Trastuzumab combined with chemotherapy for the treatment of HER2-positive metastatic breast cancer: pivotal trial data

Ann Oncol. 2001;12 Suppl 1:S57-62.


A pivotal, randomized, multicenter, phase III trial was conducted to compare chemotherapy in combination with trastuzumab (Herceptin) vs. chemotherapy (anthracycline plus cyclophosphamide [AC] or paclitaxel) alone as first-line treatment for HER2-positive metastatic breast cancer. Results from a total of 469 patients, randomized to receive either chemotherapy alone or chemotherapy plus trastuzumab, revealed that the addition of trastuzumab improved time to disease progression significantly (7.6 vs. 4.6 months. P = 0.0001) compared with chemotherapy alone. The increase was higher in the trastuzumab plus paclitaxel subgroup (6.9 vs. 3.0 months, P = 0.0001) than in the trastuzumab plus AC subgroup (8.1 vs. 6.1 months. P = 0.0003). Patients receiving combination therapy also had a greater overall response rate (49% vs. 32%, P = 0.0002) and a longer median response duration (9.3 vs. 5.9 months, P = 0.0001) than those who received chemotherapy alone. Most importantly, median follow-up of 29 months revealed a significantly increased median survival in patients receiving trastuzumab plus chemotherapy (25.4 vs. 20.3 months, P < 0.025) compared with those receiving chemotherapy alone. Trastuzumab plus chemotherapy was well tolerated; adverse events were typically mild-to-moderate chills and fever and occurred in approximately 40% of patients, primarily following the first administration only.

Publication types

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

MeSH terms

  • Adult
  • Aged
  • Antibodies, Monoclonal / administration & dosage
  • Antibodies, Monoclonal / therapeutic use*
  • Antibodies, Monoclonal, Humanized
  • Antineoplastic Agents / administration & dosage
  • Antineoplastic Agents / therapeutic use*
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Breast Neoplasms / chemistry
  • Breast Neoplasms / drug therapy*
  • Breast Neoplasms / pathology
  • Female
  • Humans
  • Middle Aged
  • Quality of Life
  • Receptor, ErbB-2 / analysis*
  • Survival Analysis
  • Trastuzumab
  • Treatment Outcome


  • Antibodies, Monoclonal
  • Antibodies, Monoclonal, Humanized
  • Antineoplastic Agents
  • Receptor, ErbB-2
  • Trastuzumab