Nierenberg Tori C, Thomas Samantha M, Halliday Ian, Botty van den Bruele Astrid, Chiba Akiko, Modell Parrish Kendra J, Woriax Hannah E, DiNome Maggie L, Westbrook Kelly E, Plichta Jennifer K
Department of Surgery, Duke University Medical Center, Durham, NC, 27710, USA.
Department of Biostatistics & Bioinformatics, Duke University, Durham, NC, USA.
Breast Cancer Res Treat. 2025 Jul;212(1):161-172. doi: 10.1007/s10549-025-07717-3. Epub 2025 May 11.
Neoadjuvant therapies can result in pathologic complete response (pCR) in patients with breast cancer, which can be predictive of long-term outcomes. Patients with estrogen receptor positive (ER +) tumors may receive either neoadjuvant chemotherapy (NAC) or neoadjuvant endocrine therapy (NET). We sought to compare survival outcomes in those with non-metastatic ER + breast cancer who received NET or NAC and achieved pCR.
All patients diagnosed with ER + /HER2- stage I-III breast cancer, who received neoadjuvant systemic therapy followed by surgery, and achieved pCR, were selected from the National Cancer Database (NCDB, 2010-2021). The Kaplan-Meier method was used to estimate overall survival (OS), and log-rank tests were used to test for differences in OS. Cox Proportional Hazards models were used to estimate the association of NAC vs NET with OS, after adjustment for covariates.
3313 patients met eligibility criteria: 3148 received NAC and 165 NET. The median follow-up for the entire cohort was 82 months (95% CI 80.4-83.1). Patients who received NAC were significantly younger (median age: NAC 49y vs NET 64y; p < 0.001), more likely to have a comorbidity score of 0 (NAC 89.3% vs NET 81.2%, p = 0.004), and more likely to have private insurance (NAC 68.9% vs NET 44.2%, p < 0.001). There were no significant differences between the NAC and NET patients based on race and ethnicity, income, education, or community type (all p > 0.05). The NAC treated patients were more likely to have larger tumors [median tumor size (IQR): NAC 3 cm (2.0-4.3) vs NET 1.3 cm (0.7-2.8); p < 0.001)], ductal histology (NAC 92.6% vs 81.2%, p < 0.001), and grade 3 tumors (NAC 70.2% vs 10.3%, p < 0.001). In the unadjusted Kaplan-Meier analysis, there was no significant difference in OS between NAC vs NET [5-year OS: NAC 0.935 vs NET 0.916; p = 0.08]. After adjustment for demographics, disease characteristics, and treatments, there remained no association between OS and study group (NAC vs NET; p = 0.63).
Patients with ER + /HER2- early-stage breast cancer who achieved pCR had similar OS, regardless of whether they received NAC or NET. As such, pCR appears to have similar prognostic value irrespective of the type of systemic therapy used to obtain this favorable outcome.
新辅助治疗可使乳腺癌患者获得病理完全缓解（pCR），这可预测长期预后。雌激素受体阳性（ER +）肿瘤患者可接受新辅助化疗（NAC）或新辅助内分泌治疗（NET）。我们旨在比较接受NET或NAC并达到pCR的非转移性ER +乳腺癌患者的生存结局。
从国家癌症数据库（NCDB，2010 - 2021年）中选取所有诊断为ER + /HER2- Ⅰ - Ⅲ期乳腺癌、接受新辅助全身治疗后手术且达到pCR的患者。采用Kaplan-Meier方法估计总生存期（OS），并使用对数秩检验来检验OS的差异。在对协变量进行调整后，使用Cox比例风险模型估计NAC与NET和OS之间的关联。
3313例患者符合入选标准：3148例接受NAC，165例接受NET。整个队列的中位随访时间为82个月（95%CI 80.4 - 83.1）。接受NAC的患者明显更年轻（中位年龄：NAC为49岁，NET为64岁；p < 0.001），合并症评分为0的可能性更高（NAC为89.3%，NET为81.2%，p = 0.004），且拥有私人保险的可能性更高（NAC为68.9%，NET为44.2%，p < 0.001）。基于种族和民族、收入、教育程度或社区类型，NAC组和NET组患者之间无显著差异（所有p > 0.05）。接受NAC治疗的患者更可能有更大的肿瘤[中位肿瘤大小（IQR）：NAC为3 cm（2.0 - 4.3），NET为1.3 cm（0.7 - 2.8）；p < 0.001]，导管组织学类型（NAC为92.6%，NET为81.2%，p < 0.001），以及3级肿瘤（NAC为70.2%，NET为10.3%，p < 0.001）。在未调整的Kaplan-Meier分析中，NAC组和NET组的OS无显著差异[5年OS：NAC为0.935，NET为0.916；p = 0.08]。在对人口统计学、疾病特征和治疗进行调整后，OS与研究组（NAC与NET）之间仍无关联（p = 0.63）。
达到pCR的ER + /HER2- 早期乳腺癌患者，无论接受NAC还是NET，其OS相似。因此，无论用于获得这一良好结局的全身治疗类型如何，pCR似乎都具有相似的预后价值。
