Ben Y. Durkee, Yushen Qian, Erqi L. Pollom, Martin T. King, Sara A. Dudley, Jenny L. Shaffer, Daniel T. Chang, Iris C. Gibbs, and Kathleen C. Horst, Stanford University School of Medicine; and Jeremy D. Goldhaber-Fiebert, Stanford University, Stanford CA.
J Clin Oncol. 2016 Mar 20;34(9):902-9. doi: 10.1200/JCO.2015.62.9105. Epub 2015 Sep 8.
The Clinical Evaluation of Pertuzumab and Trastuzumab (CLEOPATRA) study showed a 15.7-month survival benefit with the addition of pertuzumab to docetaxel and trastuzumab (THP) as first-line treatment for patients with human epidermal growth factor receptor 2 (HER2) -overexpressing metastatic breast cancer. We performed a cost-effectiveness analysis to assess the value of adding pertuzumab.
We developed a decision-analytic Markov model to evaluate the cost effectiveness of docetaxel plus trastuzumab (TH) with or without pertuzumab in US patients with metastatic breast cancer. The model followed patients weekly over their remaining lifetimes. Health states included stable disease, progressing disease, hospice, and death. Transition probabilities were based on the CLEOPATRA study. Costs reflected the 2014 Medicare rates. Health state utilities were the same as those used in other recent cost-effectiveness studies of trastuzumab and pertuzumab. Outcomes included health benefits expressed as discounted quality-adjusted life-years (QALYs), costs in US dollars, and cost effectiveness expressed as an incremental cost-effectiveness ratio. One- and multiway deterministic and probabilistic sensitivity analyses explored the effects of specific assumptions.
Modeled median survival was 39.4 months for TH and 56.9 months for THP. The addition of pertuzumab resulted in an additional 1.81 life-years gained, or 0.62 QALYs, at a cost of $472,668 per QALY gained. Deterministic sensitivity analysis showed that THP is unlikely to be cost effective even under the most favorable assumptions, and probabilistic sensitivity analysis predicted 0% chance of cost effectiveness at a willingness to pay of $100,000 per QALY gained.
THP in patients with metastatic HER2-positive breast cancer is unlikely to be cost effective in the United States.
Pertuzumab 和曲妥珠单抗的临床评估(CLEOPATRA)研究表明,与多西他赛和曲妥珠单抗(THP)联合使用相比,在人表皮生长因子受体 2(HER2)过表达转移性乳腺癌患者的一线治疗中添加培妥珠单抗可带来 15.7 个月的生存获益。我们进行了一项成本效益分析,以评估添加培妥珠单抗的价值。
我们开发了一个决策分析马尔可夫模型,以评估转移性乳腺癌美国患者中多西他赛加曲妥珠单抗(TH)联合或不联合培妥珠单抗的成本效益。该模型每周对患者的剩余寿命进行跟踪。健康状态包括稳定疾病、进展疾病、临终关怀和死亡。转移概率基于 CLEOPATRA 研究。成本反映了 2014 年医疗保险费率。健康状态效用与最近曲妥珠单抗和培妥珠单抗的其他成本效益研究中使用的效用相同。结果包括以折扣后的质量调整生命年(QALYs)表示的健康效益、以美元表示的成本以及以增量成本效益比表示的成本效益。单维和多方位确定性和概率敏感性分析探讨了特定假设的影响。
TH 的中位生存为 39.4 个月,THP 的中位生存为 56.9 个月。添加培妥珠单抗可额外获得 1.81 年的生存,或额外获得 0.62 个 QALY,每获得一个 QALY 的成本为 472,668 美元。确定性敏感性分析表明,即使在最有利的假设下,THP 也不太可能具有成本效益,概率敏感性分析预测在获得每 QALY 的意愿支付为 100,000 美元的情况下,成本效益的可能性为 0%。
在转移性 HER2 阳性乳腺癌患者中,THP 在美国不太可能具有成本效益。