Medicines Policy Research Unit, Centre for Big Data Research in Health, UNSW, Sydney, Australia.
NHMRC Clinical Trials Centre, University of Sydney, Sydney, Australia.
Breast. 2021 Aug;58:106-112. doi: 10.1016/j.breast.2021.05.001. Epub 2021 May 7.
We aim to describe the treatment patterns and overall survival (OS) outcomes in patients receiving trastuzumab emtansine (T-DM1) for HER2-positive metastatic breast cancer (HER2+MBC) in routine clinical care.
Retrospective, whole-of-population cohort study of people initiating T-DM1 for HER2+MBC between October 2015 and May 2019 in Australia. We used dispensing claims to estimate time-to-T-DM1 initiation, duration of treatment, and treatments administered prior to and following T-DM1 therapy. We estimated OS from T-DM1 initiation and stratified results based on whether patients received first- or second-line T-DM1 treatment. We benchmarked outcomes to those reported in the pivotal, EMILIA trial.
345 patients initiated T-DM1: 309 as second-line therapy for HER2+MBC and 36 as first-line therapy. 51% of patients had received endocrine therapy and 98% of second-line patients received pertuzumab prior to starting T-DM1. The median age was 57 years (53 in EMILIA); median time-to-T-DM1 initiation from start of HER2-targeted therapy for HER2+MBC was 11.6 months (IQR: 7.9-16.6); median duration of T-DM1 treatment was 6.5 months (3.1-13.5; 7.6 months in EMILIA), and median OS was 19.3 months (7.9-29.5; 29.9 months in EMILIA).
Our findings highlight differences in patient characteristics (older, more previous pertuzumab therapy) and outcomes (shorter OS) from the T-DM1 pivotal trial and provide real-world estimates that can inform patient, clinician and policy, decisions around the use of HER2-targeted therapies in routine clinical care.
本研究旨在描述在常规临床护理中接受曲妥珠单抗-美坦新偶联物（T-DM1）治疗人表皮生长因子受体 2 阳性（HER2+）转移性乳腺癌（MBC）患者的治疗模式和总生存期（OS）结局。
本研究是一项回顾性、全人群队列研究，纳入了 2015 年 10 月至 2019 年 5 月期间在澳大利亚开始接受 T-DM1 治疗的 HER2+MBC 患者。我们使用配药记录来估计 T-DM1 起始时间、治疗持续时间以及在 T-DM1 治疗之前和之后给予的治疗。我们从 T-DM1 起始时间估计 OS，并根据患者接受一线或二线 T-DM1 治疗进行分层。我们将结果与关键的 EMILIA 试验报告的结果进行了基准比较。
345 例患者开始接受 T-DM1 治疗：309 例为 HER2+MBC 的二线治疗，36 例为一线治疗。51%的患者接受了内分泌治疗，98%的二线患者在开始 T-DM1 治疗前接受了帕妥珠单抗治疗。中位年龄为 57 岁（EMILIA 为 53 岁）；从 HER2 靶向治疗开始至 HER2+MBC 开始接受 T-DM1 的中位时间为 11.6 个月（IQR：7.9-16.6）；T-DM1 治疗的中位持续时间为 6.5 个月（3.1-13.5；EMILIA 为 7.6 个月），中位 OS 为 19.3 个月（7.9-29.5；EMILIA 为 29.9 个月）。
我们的研究结果突出了患者特征（年龄较大，先前接受过更多的帕妥珠单抗治疗）和结局（OS 较短）与 T-DM1 关键试验的差异，并提供了真实世界的估计值，可用于在常规临床护理中使用 HER2 靶向治疗的患者、临床医生和政策制定者的决策。
