Intensive chemotherapy versus standard chemotherapy among patients with high risk, operable, triple negative breast cancer based on integrated mRNA-lncRNA signature (BCTOP-T-A01): randomised, multicentre, phase 3 trial

BMJ. 2024 Oct 23:387:e079603. doi: 10.1136/bmj-2024-079603.

Abstract

Objective: To evaluate the feasibility of using a multigene signature to tailor individualised adjuvant therapy for patients with operable triple negative breast cancer.

Design: Randomised, multicentre, open label, phase 3 trial.

Setting: 7 cancer centres in China between 3 January 2016 and 17 July 2023.

Participants: Female patients aged 18-70 years with early triple negative breast cancer after definitive surgery.

Interventions: After risk stratification using the integrated signature, patients at high risk were randomised (1:1) to receive an intensive adjuvant treatment comprising four cycles of docetaxel, epirubicin, and cyclophosphamide followed by four cycles of gemcitabine and cisplatin (arm A; n=166) or a standard treatment of four cycles of epirubicin and cyclophosphamide followed by four cycles of docetaxel (arm B; n=170). Patients at low risk received the same adjuvant chemotherapy as arm B (arm C; n=168).

Main outcome measures: The primary endpoint was disease-free survival in the intention-to-treat analysis for arm A versus arm B. Secondary endpoints included disease-free survival for arm C versus arm B, recurrence-free survival, overall survival, and safety.

Results: Among the 504 enrolled patients, 498 received study treatment. At a median follow-up of 45.1 months, the three year disease-free survival rate was 90.9% for patients in arm A and 80.6% for patients in arm B (hazard ratio 0.51, 95% confidence interval (CI) 0.28 to 0.95; P=0.03). The three year recurrence-free survival rate was 92.6% in arm A and 83.2% in arm B (hazard ratio 0.50, 95% CI 0.25 to 0.98; P=0.04). The three year overall survival rate was 98.2% in arm A and 91.3% in arm B (hazard ratio 0.58, 95% CI 0.22 to 1.54; P=0.27). The rates of disease-free survival (three year disease-free survival 90.1% v 80.6%; hazard ratio 0.57, 95% CI 0.33 to 0.98; P=0.04), recurrence-free survival (three year recurrence-free survival 94.5% v 83.2%; 0.42, 0.22 to 0.81; P=0.007), and overall survival (three year overall survival 100% v 91.3%; 0.14, 0.03 to 0.61; P=0.002) were significantly higher in patients in arm C than in those in arm B with the same chemotherapy regimen. The incidence of grade 3-4 treatment related adverse events were 64% (105/163), 51% (86/169), and 54% (90/166) for arms A, B, and C, respectively. No treatment related deaths occurred.

Conclusions: The multigene signature showed potential for tailoring adjuvant chemotherapy for patients with operable triple negative breast cancer. Intensive regimens incorporating gemcitabine and cisplatin into anthracycline/taxane based therapy significantly improved disease-free survival with manageable toxicity.

Trial registration: ClinicalTrials.gov NCT02641847.

Publication types

  • Clinical Trial, Phase III
  • Multicenter Study
  • Randomized Controlled Trial

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Antineoplastic Combined Chemotherapy Protocols* / therapeutic use
  • Chemotherapy, Adjuvant / methods
  • China / epidemiology
  • Cisplatin / administration & dosage
  • Cisplatin / therapeutic use
  • Cyclophosphamide* / administration & dosage
  • Cyclophosphamide* / therapeutic use
  • Deoxycytidine* / administration & dosage
  • Deoxycytidine* / analogs & derivatives
  • Deoxycytidine* / therapeutic use
  • Disease-Free Survival
  • Docetaxel* / administration & dosage
  • Docetaxel* / therapeutic use
  • Epirubicin* / administration & dosage
  • Female
  • Gemcitabine*
  • Humans
  • Middle Aged
  • Risk Assessment
  • Triple Negative Breast Neoplasms* / drug therapy
  • Triple Negative Breast Neoplasms* / genetics
  • Triple Negative Breast Neoplasms* / mortality
  • Triple Negative Breast Neoplasms* / surgery
  • Young Adult

Substances

  • Epirubicin
  • Cyclophosphamide
  • Docetaxel
  • Gemcitabine
  • Deoxycytidine
  • Cisplatin

Associated data

  • ClinicalTrials.gov/NCT02641847