Real-world Comparative Effectiveness and Safety of Pertuzumab Plus Trastuzumab vs Trastuzumab as First-line Treatment for Advanced HER2-positive Breast Cancer: A Target Trial Emulation Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 2,250
- 试验地点
- 1
- 主要终点
- Overall Survival (OS)
研究概览
简要总结
This retrospective observational cohort study emulates a target trial to compare the effectiveness and safety of two first-line treatment strategies for women with HER2-positive unresectable locally advanced, recurrent, or metastatic breast cancer in routine clinical practice: pertuzumab plus trastuzumab and docetaxel versus trastuzumab and docetaxel.
Data were obtained from electronic health records held by the National Health and Medical Big Data (Eastern) Center and linked with the Jiangsu Provincial Mortality Registry. Eligible patients initiated one of the two treatment strategies between January 1, 2020, and December 31, 2024. Time zero was defined as the date of the first administration of the qualifying first-line regimen. No minimum number of treatment cycles was required. The primary outcome was overall survival. Secondary outcomes included progression-free survival and prespecified adverse events.
详细描述
Patients were classified according to the first-line regimen initiated on the index date. Time zero was defined as the date of first administration of pertuzumab plus trastuzumab and chemotherapy or trastuzumab and chemotherapy. Eligibility criteria were assessed using information available on or before time zero. Patients were not required to complete a minimum number of treatment cycles and were followed from treatment initiation regardless of subsequent treatment discontinuation or modification.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Female patients aged 18 years or older on the index date.
- •Histologically or cytologically confirmed breast cancer.
- •HER2-positive disease, defined according to available pathology records as immunohistochemistry (IHC) 3+ or IHC 2+ with positive in situ hybridization.
- •Unresectable locally advanced, recurrent, or metastatic breast cancer on the index date.
- •No prior systemic anticancer therapy for unresectable locally advanced, recurrent, or metastatic breast cancer before the index date.
- •Initiation of first-line treatment with either:
- •pertuzumab plus trastuzumab plus docetaxel; or
- •trastuzumab plus docetaxel between January 1, 2020 and December 31,
- •Sufficient information available on or before the index date to determine eligibility, treatment assignment, and baseline covariates.
排除标准
- •Early-stage or resectable breast cancer treated with curative intent on the index date.
- •Prior systemic anticancer therapy for unresectable locally advanced, recurrent, or metastatic breast cancer before the index date.
- •Another active primary malignant tumor on the index date that was expected to materially affect survival.
- •Insufficient or invalid information to determine HER2 status, advanced disease status, index treatment regimen, or index date.
- •Absence of a valid patient identifier required for linkage between the electronic health record database and the mortality registry.
研究组 & 干预措施
Pertuzumab Plus Trastuzumab and Docetaxel
干预措施: Docetaxel (Drug)
Pertuzumab Plus Trastuzumab and Docetaxel
干预措施: Pertuzumab (Drug)
Pertuzumab Plus Trastuzumab and Docetaxel
干预措施: Trastuzumab (Drug)
Trastuzumab and Docetaxel
干预措施: Pertuzumab (Drug)
Trastuzumab and Docetaxel
干预措施: Docetaxel (Drug)
结局指标
主要结局
Overall Survival (OS)
时间窗: From the index date until death, last known follow-up, or December 31, 2024, whichever occurred first (up to 60 months)
Overall survival was defined as the time from the first administration of the index first-line treatment regimen to death from any cause.
次要结局
- Real-World Progression-Free Survival (rwPFS)(From the index date until documented real-world disease progression, death, last known progression-free follow-up, or December 31, 2024, whichever occurred first (up to 60 months))
- Proportion of Participants With Prespecified Grade 3 or Higher Adverse Events(From the index date through the documented end of first-line treatment (up to 60 months))
研究者
Xin Li
Professor,PhD,PhD supervisor,Deputy Dean of the School of Pharmacy,Nanjing Medical University
Nanjing Medical University