Celgene Corporation, 86 Morris Ave, Summit, NJ 07901, USA.
Partnership for Health Analytic Research, LLC, 280 S. Beverly Drive, Beverly Hills, CA 90212, USA.
J Comp Eff Res. 2018 Jan;7(1):67-83. doi: 10.2217/cer-2017-0027. Epub 2018 Jan 5.
With the aggregation of real-world data in healthcare, opportunities for outcomes research are growing. In this study, we summarize published literature examining comparative effectiveness research (CER), treatment patterns, quality of life (QoL) and costs in HER2-negative and triple-negative (TN) metastatic breast cancer (mBC).
PubMed (2010-January 2016) and four conferences (2013-January 2016) were searched using MeSH/keywords, including mBC, QoL, morbidity and therapeutics. Studies relating to CER, treatment patterns, QoL, costs or treatment appropriateness in US patients with HER2-negative/TN mBC were included in the review.
Of 1782 identified records, 33 studies met full inclusion criteria: seven related to CER, 18 to treatment patterns, one to treatment appropriateness/navigation, two to QoL and five to costs. Studies varied in objectives, designs and outcomes. Study designs included retrospective chart reviews (52%), retrospective secondary database analyses (27%), economic models (12%), physician surveys (6%) and patient surveys (3%). 25 studies reported results on HER2-negative mBC, six on TN mBC and two on both subtypes. The most common end points examined were treatment patterns, overall survival and progression-free survival.
Outcomes research in HER2-negative mBC in the USA was limited, specifically among TN patients, indicating an opportunity for further research in this high unmet need population. Endpoints and treatment options varied, thus, it is difficult to draw summary conclusions about these studies. Outcomes research examining real-world data in mBC has increased in recent years, and may continue to grow with the implementation of new policy programs.
随着医疗保健领域真实世界数据的积累,开展结局研究的机会越来越多。本研究总结了已发表的文献,旨在考察人表皮生长因子受体 2(HER2)阴性和三阴性(TN)转移性乳腺癌(mBC)的比较疗效研究(CER)、治疗模式、生活质量(QoL)和成本。
检索了PubMed(2010 年 1 月至 2016 年 1 月)和四个会议(2013 年 1 月至 2016 年 1 月)的文献,使用 MeSH/关键词,包括 mBC、QoL、发病率和治疗。本综述纳入了与美国 HER2 阴性/TN mBC 患者的 CER、治疗模式、QoL、成本或治疗适宜性相关的研究。
在 1782 条记录中,有 33 项研究符合全部纳入标准:7 项与 CER 相关,18 项与治疗模式相关,1 项与治疗适宜性/导航相关,2 项与 QoL 相关,5 项与成本相关。研究目的、设计和结果各不相同。研究设计包括回顾性病历审查(52%)、回顾性二次数据库分析(27%)、经济模型(12%)、医生调查(6%)和患者调查(3%)。25 项研究报告了 HER2 阴性 mBC 的结果,6 项研究报告了 TN mBC 的结果,2 项研究报告了这两种亚型的结果。最常检查的终点是治疗模式、总生存和无进展生存。
美国 HER2 阴性 mBC 的结局研究有限,尤其是在 TN 患者中,表明在这一高未满足需求人群中需要进一步开展研究。终点和治疗方案各不相同,因此,很难对这些研究得出总结性结论。近年来,mBC 的真实世界数据结局研究有所增加,随着新政策项目的实施,可能会继续增加。