Division of Surgery, Department of Gynecologic Oncology and Reproductive Medicine, the University of Texas MD Anderson Cancer Center, Houston, Texas; and the Division of Gynecologic Oncology, Duke University Medical Center, Durham, North Carolina.
Obstet Gynecol. 2019 Apr;133(4):795-802. doi: 10.1097/AOG.0000000000003171.
We sought to determine whether use of a poly (ADP-ribose) polymerase inhibitor is cost effective for maintenance treatment of platinum-sensitive recurrent ovarian cancer.
A decision analysis model compared four maintenance strategies: 1) observation, 2) BRCA germline mutation testing and selective treatment of carriers (gBRCA only), 3) BRCA germline and tumor homologous recombination deficiency testing and selective treatment of either BRCA carriers or those with tumor HRD (gBRCA and HRD only), and 4) treat all with niraparib to progression (treat all). Costs were estimated in 2016 U.S. dollars. Incremental cost-effectiveness ratios were in dollars per progression-free quality-adjusted life-year (QALY). One-way sensitivity analyses tested multiple assumptions.
Maintenance poly (ADP-ribose) polymerase inhibitor was costlier and more effective than observation. Mean costs and progression-free QALYs were $827 and 3.4 months for observation, $46,157 and 5.7 for a BRCA-only strategy, $109,368 and 8.5 for a gBRCA and homologous recombination deficiency-only strategy, and $169,127 and 8.8 for a treat-all strategy. gBRCA-only had an incremental cost-effectiveness ratio of $243,092/progression-free QALY compared with observation; other strategies did not approach cost effectiveness. Using the current U.S. Food and Drug Administration label for maintenance poly (ADP-ribose) polymerase inhibitor regardless of biomarker status, the third-party payer cost per month (28-day supply) would need to be reduced from approximately $14,700 to $3,600 to be considered cost effective compared with observation using a willingness to pay threshold of $100,000/progression-free QALY.
Maintenance poly (ADP-ribose) polymerase inhibitor therapy for platinum-sensitive recurrent ovarian cancer is not cost effective. Treatment of patients with BRCA mutation alone or with homologous recombination deficiency-positive tumors are preferred strategies compared with a treat-all strategy. Lowering the cost may make selective niraparib maintenance therapy cost effective compared with observation.
我们旨在确定聚 ADP-核糖聚合酶抑制剂（PARPi）用于铂类敏感复发性卵巢癌维持治疗是否具有成本效益。
决策分析模型比较了四种维持治疗策略：1）观察，2）BRCA 种系突变检测和对携带者的选择性治疗（仅 gBRCA），3）BRCA 种系和肿瘤同源重组缺陷检测和对 BRCA 携带者或同源重组缺陷阳性肿瘤患者的选择性治疗（仅 gBRCA 和 HRD），以及 4）所有患者均用尼拉帕利治疗直至进展（所有治疗）。成本以 2016 年美元计。增量成本效益比为每无进展生存质量调整生命年（QALY）的美元数。单因素敏感性分析测试了多种假设。
PARPi 维持治疗比观察更昂贵但更有效。观察、仅 BRCA 策略、仅 gBRCA 和同源重组缺陷策略和所有治疗策略的平均成本和无进展 QALY 分别为 827 美元和 3.4 个月、46157 美元和 5.7 个月、109368 美元和 8.5 个月和 169127 美元和 8.8 个月。仅 gBRCA 策略与观察相比，增量成本效益比为 243092 美元/无进展 QALY；其他策略均未达到成本效益。即使根据生物标志物状态使用聚 ADP-核糖聚合酶抑制剂的现行美国食品和药物管理局（FDA）维持治疗标签，对于每个月（28 天疗程）的第三方支付者成本，需要从大约 14700 美元降低至 3600 美元，才能与观察相比，以 10 万美元/无进展 QALY 的意愿支付阈值，具有成本效益。
聚 ADP-核糖聚合酶抑制剂治疗铂类敏感复发性卵巢癌不具有成本效益。与所有治疗策略相比，BRCA 突变患者或同源重组缺陷阳性肿瘤患者的治疗策略更为优选。降低成本可能使尼拉帕利维持治疗具有成本效益，与观察相比。
