Abstract Background Neoadjuvant therapy has emerged as a crucial treatment paradigm for locally advanced and early-stage breast cancer, offering clinical benefits beyond tumor downstaging. It serves as a dynamic platform for individualized efficacy prediction, new drug development, and accelerating precision medicine. In the era of immunotherapy, the neoadjuvant setting presents unique opportunities to decipher tumor biology and microenvironment evolution under therapeutic pressure. Methods This review systematically analyzes translational studies leveraging neoadjuvant platforms, focusing on their role in addressing challenges in immunotherapy, such as treatment benefit heterogeneity and response assessment complexity. We examine key clinical trials, including I-SPY 2, KEYNOTE-522, and others to elucidate how these platforms bridge laboratory discoveries with clinical practice. Results The I-SPY 2 trial demonstrated the potential of adaptive platform frameworks to accelerate precision therapy, redefine targeted therapy strategies, and validate immunotherapy-containing regimens. In triple-negative breast cancer (TNBC), neoadjuvant immunotherapy has shown significant benefits, yet patient selection remains complex. Translational research has identified limitations in conventional biomarkers and highlighted the need for multi-factor predictive models. Innovative assessment methods, including lymph node response evaluation and liquid biopsy for dynamic monitoring, have shown promise in early identification of responders. Conclusion Translational research based on neoadjuvant platforms holds immense value for refining precision immunotherapy in breast cancer. By integrating high-quality clinical cohort data, biological samples, and high-throughput sequencing, these studies help define benefit populations, identify effective regimens, and obtain drug resistance information. Future research should focus on multidimensional biomarker discovery, innovative efficacy evaluation, and dynamic monitoring to maximize patient benefits and reduce treatment risks.
Chong et al. (Sat,) studied this question.
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