软组织肉瘤 (STS) 约占成人实体恶性肿瘤的 1%，主要通过手术治疗，围手术期治疗的选择是一个具有挑战性且高度个体化的决定。评估 STS 新辅助治疗方式的临床试验主要使用临床结果或放射学反应作为终点，而不采用病理完全缓解 (pCR) 作为指定的研究终点。我们的系统评价旨在评估 STS 不同新辅助治疗方式临床试验中的 pCR 率及其与患者临床结果的相关性。纳入了 23 项 I、II 和 III 期研究，从中检索了有关每种治疗的 pCR 率以及 pCR 与临床结果的相关性的数据。在评估 pCR 的 16 项试验中，达到 pCR 的患者比例为 8% 至 58%。大多数这些试验的目的并不是建立 pCR 和临床结果之间的关联。然而，在那些研究这种相关性的研究中，发现 pCR 与 5 年疾病特异性生存 (DSS) 和 5 年总生存 (OS) 之间呈正相关。虽然 pCR 是指导三阴性乳腺癌和尿路上皮癌等其他肿瘤治疗决策的关键标志物，但尚未用于 STS 的类似情况。我们的研究结果表明，在不同的 STS 新辅助治疗中，患者达到 pCR 的情况存在差异，并且可能与患者的预后呈正相关。因此，我们建议考虑将 pCR 作为未来 STS 前瞻性试验的替代终点。版权所有 © 2024 Elsevier Ltd。保留所有权利。

Soft tissue sarcomas (STS), comprising approximately 1% of adult solid malignancies, are primarily treated with surgery, with the choice of perioperative treatment being a challenging and highly individualized decision. Clinical trials assessing neoadjuvant modalities in STS predominantly use clinical outcomes or radiologic response as endpoints, with pathologic complete response (pCR) not being employed as a designated study endpoint. Our systematic review aimed to assess the rates of pCR in clinical trials of different neoadjuvant modalities for STS and its correlation with patient clinical outcomes. 23 phase I, II and III studies were included, from which data regarding rates of pCR with each treatment, as well as correlation of pCR with clinical outcomes were retrieved. In 16 trials that assessed pCR, the percentage of patients who achieved a pCR ranged from 8 to 58%. Most of these trials did not aim to establish an association between pCR and clinical outcomes. However, among those that did investigate this correlation, a positive association was identified between pCR and both 5-year disease-specific survival (DSS) and 5-year overall survival (OS). While pCR serves as a crucial marker guiding treatment decisions in other neoplasms like triple negative breast cancer and urothelial cancer, it is not yet used in a similar setting for STS. Our findings indicate variability in patients achieving pCR across different neoadjuvant treatments for STS and a possible positive correlation with patient outcomes. Consequently, we propose considering pCR as a surrogate endpoint in future prospective trials for STS.Copyright © 2024 Elsevier Ltd. All rights reserved.