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Standardized Definitions for Efficacy End Points in Neoadjuvant Breast Cancer Clinical Trials: NeoSTEEP
Jennifer K Litton1, Meredith M Regan2, Lajos Pusztai3
1Department of Breast Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX.
Purpose:
The Standardized Definitions for Efficacy End Points (STEEP) criteria, established in 2007 and updated in 2021 (STEEP 2.0), provide standardized definitions of adjuvant breast cancer (BC) end points. STEEP 2.0 identified a need to separately address end points for neoadjuvant clinical trials. The multidisciplinary NeoSTEEP working group of experts was convened to critically evaluate and align neoadjuvant BC trial end points.
Methods:
The NeoSTEEP working group concentrated on neoadjuvant systemic therapy end points in clinical trials with efficacy outcomes-both pathologic and time-to-event survival end points-particularly for registrational intent. Special considerations for subtypes and therapeutic approaches, imaging, nodal staging at surgery, bilateral and multifocal diseases, correlative tissue collection, and US Food and Drug Administration regulatory considerations were contemplated.
Results:
The working group recommends a preferred definition of pathologic complete response (pCR) as the absence of residual invasive cancer in the complete resected breast specimen and all sampled regional lymph nodes (ypT0/Tis ypN0 per AJCC staging). Residual cancer burden should be a secondary end point to facilitate future assessment of its utility. Alternative end points are needed for hormone receptor-positive disease. Time-to-event survival end point definitions should pay particular attention to the measurement starting point. Trials should include end points originating at random assignment (event-free survival and overall survival) to capture presurgery progression and deaths as events. Secondary end points adapted from STEEP 2.0, which are defined from starting at curative-intent surgery, may also be appropriate. Specification and standardization of biopsy protocols, imaging, and pathologic nodal evaluation are also crucial.
Conclusion:
End points in addition to pCR should be selected on the basis of clinical and biologic aspects of the tumor and the therapeutic agent investigated. Consistent prespecified definitions and interventions are paramount for clinically meaningful trial results and cross-trial comparison.
Insights
New guidelines standardize efficacy end points for neoadjuvant breast cancer trials. The NeoSTEEP working group recommends a preferred definition for pathologic complete response (pCR) and emphasizes standardized trial designs for better breast cancer research.
Area of Science:
- Oncology
- Clinical Trials
- Biostatistics
Background:
- The Standardized Definitions for Efficacy End Points (STEEP) criteria, updated in 2021, established definitions for adjuvant breast cancer (BC) end points.
- STEEP 2.0 highlighted the need for distinct end points in neoadjuvant BC clinical trials.
Purpose of the Study:
- To critically evaluate and align end points for neoadjuvant breast cancer clinical trials.
- To develop standardized definitions for efficacy outcomes in neoadjuvant systemic therapy trials.
Main Methods:
- A multidisciplinary NeoSTEEP working group of experts was convened.
- Focused on neoadjuvant systemic therapy end points, including pathologic and time-to-event survival outcomes.
- Considered subtype-specific needs, imaging, nodal staging, and regulatory factors.
Main Results:
- Recommends defining pathologic complete response (pCR) as absence of residual invasive cancer in breast and lymph nodes (ypT0/Tis ypN0).
- Suggests residual cancer burden as a secondary end point and calls for alternative end points for hormone receptor-positive disease.
- Advocates for time-to-event end points measured from random assignment (event-free survival, overall survival) to capture early events.
Conclusions:
- End point selection should align with tumor biology and therapeutic agents.
- Consistent, prespecified definitions and interventions are crucial for meaningful trial results and cross-trial comparisons.
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