Evaluation of Pathologic Complete Response as a Surrogate for Long-Term Survival Outcomes in Triple-Negative Breast Cancer

Huang M, O'Shaughnessy J, Zhao J, Haiderali A, Cortes J, Ramsey S, Briggs A, Karantza V, Aktan G, Qi CZ, Gu C, Xie J, Yuan M, Cook J, Untch M, Schmid P, Fasching P (2020)

Publication Type: Journal article

Publication year: 2020

Journal

Journal of the National Comprehensive Cancer Network : JNCCN Jones and Barlett Publishers

Book Volume: 18

Pages Range: 1096-1104

Journal Issue: 8

DOI: 10.6004/jnccn.2020.7550

Abstract

BACKGROUND: Pathologic complete response (pCR) is a common efficacy endpoint in neoadjuvant therapy trials for triple-negative breast cancer (TNBC). Previous studies have shown that pCR is strongly associated with improved long-term survival outcomes, including event-free survival (EFS) and overall survival (OS). However, the trial-level associations between treatment effect on pCR and long-term survival outcomes are not well established. This study sought to evaluate these associations by incorporating more recent clinical trials in TNBC. METHODS: A literature review identified published randomized controlled trials (RCTs) of neoadjuvant therapy for TNBC that reported results for both pCR and EFS/OS. Meta-regression models were performed to evaluate the association of treatment effect on pCR and EFS/OS. Sensitivity analyses were conducted to assess the impact of divergent study designs. RESULTS: Ten comparisons from 8 RCTs (N=2,478 patients) were identified from the literature review. The log (odds ratio) of pCR was a significant predictor of the log (hazard ratio) of EFS (P=.003), with a coefficient of determination of 0.68 (95% CI, 0.41-0.95). There was a weaker association between pCR and OS (P=.18), with a coefficient of determination of 0.24 (95% CI, 0.01-0.77). Consistent results were found in the exploratory analysis and sensitivity analyses. CONCLUSIONS: This is the first study that has shown a trial-level association between pCR and survival outcomes in TNBC. By incorporating the most up-to-date RCTs, this study showed a significant trial-level association between pCR and EFS. A positive association between pCR and OS was also recorded.

Authors with CRIS profile

Peter Fasching Professur für Translationale Frauenheilkunde und Geburtshilfe

Involved external institutions

Analysis Group US United States (USA) (US) HELIOS Kliniken DE Germany (DE) Merck & Co., Inc. / Merck Sharp & Dohme Corp (MSD) US United States (USA) (US) Baylor University Medical Center US United States (USA) (US) London School of Hygiene and Tropical Medicine GB United Kingdom (GB) University of Washington US United States (USA) (US) Queen Mary, University of London GB United Kingdom (GB)

How to cite

APA:

Huang, M., O'Shaughnessy, J., Zhao, J., Haiderali, A., Cortes, J., Ramsey, S.,... Fasching, P. (2020). Evaluation of Pathologic Complete Response as a Surrogate for Long-Term Survival Outcomes in Triple-Negative Breast Cancer. Journal of the National Comprehensive Cancer Network : JNCCN, 18(8), 1096-1104. https://doi.org/10.6004/jnccn.2020.7550

MLA:

Huang, Min, et al. "Evaluation of Pathologic Complete Response as a Surrogate for Long-Term Survival Outcomes in Triple-Negative Breast Cancer." Journal of the National Comprehensive Cancer Network : JNCCN 18.8 (2020): 1096-1104.

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