Division of Gastroenterology, Department of Internal Medicine, University of Ulsan College of Medicine, Asan Medical Centre, Seoul, Republic of Korea.
Division of Gastroenterology, Department of Internal Medicine, University of Ulsan College of Medicine, Asan Medical Centre, Seoul, Republic of Korea.
Eur J Cancer. 2014 May;50(7):1259-68. doi: 10.1016/j.ejca.2014.01.008. Epub 2014 Jan 30.
Hilar cholangiocarcinoma is an uncommon cancer and its overall incidence is increasing. Photodynamic therapy (PDT) has been proposed as palliative management for unresectable hilar cholangiocarcinoma (UHC). To date, little is known about the role of the addition of systemic chemotherapy to PDT for UHC. We performed a prospective, randomised, phase II trial to compare PDT plus S-1 and PDT alone for UHC.
Patients with UHC were randomly assigned (in a 1:1 ratio) to PDT plus S-1 or PDT alone. The primary end-point was overall survival. The secondary end-points were progression-free survival, complications, re-intervention rate and quality of life. This trial is registered with clinicalTrials.gov, number NCT00869635.
Between February 2009 and May 2012, we randomly assigned 21 patients to receive PDT plus S-1 and 22 to receive PDT alone. The UHC patients treated with PDT plus S-1 showed higher 1-year survival rate compared with the patients treated with PDT alone (76.2% versus 32%, P=0.003) and prolonged overall survival (median 17 months, 95% confidence interval [CI]: 12.6-21.4, versus 8 months, 95% CI: 6-10, P=0.005, hazard ratio [HR], 0.36; 95% CI: 0.17-0.75). Regarding the secondary end-points, PDT plus S-1 was associated with prolonged progression-free survival compared with PDT alone (median 10 months [95% CI: 4.1-16] versus 2 months [95% CI: 0.4-3.5], P=0.009 (HR for progression 0.39, 95% CI: 0.19-0.83). There were no differences in the number of PDT sessions, the frequency of cholangitis, overall adverse events or the quality of life in either group.
PDT plus S-1 was well tolerated and was associated with a significant improvement of overall survival and progression-free survival compared with PDT alone in patients with UHC. These findings warrant further clinical investigation of PDT plus S-1 in patients with UHC.
肝门部胆管癌是一种罕见的癌症，其总体发病率正在上升。光动力疗法（PDT）已被提议作为不可切除的肝门部胆管癌（UHC）的姑息治疗方法。迄今为止，对于将全身化疗联合 PDT 用于 UHC 的作用知之甚少。我们进行了一项前瞻性、随机、二期试验，比较 PDT 联合 S-1 与单独 PDT 治疗 UHC。
将 UHC 患者以 1:1 的比例随机分配（分配比例）至 PDT 联合 S-1 或单独 PDT 组。主要终点是总生存期。次要终点是无进展生存期、并发症、再次干预率和生活质量。本试验在 clinicalTrials.gov 上注册，编号为 NCT00869635。
2009 年 2 月至 2012 年 5 月，我们随机分配 21 例患者接受 PDT 联合 S-1 治疗，22 例患者接受单独 PDT 治疗。与单独 PDT 组相比，接受 PDT 联合 S-1 治疗的 UHC 患者的 1 年生存率更高（76.2% vs. 32%，P=0.003），总生存期也更长（中位 17 个月，95%置信区间[CI]：12.6-21.4 个月 vs. 8 个月，95%CI：6-10 个月，P=0.005，风险比[HR]，0.36；95%CI：0.17-0.75）。关于次要终点，与单独 PDT 相比，PDT 联合 S-1 可延长无进展生存期（中位 10 个月[95%CI：4.1-16] vs. 2 个月[95%CI：0.4-3.5]，P=0.009（进展的 HR 为 0.39，95%CI：0.19-0.83）。两组的 PDT 次数、胆管炎的频率、总体不良事件或生活质量均无差异。
PDT 联合 S-1 耐受良好，与单独 PDT 相比，可显著提高 UHC 患者的总生存率和无进展生存率。这些发现为进一步研究 PDT 联合 S-1 在 UHC 患者中的应用提供了依据。
