Discussion
OCA, PBC
DISEASE PROGRESSION, PBC
Stratified therapy is an important strategy in the clinical management of PBC patients. Several agents, such as obeticholic acid (OCA), fibrates, and budesonide proved to be effective for patients with insufficient UDCA response [Notably, up to 40% of PBC patients will have a suboptimal biochemical response to UDCA, as assessed by binary response criteria and/or prognostic models [The disease progression of PBC patients varies greatly. Our data has shown the 29/71 (41%) patients with adverse outcome had an end-point within 2 years after initial diagnosis, and 82% patients in training cohort and 91% patients in validation cohort can be accurately categorized as non-responders by Xi’an criterion, which is much higher than Barcelona, Paris-I, Paris-II, Rotterdam, Rochester-II and Ehime. In a recent study by Zhang et al. [In 2017, the EASL Clinical Practice Guidelines proposed various criteria as tools to select patients for second-line therapies and for a better design of clinical trials in PBC [Zhang et al. proposed that previously published criteria, including Paris, Barcelona, Toronto, and Ehime, applied at 3 and 6 months significantly discriminated high-risk patients [However, this study had some limitations. Firstly, it was a single-center, retrospective study. Further validation in multicenter studies with a larger cohort of patients is warranted in future. Secondly, the mean follow-up period was 5 years and relatively short. Noting the mean period of developing adverse outcome which is 3.6 years, we submit that an average of 5 years of follow-up time is sufficient to forecast the prognosis of PBC patients.In summary, we have designed and validated a new early criterion for distinguishing high-risk PBC patients in a Chinese population for the first time. Our data indicated that PBC patients with ALP ≤ 2.5 × ULN, AST ≤ 2 × ULN, and TBIL ≤ 1 × ULN (Xi’an criterion) after 1 month UDCA treatment were likely to have better prognosis. For rapidly progressive patients, the Xi’an criterion is highly reliable and has an overall excellent predictive capacity than other published criteria. In addition, Xi’an criterion provides significant prognostic information in both early- and late-stage PBC and provides an additional comprehensive platform in the clinical evaluation of PBC patients. Most importantly, it can be readily applied in the rapid identification of PBC patients who require additional therapeutic approaches.