Product Development Data Science, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
David Geffen School of Medicine, University of California, Los Angeles, CA.
JCO Oncol Pract. 2023 Jul;19(7):435-445. doi: 10.1200/OP.22.00565. Epub 2023 May 11.
On the basis of the results from CLEOPATRA, pertuzumab plus trastuzumab and chemotherapy is the first-line standard of care for human epidermal growth factor receptor 2 (HER2)-positive metastatic breast cancer (MBC). However, discrepancies have been reported between clinical trial and real-world outcomes. We report real-world outcomes for patients with HER2-positive MBC treated with first-line pertuzumab plus trastuzumab and a taxane in routine clinical practice in the United States.
A retrospective analysis was conducted using electronic health record-derived deidentified data from the Flatiron Health database. Patients were grouped according to the first taxane received (paclitaxel/nab-paclitaxel or docetaxel). Median real-world progression-free survival (rwPFS) and overall survival (rwOS) was estimated using Kaplan-Meier methodology. Subgroup analyses were conducted in patients treated with docetaxel who met CLEOPATRA's key eligibility criteria.
We included 1,065 patients; 313 patients received paclitaxel/nab-paclitaxel and 752 received docetaxel. Patients who received paclitaxel/nab-paclitaxel were older, had a worse Eastern Cooperative Oncology Group Performance Status, and had more recurrent metastatic disease compared with the docetaxel group. After adjustment for potential confounders, similar median rwPFS (inverse probability of treatment weighted average treatment effect for the treated [IPTW-ATT] hazard ratio [HR], 1.09; 95% CI, 0.9 to 1.3; = .365) and rwOS (IPTW-ATT HR, 1.23; 95% CI, 0.96 to 1.58; = .101) was observed between treatment groups. In the subgroup of CLEOPATRA-eligible patients, median rwPFS and rwOS were 16.9 months and 57.8 months, respectively.
There was no statistically significant difference in real-world outcomes between patients treated with paclitaxel/nab-paclitaxel and those treated with docetaxel. Selecting patients using key CLEOPATRA eligibility criteria resulted in rwPFS and rwOS similar to those observed in CLEOPATRA, highlighting the importance of ensuring similar patient populations when comparing clinical trial and real-world data.
基于 CLEOPATRA 研究的结果，曲妥珠单抗联合帕妥珠单抗和化疗是人表皮生长因子受体 2（HER2）阳性转移性乳腺癌（MBC）的一线标准治疗。然而，临床试验和真实世界结果之间存在差异。我们报告了在美国常规临床实践中，HER2 阳性 MBC 患者接受一线曲妥珠单抗联合帕妥珠单抗和紫杉烷治疗的真实世界结局。
使用 Flatiron Health 数据库中电子健康记录衍生的去识别数据进行回顾性分析。根据接受的第一种紫杉烷（紫杉醇/白蛋白结合紫杉醇或多西他赛）对患者进行分组。使用 Kaplan-Meier 方法估计真实世界无进展生存期（rwPFS）和总生存期（rwOS）的中位数。对符合 CLEOPATRA 关键入选标准的接受多西他赛治疗的患者进行亚组分析。
我们纳入了 1065 例患者；313 例患者接受紫杉醇/白蛋白结合紫杉醇，752 例患者接受多西他赛。与多西他赛组相比，接受紫杉醇/白蛋白结合紫杉醇的患者年龄更大，东部肿瘤协作组体能状态更差，且有更多复发性转移性疾病。在调整潜在混杂因素后，两组中位 rwPFS（倾向评分加权治疗效果逆概率[IPTW-ATT]风险比[HR]，1.09；95%CI，0.9 至 1.3；=.365）和 rwOS（IPTW-ATT HR，1.23；95%CI，0.96 至 1.58；=.101）相似。在符合 CLEOPATRA 标准的患者亚组中，中位 rwPFS 和 rwOS 分别为 16.9 个月和 57.8 个月。
接受紫杉醇/白蛋白结合紫杉醇和多西他赛治疗的患者的真实世界结局无统计学显著差异。根据 CLEOPATRA 的关键入选标准选择患者可使 rwPFS 和 rwOS 与 CLEOPATRA 观察到的结果相似，这突出了在比较临床试验和真实世界数据时确保相似患者人群的重要性。
