Background: Appendiceal adenocarcinoma is a rare malignancy, and data guiding its systemic treatment in metastatic settings are limited. This study aimed to determine the clinical outcomes, treatment efficacy, biomarkers, and prognostic factors in patients with metastatic or unresectable appendiceal adenocarcinoma receiving palliative chemotherapy. Methods: We retrospectively reviewed patients with metastatic appendiceal adenocarcinoma who received first-line palliative systemic chemotherapy at the Peter MacCallum Cancer Centre between January 2015 and December 2024. Results: Of the 40 patients included, fluoropyrimidine-based doublet regimens were most commonly used (82.5%) in first-line setting, achieving an objective response rate of 39.4%. Median overall survival (OS) was 21.6 months, and median first-line progression-free survival (PFS) was 8.9 months. 22 patients (55.0%) received second-line treatment. Median OS and PFS were 21.6 and 8.9 months, respectively, among patients treated with oxaliplatin-based doublet regimens, and 66.4 and 10.8 months, respectively, among those treated with irinotecan-based doublet regimens. Molecular biomarker testing was performed in 35 patients (87.5%).KRASandNRASmutations were identified in 68.6% and 2.9% of tested patients, respectively. Factors associated with poorer OS included male sex, elevated carcinoembryonic antigen levels, and overweight status. Bevacizumab use was not clearly associated with survival. Conclusions: Palliative systemic chemotherapy, particularly fluoropyrimidine-based doublet regimens, appears to be a reasonable and effective treatment option for patients with advanced appendiceal adenocarcinoma. Although this study was underpowered for formal comparison, the numerically longer OS and PFS of irinotecan-based regimens are hypothesis-generating and support further prospective evaluation. Molecular profiling emphasizes the need for personalized targeted therapeutic strategies. The identified prognostic factors may help guide risk stratification and patient counseling for treatment planning.
背景：阑尾腺癌是一种罕见的恶性肿瘤，关于其转移性病变全身治疗的数据有限。本研究旨在评估接受姑息化疗的转移性或不可切除阑尾腺癌患者的临床结局、治疗效果、生物标志物及预后因素。方法：我们回顾性分析了2015年1月至2024年12月期间在彼得·麦卡勒姆癌症中心接受一线姑息全身化疗的转移性阑尾腺癌患者。结果：在纳入的40例患者中，一线治疗最常采用以氟尿嘧啶为基础的双药方案（82.5%），客观缓解率为39.4%。中位总生存期为21.6个月，一线治疗中位无进展生存期为8.9个月。22例患者（55.0%）接受了二线治疗。在以奥沙利铂为基础的双药方案治疗患者中，中位总生存期和无进展生存期分别为21.6个月和8.9个月；而在以伊立替康为基础的双药方案治疗患者中，分别为66.4个月和10.8个月。35例患者（87.5%）接受了分子生物标志物检测，其中分别有68.6%和2.9%的患者检出KRAS和NRAS突变。与较差总生存期相关的因素包括男性、癌胚抗原水平升高和超重状态。贝伐珠单抗的使用与生存期无明确关联。结论：姑息全身化疗，特别是以氟尿嘧啶为基础的双药方案，似乎是晚期阑尾腺癌患者合理且有效的治疗选择。尽管本研究样本量不足以进行正式比较，但伊立替康方案在数值上更长的总生存期和无进展生存期具有假设生成意义，支持进一步的前瞻性评估。分子谱分析强调了制定个体化靶向治疗策略的必要性。已确定的预后因素可能有助于指导风险分层和患者治疗规划的咨询。
肿瘤(癌症)患者之家