Minami Yagi1, Kosuke Matsumoto1, Atsumasa Komori2, Masanori Abe3, Naoaki Hashimoto4, Mie Inao5, Tadashi Namisaki6, Kazuhito Kawata7, Masashi Ninomiya8, Hideki Fujii9, Atsushi Takahashi10, Jong-Hon Kang11, Masaaki Takamura12, Mie Arakawa13, Satoru Joshita14, Ken Sato15, Jun Itakura16, Takako Nomura17, Keisuke Kakisaka18, Akira Kaneko19, Yamato Tamura1, Ryo Miura1, Mitsuhiko Aiso1, Toshihiko Arizumi1, Yoshinari Asaoka1, Kentaro Kikuchi20, Yasuhiro Takikawa18, Tsutomu Masaki17, Takeji Umemura14, Akira Honda21, Hiromasa Ohira10, Norifumi Kawada9, Hitoshi Yoshiji6, Satoshi Mochida5, Hajime Takikawa22, Atsushi Tanaka1. 1. Department of Medicine, Teikyo University School of Medicine, Tokyo, Japan. 2. Clinical Research Center, National Hospital Organization (NHO) Nagasaki Medical Center, Nagasaki, Japan. 3. Department of Gastroenterology and Metabology, Ehime University Graduate School of Medicine, Ehime, Japan. 4. Department of Gastroenterology, Tokyo Teishin Hospital, Tokyo, Japan. 5. Department of Gastroenterology and Hepatology, Faculty of Medicine, Saitama Medical University, Saitama, Japan. 6. Third Department of Internal Medicine, Nara Medical University, Nara, Japan. 7. Hepatology Division, Department of Internal Medicine, Hamamatsu University School of Medicine, Hamamatsu, Japan. 8. Division of Gastroenterology, Tohoku University Graduate School of Medicine, Sendai, Japan. 9. Department of Hepatology, Graduate School of Medicine, Osaka City University, Osaka, Japan. 10. Department of Gastroenterology, Fukushima Medical University School of Medicine, Fukushima, Japan. 11. Center for Gastroenterology, Teine Keijinkai Hospital, Sapporo, Japan. 12. Department of Gastroenterology and Hepatology, Niigata University Graduate School of Medical and Dental Sciences, Niigata, Japan. 13. Department of Gastroenterology, Faculty of Medicine, Oita University, Yufu City, Japan. 14. Division of Hepatology and Gastroenterology, Department of Medicine, Shinshu University School of Medicine, Matsumoto, Japan. 15. Department of Gastroenterology and Hepatology, Gunma University Graduate School of Medicine, Maebashi, Japan. 16. Department of Gastroenterology and Hepatology, Musashino Red Cross Hospital, Tokyo, Japan. 17. Department of Gastroenterology and Neurology, Kagawa University School of Medicine, Kagawa, Japan. 18. Division of Hepatology, Department of Internal Medicine, Iwate Medical University, Morioka, Japan. 19. Department of Gastroenterology, Minoh City Hospital, Osaka, Japan. 20. The Fourth Department of Internal Medicine, Teikyo University Mizonokuchi Hospital, Tokyo, Japan. 21. Division of Gastroenterology and Hepatology, Department of Internal Medicine, Tokyo Medical University Ibaraki Medical Center, Ibaraki, Japan. 22. Faculty of Medical Technology, Teikyo University, Tokyo, Japan.
Abstract
BACKGROUND/ PURPOSE: Although ursodeoxycholic acid (UDCA) is a first-line treatment for primary biliary cholangitis (PBC), 20%-30% of patients with PBC exhibit an incomplete response to UDCA. Recently, the UDCA Response Score was proposed for predicting response to UDCA using pretreatment parameters in patients with PBC. We aimed to validate the UDCA Response Score in Japanese patients with PBC. METHODS: Registry data of Japanese patients (n = 873) were collected. Patients with data on all clinical parameters required for calculating the UDCA Response Score were selected. The endpoint was UDCA response, defined as alkaline phosphatase <1.67 times the upper limit of the normal value after 12 months of UDCA treatment. RESULTS: All parameters were available in 804 patients (male/female = 120/684, age 58.9 [interquartile range 51.1-66.9] years). Bezafibrate was commenced within 12 months of UDCA in 78 patients (9.7%) because of the lack of an early response. We found that the endpoint was not reached in these 78 patients, and the area under the receiver operating characteristic curve (AUROC) of the score was 0.74 (95% confidence interval [CI] 0.70-0.79). The AUROC was 0.77 (95% CI 0.70-0.83) in patients undergoing UDCA monotherapy (n = 726). Finally, the AUROC of the modified UDCA Response Score using only data from the treatment start date was 0.80 (95% CI 0.70-0.90) in patients receiving a combination therapy of UDCA and bezafibrate (n = 160). CONCLUSION: The validity of the UDCA Response Score was acceptable in Japanese patients; this score will be informative in patients treated with a combination therapy of UDCA and bezafibrate.
BACKGROUND/ PURPOSE: Although ursodeoxycholic acid (UDCA) is a first-line treatment for primary biliary cholangitis (PBC), 20%-30% of patients with PBC exhibit an incomplete response to UDCA. Recently, the UDCA Response Score was proposed for predicting response to UDCA using pretreatment parameters in patients with PBC. We aimed to validate the UDCA Response Score in Japanese patients with PBC. METHODS: Registry data of Japanese patients (n = 873) were collected. Patients with data on all clinical parameters required for calculating the UDCA Response Score were selected. The endpoint was UDCA response, defined as alkaline phosphatase <1.67 times the upper limit of the normal value after 12 months of UDCA treatment. RESULTS: All parameters were available in 804 patients (male/female = 120/684, age 58.9 [interquartile range 51.1-66.9] years). Bezafibrate was commenced within 12 months of UDCA in 78 patients (9.7%) because of the lack of an early response. We found that the endpoint was not reached in these 78 patients, and the area under the receiver operating characteristic curve (AUROC) of the score was 0.74 (95% confidence interval [CI] 0.70-0.79). The AUROC was 0.77 (95% CI 0.70-0.83) in patients undergoing UDCA monotherapy (n = 726). Finally, the AUROC of the modified UDCA Response Score using only data from the treatment start date was 0.80 (95% CI 0.70-0.90) in patients receiving a combination therapy of UDCA and bezafibrate (n = 160). CONCLUSION: The validity of the UDCA Response Score was acceptable in Japanese patients; this score will be informative in patients treated with a combination therapy of UDCA and bezafibrate.
Authors: Jakub Gazda; Martin Janicko; Sylvia Drazilova; Ivica Grgurevic; Tajana Filipec Kanizaj; Tomas Koller; Beatrica Bodorovska; Maja Mijic; Ivana Mikolasevic; Ivana Knezevic Stromar; Branislav Kucinsky; Matej Gazda; Peter Jarcuska Journal: Can J Gastroenterol Hepatol Date: 2021-06-22