Troester Alexander, Parikh Romil, Southwell Bronwyn, Ester Elizabeth, Sultan Shahnaz, Greeno Edward, Arsoniadis Elliot, Church Timothy R, Wilt Timothy, Butler Mary, Goffredo Paolo
Department of Surgery, University of Minnesota, Minneapolis, MN, USA.
School of Public Health, University of Minnesota, Minneapolis, MN, USA.
J Natl Cancer Inst. 2025 Feb 1;117(2):240-252. doi: 10.1093/jnci/djae195.
We sought to assess the effectiveness and harms of initial treatment strategies for stage I through III anal squamous cell anal cancer.
We searched MEDLINE, Embase, and Cochrane Central Register of Controlled Trials between January 1, 2000, and March 2024, for randomized controlled trials and nonrandomized studies of interventions comparing initial treatment strategies. Individual study risk of bias and overall strength of evidence were evaluated for a prespecified outcome list using standardized methods.
We identified 33 eligible studies and extracted data. Six were deemed low to moderate risk of bias. Compared with radiation therapy alone, chemoradiation therapy (CRT) with 5-fluorouracil (5-FU) and mitomycin C probably shows a benefit in locoregional failure, disease-specific survival, and colostomy-free survival (moderate strength of evidence) yet may result in greater overall and acute hematological toxicity, with no difference in late harms (low strength of evidence). CRT with 5-FU plus mitomycin C may show a benefit in locoregional failure, disease-specific survival, and colostomy-free survival rates compared with 5-FU alone (low strength of evidence). CRT with 5-FU plus cisplatin vs 5-FU plus mitomycin C probably results in no differences in several effectiveness outcomes or overall acute or late harms and probably increases hematological toxicity with mitomycin C (moderate strength of evidence). Compared with CRT using capecitabine plus mitomycin C, CRT with capecitabine plus mitomycin C and paclitaxel may improve overall survival, disease-specific survival, and colostomy-free survival yet cause more acute harms (low strength of evidence). Evidence was insufficient for remaining comparisons.
CRT with 5-FU plus mitomycin C or 5-FU plus cisplatin is likely more effective yet incurs greater acute hematological toxicity than radiation therapy alone or single-agent CRT. Adding paclitaxel to capecitabine plus mitomycin C may increase treatment efficacy and toxicity. Evidence is insufficient comparing posttreatment surveillance strategies and patient-reported outcomes, highlighting research opportunities.
我们试图评估I期至III期肛管鳞状细胞癌初始治疗策略的有效性和危害。
我们检索了2000年1月1日至2024年3月期间的MEDLINE、Embase和Cochrane对照试验中央登记库，以查找比较初始治疗策略的随机对照试验和干预措施的非随机研究。使用标准化方法对预先指定的结局列表评估个体研究的偏倚风险和总体证据强度。
我们确定了33项符合条件的研究并提取了数据。六项被认为偏倚风险低至中度。与单纯放疗相比，含5-氟尿嘧啶（5-FU）和丝裂霉素C的放化疗（CRT）可能在局部区域失败、疾病特异性生存和无结肠造口生存方面显示出益处（证据强度中等），但可能导致更高的总体和急性血液学毒性，晚期危害无差异（证据强度低）。与单独使用5-FU相比，含5-FU加丝裂霉素C的CRT可能在局部区域失败、疾病特异性生存和无结肠造口生存率方面显示出益处（证据强度低）。含5-FU加顺铂的CRT与含5-FU加丝裂霉素C的CRT相比，在几个有效性结局或总体急性或晚期危害方面可能没有差异，并且含丝裂霉素C的CRT可能增加血液学毒性（证据强度中等）。与使用卡培他滨加丝裂霉素C的CRT相比，含卡培他滨加丝裂霉素C和紫杉醇的CRT可能改善总体生存、疾病特异性生存和无结肠造口生存，但会导致更多急性危害（证据强度低）。其余比较的证据不足。
含5-FU加丝裂霉素C或5-FU加顺铂的CRT可能比单纯放疗或单药CRT更有效，但会导致更大的急性血液学毒性。在卡培他滨加丝裂霉素C中添加紫杉醇可能会增加治疗效果和毒性。比较治疗后监测策略和患者报告结局的证据不足，突出了研究机会。
