALY 164044 美元，超过了中国每 QALY 28106 美元的意愿支付阈值。
尽管有生存获益，但西妥昔单抗联合 FOLFOX-4 对于中国 RAS wt mCRC 患者的一线治疗方案来说并不是一种具有成本效益的治疗方法。
TAILOR 试验（NCT01228734）；结果公布后。
