Department of Internal Medicine, Medical Oncology, Kyorin University School of Medicine, Shinkawa, Mitaka, Tokyo, Japan.
Crit Rev Oncol Hematol. 2011 Oct;80(1):31-9. doi: 10.1016/j.critrevonc.2010.10.009. Epub 2010 Nov 19.
There had been no standard chemotherapy established for advanced biliary tract cancer (BTC) until 2009, when the combination of cisplatin and gemcitabine (GC) was adopted as a first line standard chemotherapy option based on the results from two randomized studies: ABC-02, a UK investigator-initiated trial and the largest randomized phase III study in this tumor type with 410 patients; and BT22, a Japanese, industry-sponsored, randomized phase II study with 83 patients. In this review, investigators from both studies collaborated to compare protocols, patient characteristics, and outcomes of both studies including sub-analyses of study results. Although both studies showed GC combination therapy to be more effective than monotherapy, a detailed comparison revealed disparities between efficacy and safety end-points between the studies, which did not necessarily arise from different populations but from differences in protocol design. This review provides clinicians with insights for advanced BTC clinical study design and interpretation of historical studies.
直到 2009 年，对于晚期胆道癌（BTC）还没有确立标准的化疗方案，当时基于两项随机研究的结果，顺铂联合吉西他滨（GC）被采纳为一线标准化疗选择：ABC-02，一项英国研究者发起的试验，也是该肿瘤类型中最大的随机 III 期研究，纳入了 410 例患者；以及 BT22，一项日本的、由产业赞助的、随机的 II 期研究，纳入了 83 例患者。在这项综述中，来自这两项研究的研究者合作比较了两项研究的方案、患者特征和结果，包括对研究结果的亚组分析。尽管这两项研究均显示 GC 联合治疗比单药治疗更有效，但详细比较揭示了这两项研究在疗效和安全性终点之间的差异，这些差异并非源于不同人群，而是源于方案设计的不同。本综述为临床医生提供了有关晚期 BTC 临床研究设计和解读历史研究的见解。
