Department of Medical Oncology, Cancer Center, West China Hospital, Sichuan University, Sichuan, China; West China Biomedical Big Data Center, Sichuan University, Sichuan, China.
Department of Pathology, Sichuan Cancer Hospital & Institute, Sichuan Cancer Center, School of Medicine, University of Electronic Science and Technology of China, Chengdu, China.
Oral Oncol. 2020 Aug;107:104754. doi: 10.1016/j.oraloncology.2020.104754. Epub 2020 May 17.
The KEYNOTE-048 trial indicated pembrolizumab plus chemotherapy is an appropriate first-line treatment for recurrent or metastatic head and neck squamous cell carcinoma (HNSCC), while pembrolizumab monotherapy is optimal for PD-L1 positive patient. This study was powered to determine the most cost-effective strategy for patient with different combined positive score (CPS).
A Markov model was developed to predict progression-free survival, disease progression, or death in patients with recurrent or metastatic HNSCC based on data from the KEYNOTE-048 trial. Cost was obtained from West China Hospital, while utilities were referred to published studies. By using Monte Carlo simulations, acceptability curves were depicted to address the uncertainty of model inputs.
Compared with cetuximab plus chemotherapy, pembrolizumab monotherapy resulted in an incremental cost-effectiveness ratio (ICER) of $14,995 per quality adjusted life year (QALY) in total population and $22,779 per QALY in patients with CPS ≥ 1. Comparing pembrolizumab plus chemotherapy with standard therapy led to an ICER of $43,230 per QALY in total population and $26,157 per QALY in patients with CPS ≥ 1. For patients with CPS ≥ 20, ICERs yield by immunotherapy with or without chemotherapy exceeded the threshold of willingness to pay we set, when compared with standard therapy. Pembrolizumab plus chemotherapy was dominated by pembrolizumab alone in this patient population.
For HNSCC patients with different CPS, pembrolizumab alone was the optimal choice for total population and patients with CPS ≥ 1. Among patients with high CPS, immunotherapy with or without chemotherapy was not preferred over the standard therapy.
KEYNOTE-048 试验表明，帕博利珠单抗联合化疗是复发性或转移性头颈部鳞状细胞癌（HNSCC）的合适一线治疗方法，而帕博利珠单抗单药治疗是 PD-L1 阳性患者的最佳选择。本研究旨在确定不同联合阳性评分（CPS）患者的最具成本效益的治疗策略。
根据 KEYNOTE-048 试验的数据，建立了一个马尔可夫模型，以预测复发性或转移性 HNSCC 患者的无进展生存期、疾病进展或死亡。成本来自华西医院，而效用则参考已发表的研究。通过使用蒙特卡罗模拟，绘制可接受性曲线来解决模型输入的不确定性。
与西妥昔单抗联合化疗相比，帕博利珠单抗单药治疗在总人群中的增量成本效益比（ICER）为每质量调整生命年（QALY）14995 美元，在 CPS≥1 的患者中为每 QALY 22779 美元。与标准治疗相比，帕博利珠单抗联合化疗在总人群中的 ICER 为每 QALY43230 美元，在 CPS≥1 的患者中为每 QALY26157 美元。对于 CPS≥20 的患者，与标准治疗相比，免疫治疗联合或不联合化疗的 ICER 均超过了我们设定的意愿支付阈值。在这部分患者中，帕博利珠单抗联合化疗优于帕博利珠单抗单药治疗。
对于不同 CPS 的 HNSCC 患者，帕博利珠单抗单药治疗是总人群和 CPS≥1 患者的最佳选择。在高 CPS 患者中，免疫治疗联合或不联合化疗并不优于标准治疗。
