Application of Neoadjuvant Docetaxel Plus Cisplatin in Early-Stage Triple-Negative Breast Cancer (HELEN-001): Results from a Phase II Trial

Abstract Purpose: This study investigated the effects of taxane-cisplatin combinations on pathological complete response (pCR) rates and survival outcomes in triple-negative breast cancer (TNBC). Methods: The HELEN-001 trial enrolled patients aged 18–70 years with stage II–III TNBC, randomly assigning them to receive either docetaxel (75 mg/m²) plus cisplatin (75 mg/m²) (TP) or docetaxel (75 mg/m²), doxorubicin (50 mg/m²), and cyclophosphamide (500 mg/m²) (TAC). Treatments were administered every three weeks for six cycles, with the primary endpoint being pCR (ypT0/isN0) and secondary endpoints including event-free survival (EFS), overall response rate (ORR), breast-conserving surgery rate, and toxicity. Results: From November 2018 to June 2022, 212 Asian female patients were enrolled across six hospitals in China, with 106 patients in each group. The pCR rate was significantly higher for TP (51.9%) than for TAC (35.8%, P=0.028). After a median follow-up of 40 months, EFS was 86.1% in the TP group and 80.0% in the TAC group (HR= 0.639; P=0.196). In germline BRCA1/2 mutation carriers, EFS was significantly higher with TP than with TAC (100% vs. 53.8%, P=0.008). Grade 3 or higher adverse events occurred in 54% of patients in the TP group and 48% in the TAC group. Conclusion: The TP regimen demonstrated significantly improved pCR rates with a manageable toxicity profile, suggesting the potential benefit of taxane plus platinum regimens in patients with TNBC.