JGH open : an open access journal of gastroenterology and hepatology · 2019
Evaluation of the United Kingdom-primary biliary cholangitis and global primary biliary cholangitis group prognostic models for primary biliary cholangitis patients treated with ursodeoxycholic acid in the U.S. population.
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Alomari Mohammad, Covut Fahrettin, Al Momani Laith, Chadalavada Pravallika, Hitawala Asif, Young Mark F, Romero-Marrero Carlos
Аннотация
BACKGROUND AND AIM: The United Kingdom-primary biliary cholangitis (UK-PBC) and global primary biliary cholangitis group (GLOBE) prognostic models have been recently developed to predict long-term outcomes in primary biliary cholangitis (PBC). However, these predictive scores have not yet been well evaluated in the U.S. population.
METHODS: We retrospectively reviewed newly diagnosed PBC patients at the Cleveland Clinic between November 1998 and February 2017. Adverse events were defined as liver transplantation, liver-related mortality, and all-cause mortality. Transplant-free survival (TFS) was estimated using the Kaplan-Meier method. Predictive performances of all prognostic models were evaluated using the C-statistic.
RESULTS: We identified 352 patients who used ursodeoxycholic acid therapy. Of them, 311 (88.4%) only had PBC, while 41 (11.6%) were diagnosed with PBC-autoimmune hepatitis overlap. A total of 22 (6%), 47 (13%), and 55 (16%) patients had adverse events within 5, 10, and 15 years after diagnosis, respectively. In patients with PBC only, the C-statistic in predicting 15-year adverse events was 0.75 per GLOBE compared to 0.74 per UK-PBC (P = 0.94), 0.73 per Rotterdam (P = 0.44), 0.66 per Barcelona (P = 0.004), 0.65 per Paris 1 (P = 0.005), 0.62 per Paris 2 (P < 0.0001), 0.60 per Toronto (P < 0.0001), and 0.60 per Mayo (P < 0.0001) scores. Median follow-up was 9.2 years. Ten-year TFS for patients who had optimal versus suboptimal treatment response was 92 versus 74% per Paris 1 (P < 0.0001), 95 versus 79% per Paris 2 (P = 0.0002), 93 versus 65% per Barcelona (P < 0.0001), and 96 versus 68% per Rotterdam (P < 0.0001) risk scores, respectively.
CONCLUSION: In our cohort of PBC patients, the UK-PBC and GLOBE scores were both accurate and reasonably valid prognostic models in the U.S. population.
METHODS: We retrospectively reviewed newly diagnosed PBC patients at the Cleveland Clinic between November 1998 and February 2017. Adverse events were defined as liver transplantation, liver-related mortality, and all-cause mortality. Transplant-free survival (TFS) was estimated using the Kaplan-Meier method. Predictive performances of all prognostic models were evaluated using the C-statistic.
RESULTS: We identified 352 patients who used ursodeoxycholic acid therapy. Of them, 311 (88.4%) only had PBC, while 41 (11.6%) were diagnosed with PBC-autoimmune hepatitis overlap. A total of 22 (6%), 47 (13%), and 55 (16%) patients had adverse events within 5, 10, and 15 years after diagnosis, respectively. In patients with PBC only, the C-statistic in predicting 15-year adverse events was 0.75 per GLOBE compared to 0.74 per UK-PBC (P = 0.94), 0.73 per Rotterdam (P = 0.44), 0.66 per Barcelona (P = 0.004), 0.65 per Paris 1 (P = 0.005), 0.62 per Paris 2 (P < 0.0001), 0.60 per Toronto (P < 0.0001), and 0.60 per Mayo (P < 0.0001) scores. Median follow-up was 9.2 years. Ten-year TFS for patients who had optimal versus suboptimal treatment response was 92 versus 74% per Paris 1 (P < 0.0001), 95 versus 79% per Paris 2 (P = 0.0002), 93 versus 65% per Barcelona (P < 0.0001), and 96 versus 68% per Rotterdam (P < 0.0001) risk scores, respectively.
CONCLUSION: In our cohort of PBC patients, the UK-PBC and GLOBE scores were both accurate and reasonably valid prognostic models in the U.S. population.