Standardized Definitions for Efficacy End Points in Neoadjuvant Breast Cancer Clinical Trials: NeoSTEEP

作者：Jennifer K. Litton, Meredith M. Regan, Lajos Pusztai, Hope S. Rugo, Sara M. Tolaney, Elizabeth Garrett‐Mayer, Laleh Amiri‐Kordestani, Reva Basho, Ana F. Best, Jean-François Boileau, Carsten Denkert, Jared C. Foster, Nadia Harbeck, Heather A. Jacene, Tari A. King, Ginny Mason, Ciara C. O’Sullivan, Tatiana M. Prowell, Andrea L. Richardson, Karla A. Sepulveda, Mary Lou Smith, Judy A. Tjoe, Gulisa Turashvili, Wendy A. Woodward, Lynn Pearson Butler, Elena I. Schwartz, Larissa A. Korde · 发表于：Journal of Clinical Oncology · 年份：2023 · DOI：10.1200/jco.23.00435 · 被引用次数：116 · 研究领域：Breast Cancer Treatment Studies、Esophageal Cancer Research and Treatment、Medical Imaging Techniques and Applications

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 a...