Do Hyun Park1, Sang Soo Lee2, So Eun Park2, Jae Lyun Lee3, Jun Ho Choi2, Hee Jung Choi2, Ji Woong Jang2, Hyoung Jung Kim4, Jun Bum Eum2, Dong-Wan Seo2, Sung Koo Lee2, Myung-Hwan Kim2, Jung Bok Lee5. 1. Division of Gastroenterology, Department of Internal Medicine, University of Ulsan College of Medicine, Asan Medical Centre, Seoul, Republic of Korea. Electronic address: dhpark@amc.seoul.kr. 2. Division of Gastroenterology, Department of Internal Medicine, University of Ulsan College of Medicine, Asan Medical Centre, Seoul, Republic of Korea. 3. Division of Oncology, Department of Internal Medicine, University of Ulsan College of Medicine, Asan Medical Centre, Seoul, Republic of Korea. 4. Department of Radiology, University of Ulsan College of Medicine, Asan Medical Centre, Seoul, Republic of Korea. 5. Department of Clinical Epidemiology and Biostatistics, University of Ulsan College of Medicine, Asan Medical Centre, Seoul, Republic of Korea.
Abstract
BACKGROUND: 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. METHODS: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. FINDINGS: 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. INTERPRETATIONS: 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.
RCT Entities:
BACKGROUND: 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. METHODS: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. FINDINGS: 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. INTERPRETATIONS: 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.
Authors: Werner Dolak; Hubert Schwaighofer; Brigitte Hellmich; Bernhard Stadler; Georg Spaun; Wolfgang Plieschnegger; Arnold Hebenstreit; Jutta Weber-Eibel; Franz Siebert; Klaus Emmanuel; Peter Knoflach; Michael Gschwantler; Wolfgang Vogel; Michael Trauner; Andreas Püspök Journal: United European Gastroenterol J Date: 2016-07-07 Impact factor: 4.623
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