遗传性 BRCA1/2 突变的转移性乳腺癌患者的临床结局、治疗模式和卫生资源利用：一项真实世界回顾性研究。
Clinical Outcomes, Treatment Patterns, and Health Resource Utilization Among Metastatic Breast Cancer Patients with Germline BRCA1/2 Mutation: A Real-World Retrospective Study.
机构信息
Health Economics and Outcomes Research, Pfizer Inc., San Francisco, CA, USA.
Statistics, Pfizer Inc., New York, NY, USA.
出版信息
Adv Ther. 2019 Mar;36(3):708-720. doi: 10.1007/s12325-018-0867-x. Epub 2019 Jan 17.
INTRODUCTION
With evolving treatment guidelines for germline BRCA1/2 mutation (gBRCAm) in breast cancer, we present the latest gBRCA testing rates among metastatic breast cancer (mBC) patients with hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) or triple-negative breast cancer (TNBC). Among these patients with gBRCAm, we analyzed clinical outcomes, treatment patterns, and health resource utilization (HRU).
METHODS
The Flatiron Health electronic health record database was used to assess gBRCA testing rates in a real-world retrospective analysis of US patients at least 18 years old with HR+/HER2- or TNBC, and with mBC diagnosed from January 2011 to February 2018. Outcomes were compared between gBRCAm patients with HR+/HER2- vs TNBC, adjusting for imbalances utilizing inverse probability treatment weighting; effects of HR+/HER2- vs TNBC on overall survival (OS) were assessed, antineoplastic treatments summarized, and HRU analyzed using t tests.
RESULTS
The study included 12,021 mBC patients (HR+/HER2-, 10,291; TNBC, 1730). Results for gBRCA testing were available for 2005 (16.7%) patients (HR+/HER2-, 1587; TNBC, 418). A total of 229 (1.9%) patients (HR+/HER2-, 165; TNBC, 64) had gBRCAm. Significantly worse OS in gBRCAm mBC was observed in TNBC vs HR+/HER2- [hazard ratio (95% confidence interval), 0.45 (0.27-0.74); p = 0.002]. Estimated median and 4-year OS rates for gBRCAm mBC patients with either HR+/HER2- or TNBC were 38.0 months, 23.4 months and 35.6%, 21.2% respectively. The most common first-line treatment post diagnosis for gBRCAm HR+/HER2- was letrozole (8%) vs capecitabine (14%) for gBRCAm TNBC. The number of HRU treatment visits per patient per year was significantly (p < 0.05) higher among gBRCAm mBC patients with TNBC vs HR+/HER2-.
CONCLUSION
Among HER2- mBC patients, gBRCA testing rates are low. Among gBRCAm HER2- mBC patients, the poor OS and HRU burden observed, especially in patients with TNBC, demonstrate an unmet need for more efficacious, targeted, and less HRU-intensive treatment options.
FUNDING
Pfizer.
介绍
随着胚系 BRCA1/2 突变（gBRCAm）治疗指南的不断发展，我们报告了激素受体阳性/人表皮生长因子受体 2 阴性（HR+/HER2-）或三阴性乳腺癌（TNBC）转移性乳腺癌（mBC）患者中最新的 gBRCA 检测率。在这些 gBRCAm 患者中，我们分析了临床结局、治疗模式和健康资源利用（HRU）。
方法
利用 Flatiron Health 电子病历数据库，对 2011 年 1 月至 2018 年 2 月期间至少 18 岁、患有 HR+/HER2-或 TNBC 且 mBC 诊断的美国患者进行了一项真实世界回顾性分析，评估 gBRCA 检测率。使用逆概率治疗加权法比较 gBRCAm HR+/HER2-与 TNBC 患者之间的结局差异；使用对数秩检验评估 HR+/HER2-与 TNBC 对总生存期（OS）的影响，总结抗肿瘤治疗，并使用 t 检验分析 HRU。
结果
该研究纳入了 12021 例 mBC 患者（HR+/HER2-，10291 例；TNBC，1730 例）。2005 例（16.7%）患者的 gBRCA 检测结果可供分析（HR+/HER2-，1587 例；TNBC，418 例）。共有 229 例（1.9%）患者（HR+/HER2-，165 例；TNBC，64 例）存在 gBRCAm。在 gBRCAm mBC 患者中，TNBC 与 HR+/HER2-相比，OS 明显更差[风险比（95%置信区间），0.45（0.27-0.74）；p=0.002]。gBRCAm mBC 患者的估计中位和 4 年 OS 率分别为 38.0 个月、23.4 个月和 35.6%、21.2%。gBRCAm HR+/HER2-患者的一线治疗最常见的是来曲唑（8%），而 gBRCAm TNBC 患者则是卡培他滨（14%）。与 HR+/HER2-患者相比，gBRCAm mBC 患者每年每例患者的 HRU 治疗就诊次数明显更高（p<0.05）。
结论
在 HR-/HER2-mBC 患者中，gBRCA 检测率较低。在 gBRCAm HR-/HER2-mBC 患者中，观察到较差的 OS 和 HRU 负担，特别是在 TNBC 患者中，这表明需要更有效、更有针对性且 HRU 负担更小的治疗方案。
资金来源
辉瑞公司。
Zotero 插件