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Pathological Complete Response in Neoadjuvant Treatment of Breast Cancer

  • Breast Oncology
  • Published:
Annals of Surgical Oncology Aims and scope Submit manuscript

Abstract

Background

There has been recent interest in using pathological complete response (pCR) as a potential surrogate endpoint for long-term outcomes in the neoadjuvant treatment of high-risk, early-stage breast cancer.

Methods

We review the clinical trials that have contributed to our understanding of the association between pCR and long-term outcomes, describe the various definitions of pCR, describe patient populations in which pCR may predict long-term benefit, and discuss the implications of pCR on drug development and accelerated approval for neoadjuvant treatment of breast cancer.

Results

Varying definitions of pCR across clinical trials conducted in heterogeneous patient populations make understanding the association of pCR with long-term outcomes challenging. The US Food and Drug Administration established the Collaborative Trials in Neoadjuvant Breast Cancer group to evaluate the potential use of pCR as a regulatory endpoint. The group demonstrated that pCR defined as no residual invasive cancer in the breast and axillary nodes with presence or absence of in situ cancer (ypT0/is ypN0 or ypT0 ypN0) provided a better association with improved outcomes compared to eradication of invasive tumor from the breast alone (ypT0/is).

Conclusion

Even though pCR was not validated as a surrogate endpoint for long-term outcomes, the promising data regarding the strong association of pCR with substantially improved outcomes in individual patients with more aggressive subtypes of breast cancer supported the opening of an accelerated approval pathway for patients with high-risk, early-stage breast cancer.

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Disclosure

The authors have no conflicts of interest pertaining to this publication.

Author information

Authors and Affiliations

  1. Office of Hematology and Oncology Products, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD, USA

    Patricia Cortazar MD

  2. Massey Cancer Center, Virginia Commonwealth University, Richmond, VA, USA

    Charles E. Geyer Jr. MD

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  1. Patricia Cortazar MD
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  2. Charles E. Geyer Jr. MD
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Corresponding author

Correspondence to Patricia Cortazar MD.

Additional information

Acknowledgments: This educational review series, “Neoadjuvant Therapy in Breast Cancer” is supported by an educational grant from Genentech, Inc. The Society of Surgical Oncology offers CME/MOC for this educational review series. Visit www.moc.surgonc.org for additional information.

Annals of Surgical Oncology educational reviews represent the journal’s commitment to the peer review and publication of high quality research necessary to define the safety, toxicity, or effectiveness of potential therapeutic agents compared with conventional alternatives.

This Educational Review Series may include information regarding the use of medications that may be outside the approved labeling for these products. Physicians should consult the current prescribing information for these products. Authors of Annals of Surgical Oncology educational reviews are provided at the time of article solicitation with this statement regarding off-label pharmaceutical information and research.

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Cortazar, P., Geyer, C.E. Pathological Complete Response in Neoadjuvant Treatment of Breast Cancer. Ann Surg Oncol 22, 1441–1446 (2015). https://doi.org/10.1245/s10434-015-4404-8

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  • DOI: https://doi.org/10.1245/s10434-015-4404-8

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