Identification of Patients With Documented Pathologic Complete Response in the Breast After Neoadjuvant Chemotherapy for Omission of Axillary Surgery

JAMA Surg. 2017 Jul 1;152(7):665-670. doi: 10.1001/jamasurg.2017.0562.


Importance: A pathologic complete response (pCR; no invasive or in situ cancer) occurs in 40% to 50% of patients with HER2-positive (HER2+) and triple-negative (TN) breast cancer. The need for surgery if percutaneous biopsy of the breast after neoadjuvant chemotherapy (NCT) indicates pCR in the breast (hereinafter referred to as breast pCR) has been questioned, and appropriate management of the axilla in such patients is unknown.

Objective: To identify patients among exceptional responders to NCT with a low risk for axillary metastases when breast pCR is documented who may be eligible for an omission of surgery clinical trial design.

Design, setting, and participants: This prospective cohort study at a single-institution academic national comprehensive cancer center included 527 consecutive patients with HER2+/TN (T1/T2 and N0/N1) cancer treated with NCT followed by standard breast and nodal surgery from January 1, 2010, through December 31, 2014.

Main outcomes and measures: Patients who achieved a breast pCR were compared with patients who did not based on subtype, initial ultrasonographic findings, and documented pathologic nodal status. Incidence of positive findings for nodal disease on final pathologic review was calculated for patients with and without pCR and compared using relative risk ratios with 95% CIs.

Results: The analysis included 527 patients (median age, 51 [range, 23-84] years). Among 290 patients with initial nodal ultrasonography showing N0 disease, 116 (40.4%) had a breast pCR and 100% had no evidence of axillary lymph node metastases after NCT. Among 237 patients with initial biopsy-proved N1 disease, 69 of 77 (89.6%) with and 68 of 160 (42.5%) without a breast pCR had no evidence of residual nodal disease (P < .01). Patients without a breast pCR had a relative risk for positive nodal metastases of 7.4 (95% CI, 3.7-14.8; P < .001) compared with those with a breast pCR.

Conclusions and relevance: Breast pCR is highly correlated with nodal status after NCT, and the risk for missing nodal metastases without axillary surgery in this cohort is extremely low. These data provide the fundamental basis and rationale for management of the axilla in clinical trials of omission of cancer surgery when image-guided biopsy indicates a breast pCR.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, N.I.H., Extramural

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Antineoplastic Agents / therapeutic use
  • Axilla
  • Biopsy, Fine-Needle
  • Breast Neoplasms / drug therapy
  • Breast Neoplasms / pathology
  • Breast Neoplasms / surgery*
  • Carcinoma, Ductal, Breast / drug therapy
  • Carcinoma, Ductal, Breast / secondary
  • Carcinoma, Ductal, Breast / surgery*
  • Carcinoma, Intraductal, Noninfiltrating / drug therapy
  • Carcinoma, Intraductal, Noninfiltrating / pathology
  • Carcinoma, Intraductal, Noninfiltrating / surgery*
  • Chemotherapy, Adjuvant
  • Clinical Trials as Topic
  • Female
  • Humans
  • Lymph Node Excision*
  • Lymph Nodes / diagnostic imaging
  • Lymph Nodes / physiology*
  • Lymphatic Metastasis
  • Middle Aged
  • Neoadjuvant Therapy
  • Neoplasm Staging
  • Patient Selection
  • Prospective Studies
  • Receptor, ErbB-2 / analysis*
  • Risk Assessment
  • Ultrasonography
  • Young Adult


  • Antineoplastic Agents
  • ERBB2 protein, human
  • Receptor, ErbB-2