Sclafani Francesco, Morano Federica, Cunningham David, Baratelli Chiara, Kalaitzaki Eleftheria, Watkins David, Starling Naureen, Chau Ian, Rao Sheela
Department of Medicine, The Royal Marsden NHS Foundation Trust, London and Surrey, United Kingdom.
Department of Clinical Research & Development, The Royal Marsden NHS Foundation Trust, London and Surrey, United Kingdom.
Oncologist. 2017 Apr;22(4):402-408. doi: 10.1634/theoncologist.2016-0241. Epub 2017 Feb 16.
Although treatment of localized anal cancer (AC) is well established, very little evidence is available to inform the management of advanced tumors, and the prognosis of these patients remains poor. We have analyzed treatment pathways and outcomes of a single-institution series of advanced AC patients in order to provide insight into the management of this rare condition.
Inclusion criteria included epidermoid histology, inoperable locally recurrent or metastatic disease, and availability of full medical records. The primary objective was overall survival (OS). Prognostic factors were analyzed in univariate models.
Sixty-four patients (1997-2014) were included: 16 (25.0%) with inoperable locally advanced and 48 (75.0%) with metastatic tumors. Fifty-one (79.7%) received at least one line of chemotherapy; of these, 37% underwent multimodality treatment. A combination of a platinum agent plus a fluoropyrimidine was the most common first-line regimen (74.5%), with an objective response rate (ORR) of 34.4% (95% confidence interval [CI], 18.6%-53.2%). Paclitaxel-based chemotherapy was used in 15 patients as front-line or salvage treatment, and the overall ORR was 53.3% (95% CI, 26.6%-78.7%). Median progression-free survival (PFS) after first- and second-line chemotherapy was 5.8 (interquartile range [IQR], 2.8-7.6) and 3.2 (IQR, 2.5-7.1) months, respectively. Five-year OS in the overall population was 15% (95% CI, 7.0%-25.0%). Age ≤65 years and liver metastases were predictive of better PFS (hazard ratio [HR], 0.39; 95% CI, 0.16-0.97; = .04) and worse OS (HR, 2.25; 95% CI, 1.25-4.03; = .01), respectively.
A platinum agent plus a fluoropyrimidine and paclitaxel-based chemotherapy are active regimens for advanced AC. Clinical trials are needed to standardize treatment pathways, investigate the potential of novel therapeutics, and improve the poor prognosis of this rare condition. 2017;22:402-408 Because of the lack of randomized trials, the optimal management of advanced anal cancer is uncertain. Despite its retrospective analysis and relatively small sample size, this is the second largest study ever conducted in this setting, and, as such, it has the potential to serve as a valuable source of information for everyday clinical practice. These findings suggest that chemotherapy with a platinum agent plus a fluoropyrimidine or paclitaxel-containing regimens are reasonable treatment options for patients with inoperable locally recurrent or metastatic anal carcinoma.
尽管局部肛管癌（AC）的治疗方法已确立，但关于晚期肿瘤治疗的证据非常有限，这些患者的预后仍然很差。我们分析了单机构一系列晚期AC患者的治疗途径和结果，以便深入了解这种罕见疾病的管理。
纳入标准包括表皮样组织学、不可手术的局部复发或转移性疾病以及完整病历。主要目标是总生存期（OS）。在单变量模型中分析预后因素。
纳入64例患者（1997 - 2014年）：16例（25.0%）为不可手术的局部晚期患者，48例（75.0%）为转移性肿瘤患者。51例（79.7%）接受了至少一线化疗；其中，37%接受了多模式治疗。铂类药物加氟嘧啶联合方案是最常见的一线方案（74.5%），客观缓解率（ORR）为34.4%（95%置信区间[CI]，18.6% - 53.2%）。15例患者使用基于紫杉醇的化疗作为一线或挽救治疗，总体ORR为53.3%（95% CI，26.6% - 78.7%）。一线和二线化疗后的中位无进展生存期（PFS）分别为5.8个月（四分位间距[IQR]，2.8 - 7.6）和3.2个月（IQR，2.5 - 7.1）。总体人群的5年OS为15%（95% CI，7.0% - 25.0%）。年龄≤65岁和肝转移分别是PFS较好（风险比[HR]，0.39；95% CI，0.16 - 0.97；P = 0.04）和OS较差（HR，2.25；95% CI，1.25 - 4.03；P = 0.01）的预测因素。
铂类药物加氟嘧啶和基于紫杉醇的化疗是晚期AC的有效方案。需要进行临床试验以规范治疗途径，研究新型疗法的潜力，并改善这种罕见疾病的不良预后。2017；22：402 - 408由于缺乏随机试验，晚期肛管癌的最佳管理尚不确定。尽管其为回顾性分析且样本量相对较小，但这是在此背景下进行的第二大研究，因此，它有可能成为日常临床实践中有价值的信息来源。这些发现表明，铂类药物加氟嘧啶或含紫杉醇方案的化疗是不可手术的局部复发或转移性肛管癌患者合理的治疗选择。
