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General principles of neoadjuvant therapy for breast cancer

William M Sikov, MD, FACP
Section Editor
Harold Burstein, MD, PhD
Deputy Editor
Sadhna R Vora, MD


Neoadjuvant therapy refers to the systemic treatment of breast cancer prior to definitive surgical therapy (ie, preoperative therapy). Typically, neoadjuvant treatment has taken the form of chemotherapy, although there is increasing interest in expanding the role of neoadjuvant endocrine therapy in certain subsets of patients.

This topic will review patient selection, pretreatment assessment, chemotherapy options, and evaluation and treatment after neoadjuvant therapy. Specific issues related to neoadjuvant endocrine therapy, human epidermal growth factor receptor 2 (HER2)-directed therapy, and adjuvant management are discussed elsewhere. (See "Epidemiology, risk factors and the clinical approach to ER/PR negative, HER2-negative (Triple-negative) breast cancer", section on 'Neoadjuvant chemotherapy' and "Neoadjuvant therapy for newly diagnosed hormone-positive breast cancer" and "Neoadjuvant therapy for patients with HER2-positive breast cancer" and "Adjuvant endocrine therapy for non-metastatic, hormone receptor-positive breast cancer" and "Adjuvant chemotherapy for HER2-negative breast cancer" and "Adjuvant systemic therapy for HER2-positive breast cancer".)


While all systemic therapy given for nonmetastatic invasive breast cancer is intended to reduce the risk of distant recurrence, the purpose of administering it neoadjuvantly is to downstage the tumor, allowing for less extensive surgery, improved cosmetic outcomes, and reduced postoperative complications such as lymphedema [1-5]. Neoadjuvant therapy also permits an early evaluation of the effectiveness of systemic therapy. The surrogate endpoint, the presence or absence of residual invasive cancer after neoadjuvant chemotherapy, is a strong prognostic factor for risk of recurrence, especially in estrogen receptor (ER)-negative and/or human epidermal growth factor receptor 2 (HER2)-positive breast cancer. (See 'Poor response or progression on neoadjuvant therapy' below.)

Although it was hypothesized that overall survival would be improved through neoadjuvant chemotherapy, as it provides earlier initiation of systemic therapy in patients at higher risk of distant recurrence, randomized trials have demonstrated equivalent mortality for pre- or postoperative delivery of systemic therapy [1,3,6-12].

In addition to these clinical objectives, neoadjuvant therapy gives researchers the opportunity to obtain radiologic imaging, tumor specimens, and blood samples prior to, during, and in patients with sufficient residual disease at surgery, after the preoperative treatment. This enables research aimed at identifying tumor- or patient-specific biomarkers of response or resistance.


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Literature review current through: Sep 2016. | This topic last updated: Oct 14, 2016.
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