NeoALTTO and CALGB 40601 study designs and patient populations
tumor, death, primary malignant neoplasm, breast cancer
TUMOR, RECURRENCE, BREAST CANCER, EVENT, DISEASE, MAY, SECONDARY, BREAST, PRIMARY MALIGNANT NEOPLASM
The NeoALTTO phase III trial randomized 455 HER2-positive early-stage BC patients to receive neoadjuvant trastuzumab (T), lapatinib (L) or T + LThe primary endpoint of the trial was pCR defined as either absence of invasive tumor cells in the breast (ypT0/is) as defined by the National Surgical Adjuvant Breast and Bowel Project criteria, later amended to the absence of invasive tumor cells in the breast and in the axillary lymph nodes (ypT0/is ypN0) according to the Food and Drug Administration criteria. The main secondary endpoint was EFS, defined as the time from randomization to first event, i.e., breast cancer relapse after surgery, second primary malignant neoplasm, or death without recurrence for women who received surgery for breast cancer, or, for those who did not undergo surgery, death during clinical follow-up or non-completion of any neoadjuvant investigational drugs due to progressive disease. OS was defined as the time from randomization to death from any cause.In the phase III CALGB 40601 trial, 305 eligible patients with newly diagnosed, histologically confirmed, untreated clinical stage II to III HER2-positive disease were randomized to receive neoadjuvant weekly paclitaxel in combination with either T, L, or T + L for 16 weeksEthics committee and relevant health authorities at each participating site approved the NeoALTTO and CALGB 40601 studies and all patients provided written informed consent including future biomarker research. For NeoALTTO, the sites which run the trial are listed in Supplementary data The NeoALTTO trial is registered at In NeoALTTO, clinical data were collected at Institut Jules Bordet and Frontier Science Scotland; analyses are based on the on the clinical study database frozen on May 26, 2016. In CALGB 40601, clinical data were collected at the CALGB (Alliance) Statistics and Data Center; analyses are based on the on the clinical study database frozen on October 06, 2021.