Breast Center, Key Laboratory of Carcinogenesis and Translational Research (Ministry of Education), Peking University Cancer Hospital & Institute, Beijing, 100142, China.
Department of Pathology, Key Laboratory of Carcinogenesis and Translational Research (Ministry of Education), Peking University Cancer Hospital & Institute, Beijing, China.
Breast Cancer Res Treat. 2022 Oct;195(3):301-310. doi: 10.1007/s10549-022-06686-1. Epub 2022 Aug 2.
For estrogen receptor (ER)-positive breast cancer, neoadjuvant endocrine therapy (NET) has been shown to be as effective as neoadjuvant chemotherapy (NACT). We evaluated the prognostic significance of Preoperative Endocrine Prognostic Index (PEPI).
We conducted a prospective, multi-center, non-randomized, controlled trial that enrolled postmenopausal early-stage strongly ER-positive (≥ 50%) and HER2-negative breast cancer patients. All patients were given 4-month NET before surgery. The primary objective was to investigate the 5-year recurrence-free survival (RFS) in patients who had PEPI 0-1 or pathological complete response (pCR) without chemotherapy. Patients who had PEPI 0-1 or pCR were recommended to receive adjuvant endocrine therapy only and patients had PEPI ≥ 2 may receive adjuvant chemotherapy at the discretion of the treating physician.
A total of 410 patients were included and 352 patients constituted the per-protocol population. Overall, 9 patients (2.5%) had pCR (ypT0/is ypN0), 128 patients (36.4%) had PEPI = 0, and 56 patients (15.9%) had PEPI = 1. After a median follow-up of 60 months (4-104 months), patients who had PEPI 0-1 or pCR showed an improved 5-year RFS [99.5% (95% CI 98.5-99.9%) for PEPI 0-1 or pCR group vs. 93.7% (95% CI 89.6-97.8%) for PEPI ≥ 2 group, P = 0.028]. No survival difference was detected between patients received adjuvant chemotherapy vs. no chemotherapy among PEPI ≥ 2 cases.
PEPI 0-1 or pCR may be used to define a group of ER-positive and HER2-negative postmenopausal early breast cancer patients with low relapse risk for whom adjuvant chemotherapy can be safely withheld. Studies on the identification and alternative treatment options for endocrine-resistant tumors are warranted.
NCT01613560.
对于雌激素受体（ER）阳性乳腺癌，新辅助内分泌治疗（NET）已被证明与新辅助化疗（NACT）同样有效。我们评估了术前内分泌预后指数（PEPI）的预后意义。
我们进行了一项前瞻性、多中心、非随机、对照临床试验，招募了绝经后早期强 ER 阳性（≥50%）和 HER2 阴性的乳腺癌患者。所有患者均接受 4 个月的 NET 治疗，然后进行手术。主要目的是研究无化疗的 PEPI 0-1 或病理完全缓解（pCR）患者的 5 年无复发生存率（RFS）。PEPI 0-1 或 pCR 的患者建议仅接受辅助内分泌治疗，PEPI≥2 的患者可能根据治疗医生的判断接受辅助化疗。
共纳入 410 例患者，352 例患者为符合方案人群。总体而言，9 例（2.5%）患者达到 pCR（ypT0/is ypN0），128 例（36.4%）患者 PEPI=0，56 例（15.9%）患者 PEPI=1。中位随访 60 个月（4-104 个月）后，PEPI 0-1 或 pCR 的患者 5 年 RFS 得到改善[PEPI 0-1 或 pCR 组为 99.5%（95%CI 98.5-99.9%），PEPI≥2 组为 93.7%（95%CI 89.6-97.8%），P=0.028]。PEPI≥2 病例中，接受辅助化疗与未接受化疗的患者生存无差异。
PEPI 0-1 或 pCR 可用于定义一组复发风险低的绝经后早期 ER 阳性和 HER2 阴性乳腺癌患者，可安全避免辅助化疗。有必要对内分泌耐药肿瘤的识别和替代治疗选择进行研究。
NCT01613560。
