全身治疗在壶腹（AA）和十二指肠腺癌（DA）治疗中的作用仍知之甚少。本研究试图综合支持在 AA 和 DA 中使用新辅助治疗 (NAT) 的当前证据。该研究检索了 PubMed、Cochrane Library (Wiley)、Embase (Elsevier)、CINAHL (EBSCO) 和 ClinicalTrials.gov 数据库以查找观察性或2002 年至 2022 年间发表的随机研究评估了接受全身治疗和手术切除的非转移性 AA 或 DA 患者的生存结果。提取的数据包括总生存期、无进展生存期和病理反应（PR）率。在本研究确定的 347 份摘要中，对 29 份报告进行了全面审查，其中 15 份纳入最终审查。 2007 年至 2022 年发表的选定研究是回顾性的。八项是单中心研究；五个使用国家癌症数据库 (NCDB)；其中两项是欧洲多中心/国家研究。总体而言，没有研究发现 NAT 和前期手术（有或没有辅助治疗）之间的生存差异。两项 NCDB 研究报告称，NAT/AT 治疗比手术治疗的生存期更长。五项单中心研究报告了很大一部分 NAT 患者获得 PR，一项研究将主要 PR 确定为生存的独立预测因子。与 NAT 相关的其他结果包括从不可切除疾病转变为可切除疾病、淋巴结阳性率降低以及局部复发率降低。支持在 AA 和 DA 中使用 NAT 的证据薄弱。不存在随机研究，观察数据显示的结果好坏参半。对于 DA 和 AA 患者，NAT 似乎是安全的，但需要更好的证据来了解 DA 和 AA 壶腹周围恶性肿瘤的首选多学科治疗。© 2023。外科肿瘤学会。

The role of systemic therapy in the management of ampullary (AA) and duodenal adenocarcinoma (DA) remains poorly understood. This study sought to synthesize current evidence supporting the use of neoadjuvant therapy (NAT) in AA and DA.The study searched PubMed, Cochrane Library (Wiley), Embase (Elsevier), CINAHL (EBSCO), and ClinicalTrials.gov databases for observational or randomized studies published between 2002 and 2022 evaluating survival outcomes for patients with non-metastatic AA or DA who received systemic therapy and surgical resection. The data extracted included overall survival, progression-free survival, and pathologic response (PR) rate.From the 347 abstracts identified in this study, 29 reports were reviewed in full, and 15 were included in the final review. The selected studies published from 2007 to 2022 were retrospective. Eight were single-center studies; five used the National Cancer Database (NCDB); and two were European multicenter/national studies. Overall, no studies identified survival differences between NAT and upfront surgery (with or without adjuvant therapy). Two NCDB studies reported longer survival with NAT/AT than with surgery. Five single-center studies reported a significant portion of NAT patients who achieved PR, and one study identified major PR as an independent predictor of survival. Other outcomes associated with NAT included conversion from unresectable to resectable disease, reduced lymph node positivity, and decreased local recurrence rate.Evidence supporting the use of NAT in AA and DA is weak. No randomized studies exist, and observational data show mixed results. For patients with DA and AA, NAT appears safe, but better evidence is needed to understand the preferred multidisciplinary management of DA and AA periampullary malignancies.© 2023. Society of Surgical Oncology.