Goldstein Daniel A, Chen Qiushi, Ayer Turgay, Howard David H, Lipscomb Joseph, Harvey R Donald, El-Rayes Bassel F, Flowers Christopher R
Department of Hematology and Medical Oncology, Winship Cancer Institute, Emory University, Atlanta, GA.
H. Milton Stewart School of Industrial and Systems Engineering, Georgia Institute of Technology, Atlanta, GA.
Clin Colorectal Cancer. 2014 Dec;13(4):219-25. doi: 10.1016/j.clcc.2014.09.007. Epub 2014 Sep 21.
Dosing chemotherapy based on BSA results in marked interindividual variability in drug exposure. A randomized trial showed increased OS and decreased toxicity with PK-guided compared with BSA-based 5-FU dosing in patients with mCRC. The objective of this study was to compare the cost effectiveness of PK-based 5-FU dosing with BSA-based 5-FU dosing in patients with mCRC receiving FOLFOX (5-FU, leucovorin, and oxaliplatin).
We developed a Markov model to evaluate the cost effectiveness of PK FOLFOX compared with BSA FOLFOX. Progression risks and cause-specific mortality were extrapolated from the fitted survival models. Costs for administration and management of adverse events were estimated based on 2013 Medicare reimbursement rates and average sale prices.
PK FOLFOX provided 2.03 QALYs at a cost of $50,205 compared with BSA FOLFOX, which provided 1.46 QALYs at a cost of $37,173. The incremental cost-effectiveness ratio (ICER) was $22,695 per QALY. The ICER remained < $50,000 per QALY in all univariate and multivariate sensitivity analyses.
At a $50,000 per QALY threshold, PK FOLFOX is cost effective for mCRC. Because of the cost effectiveness profile and OS advantage with PK FOLFOX, it should be evaluated further in comparative effectiveness studies.
基于体表面积（BSA）给药的化疗会导致药物暴露存在显著的个体间差异。一项随机试验表明，与基于BSA的5-氟尿嘧啶（5-FU）给药相比，对于转移性结直肠癌（mCRC）患者，采用基于药代动力学（PK）指导的给药可提高总生存期（OS）并降低毒性。本研究的目的是比较在接受FOLFOX（5-FU、亚叶酸钙和奥沙利铂）治疗的mCRC患者中，基于PK的5-FU给药与基于BSA的5-FU给药的成本效益。
我们建立了一个马尔可夫模型，以评估PK FOLFOX与BSA FOLFOX相比的成本效益。进展风险和特定原因死亡率是从拟合的生存模型中推断出来的。不良事件的给药和管理成本是根据2013年医疗保险报销率和平均销售价格估算的。
PK FOLFOX的成本为50,205美元，提供了2.03个质量调整生命年（QALY）；相比之下，BSA FOLFOX的成本为37,173美元，提供了1.46个QALY。增量成本效益比（ICER）为每QALY 22,695美元。在所有单变量和多变量敏感性分析中，ICER均保持在每QALY < 50,000美元。
以每QALY 50,000美元为阈值，PK FOLFOX对mCRC具有成本效益。由于PK FOLFOX的成本效益情况和OS优势，应在比较效果研究中对其进行进一步评估。
