Temerty Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada.
Canadian Centre for Applied Research in Cancer Control, Toronto, Ontario, Canada.
JAMA Netw Open. 2022 Feb 1;5(2):e2145460. doi: 10.1001/jamanetworkopen.2021.45460.
To date, limited studies have examined the comparative outcomes of pertuzumab treatment in the real-world setting. End-of-study analyses of the CLEOPATRA trial found median overall survival (OS) of 57.1 months in patients receiving pertuzumab compared with 40.8 months in control patients, a benefit of 16.3 months. However, studies examining the real-world use of pertuzumab have found conflicting results.
To assess the real-world comparative effectiveness and safety of pertuzumab, trastuzumab, and chemotherapy for patients with metastatic breast cancer in Ontario, Canada.
DESIGN, SETTING, AND PARTICIPANTS: A population-based retrospective comparative effectiveness research study was conducted. Patients receiving first-line treatments for metastatic breast cancer from January 1, 2008, to March 31, 2018, in Ontario were identified. Data analysis was performed from November 13, 2019, to August 1, 2021. Thirteen patients had received treatment before diagnosis or were not Ontario residents and were excluded from the analysis. Of the remaining 1823 patients identified, 912 received pertuzumab and 911 were control patients. Using propensity-score methods, 579 pairs of patients receiving pertuzumab were matched to those in the control group, resulting in a total of 1158 patients in the final cohort.
Patients in the case group received pertuzumab with trastuzumab and chemotherapy and those in the control group received trastuzumab and chemotherapy.
Overall survival (the primary outcome) and hazard ratios (HRs) were calculated using Kaplan-Meier and Cox proportional hazards regression methods. Secondary outcomes included cumulative incidence of safety end points including resource use and adverse events. Follow-up duration was up to 5 years from the start of therapy, with maximum follow-up to March 31, 2019.
Of the 1158 matched patients (579 pairs) receiving pertuzumab and controls, 1151 (99%) were women (mean [SD] age, 58.2 [12.97] years). The median OS was higher in patients receiving pertuzumab (40.2; 95% CI, 35.6-47.8 months) than in the control patients (25.3; 95% CI, 22.8-27.6 months), a median OS improvement of 14.9 months. Pertuzumab was associated with reduced mortality (HR, 0.66; 95% CI, 0.57-0.79). The cumulative incidence of direct hospitalization at 1 year was lower among patients receiving pertuzumab (11.7%) compared with the control patients (19.0%) (P < .001).
Although the median OS in both the pertuzumab and control groups were shorter in this study than those observed in the CLEOPATRA trial, there appears to be a similar significant OS benefit with pertuzumab in the real-world setting.
迄今为止，有限的研究考察了在真实环境中曲妥珠单抗治疗的比较结果。CLEOPATRA 试验的终期分析发现，接受曲妥珠单抗治疗的患者中位总生存期（OS）为 57.1 个月，而对照组患者为 40.8 个月，获益为 16.3 个月。然而，研究发现，在真实世界中使用曲妥珠单抗的结果存在矛盾。
评估在加拿大安大略省接受曲妥珠单抗、帕妥珠单抗和化疗的转移性乳腺癌患者的真实世界比较疗效和安全性。
设计、设置和参与者：进行了一项基于人群的回顾性比较有效性研究。从 2008 年 1 月 1 日至 2018 年 3 月 31 日，在安大略省接受转移性乳腺癌一线治疗的患者被确定。数据分析于 2019 年 11 月 13 日至 2021 年 8 月 1 日进行。13 名患者在诊断前接受过治疗或不是安大略省居民，被排除在分析之外。在确定的 1823 名患者中，912 名接受了曲妥珠单抗治疗，911 名是对照组患者。使用倾向评分方法，将 579 对接受曲妥珠单抗的患者与对照组进行匹配，最终共有 1158 名患者进入最终队列。
病例组患者接受曲妥珠单抗联合化疗治疗，对照组患者接受曲妥珠单抗联合化疗治疗。
使用 Kaplan-Meier 和 Cox 比例风险回归方法计算总生存期（主要结局）和风险比（HR）。次要结局包括安全性终点的累积发生率，包括资源使用和不良事件。随访时间最长为治疗开始后的 5 年，最大随访时间至 2019 年 3 月 31 日。
在接受曲妥珠单抗和对照组治疗的 1158 例匹配患者（579 对）中，1151 例（99%）为女性（平均[标准差]年龄，58.2[12.97]岁）。接受曲妥珠单抗治疗的患者中位 OS 较高（40.2 个月；95%CI，35.6-47.8 个月），而对照组患者中位 OS 较低（25.3 个月；95%CI，22.8-27.6 个月），中位 OS 改善了 14.9 个月。曲妥珠单抗与死亡率降低相关（HR，0.66；95%CI，0.57-0.79）。接受曲妥珠单抗治疗的患者在 1 年内直接住院的累积发生率较低（11.7%），而对照组患者的累积发生率较高（19.0%）（P<.001）。
尽管本研究中两组患者的中位 OS 均短于 CLEOPATRA 试验中的观察结果，但曲妥珠单抗在真实环境中似乎具有相似的显著 OS 获益。
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