日本真实世界转移性胆道癌患者的治疗结局：日本胆道癌研究组真实世界数据项目 04（TREAD 04）。

Real-world treatment outcomes of metastatic biliary tract cancer patients in Japan: the Tokushukai REAl-world data project 04 (TREAD 04).

机构信息

Department of General Surgery, Shonankamakura General Hospital, Kamakura, Japan.

Department of Medical Oncology and Hematology, Kobe University Graduate School of Medicine, Kobe, Japan.

出版信息

Jpn J Clin Oncol. 2024 Jan 7;54(1):70-80. doi: 10.1093/jjco/hyad133.

DOI:10.1093/jjco/hyad133

PMID:37801431

Abstract

OBJECTIVES

To investigate temporal trends in treatment patterns and prognostic factors for overall survival in patients with metastatic biliary tract cancer.

METHODS

From the Tokushukai REAl-world Data project, we identified 945 patients with metastatic biliary tract cancer treated with gemcitabine, tegafur/gimeracil/oteracil, gemcitabine plus cisplatin, gemcitabine plus tegafur/gimeracil/oteracil or gemcitabine plus cisplatin and tegafur/gimeracil/oteracil between April 2010 and March 2022. Stratified/conventional Cox regression analyses were conducted to examine the association between overall survival and patient- and tumour-related factors, study period, hospital volume, hospital type and first-line chemotherapy regimen. Using inverse probability of treatment weighting with propensity scores, overall survival was also compared between monotherapy and combination therapy groups.

RESULTS

We enrolled 366 patients (199 men; median age, 72 years). Over a median follow-up of 5.2 months, the median overall survival was 7.0 months (95% confidence interval 6.2-9.0), and the median time to treatment failure was 3.5 months (95% confidence interval 3.1-4.5). Median overall survival and time to treatment failure for gemcitabine/tegafur-gimeracil-oteracil/gemcitabine plus cisplatin/gemcitabine plus tegafur-gimeracil-oteracil/gemcitabine plus cisplatin and tegafur-gimeracil-oteracil regimen were 6.2/6.6/7.9/16.2/15.1 and 2.8/3.4/4.1/15.3/7.4 months, respectively. Primary disease site, previous surgery, previous endoscopic procedures and hospital type were identified as significant prognostic factors. Inverse probability of treatment weighting analysis demonstrated that combination therapy had a significantly better prognosis than monotherapy (hazard ratio 0.61, 95% confidence interval 0.43-0.88, P = 0.006).

CONCLUSIONS

Our real-world data analysis showed that standard care for metastatic biliary tract cancer is widely used in hospitals throughout Japan and verified the survival benefits of combination therapy over monotherapy observed in prior clinical trials.

CLINICAL TRIAL NUMBER

UMIN000050590 (http://www.umin.ac.jp/ctr/index.htm).

摘要

目的

研究转移性胆道癌患者整体生存的治疗模式和预后因素的时间趋势。

方法

我们从 Tokushukai REAl-world Data 项目中确定了 945 名转移性胆道癌患者，他们接受了吉西他滨、替加氟/替加氟/奥替拉西/吉西他滨、吉西他滨联合顺铂、吉西他滨联合替加氟/替加氟/奥替拉西或吉西他滨联合顺铂和替加氟/替加氟/奥替拉西治疗。采用分层/常规 Cox 回归分析，研究患者和肿瘤相关因素、研究期间、医院容量、医院类型和一线化疗方案与总生存之间的关系。采用倾向评分逆概率治疗加权法比较单药治疗组和联合治疗组的总生存率。

结果

共纳入 366 名患者（199 名男性；中位年龄 72 岁）。中位随访 5.2 个月后，中位总生存期为 7.0 个月（95%置信区间 6.2-9.0），中位治疗失败时间为 3.5 个月（95%置信区间 3.1-4.5）。吉西他滨/替加氟-替加氟-奥替拉西/吉西他滨联合顺铂/吉西他滨联合替加氟-替加氟-奥替拉西/吉西他滨联合顺铂和替加氟-替加氟-奥替拉西方案的中位总生存期和治疗失败时间分别为 6.2/6.6/7.9/16.2/15.1 和 2.8/3.4/4.1/15.3/7.4 个月。原发疾病部位、既往手术、既往内镜治疗和医院类型是显著的预后因素。逆概率治疗加权分析表明，联合治疗的预后明显优于单药治疗（风险比 0.61，95%置信区间 0.43-0.88，P=0.006）。