Effect of deferred or no treatment with ursodeoxycholic acid in patients with early primary biliary cholangitis. Issue 9 (7th March 2018)

• Record Type: Journal Article
• Authors: Tanaka, Atsushi; Hirohara, Junko; Nakano, Toshiaki; Yagi, Minami; Namisaki, Tadashi; Yoshiji, Hitoshi; Nakanuma, Yasuni; Takikawa, Hajime
• Is Part Of: Hepatology research. Volume 48:Issue 9(2018)
• Page Start: 727
• Page End: 734
• Publication Date: 2018-03-07
• Subjects: alkaline phosphatase -- asymptomatic -- outcome -- staging -- ursodeoxycholic acid; Liver -- Diseases -- Periodicals; Liver Diseases -- Periodicals; Foie -- Maladies -- Périodiques; 616.362
• DOI: 10.1111/hepr.13068
• Languages: English
• ISSNs: 1386-6346

Abstract

Abstract : Aim: As primary biliary cholangitis (PBC) is a heterogeneous disease, we hypothesized that there is a population of patients with early PBC who do not require prompt treatment with ursodeoxycholic acid (UDCA). In this study, we analyzed data from a large‐scale PBC cohort in Japan, and retrospectively investigated whether outcomes of early PBC patients were affected with prompt or deferred/no UDCA treatment. Methods: We defined early PBC as asymptomatic, serum alkaline phosphatase <1.67‐fold the upper limit of normal, normal bilirubin, and histological stages I–II at presentation. We compared the outcomes of early PBC patients between the treatment regimens; prompt treatment group (UDCA was initiated within 1 year after diagnosis) and deferred/no treatment group (UDCA initiated >1 year after diagnosis or never initiated). Furthermore, we examined the outcomes of early PBC patients alternatively defined only with symptomatology and biochemistry. Results: We identified 562 early PBC patients (prompt: n = 509; deferred/no treatment: n = 53). Incidence rates (per 1000 patient‐years) for liver‐related mortality or liver transplantation and decompensating events were 0.5 and 5.4, respectively, in the prompt treatment group, and 0 and 8.7, respectively, in the deferred/no treatment group. Multivariate analyses showed that age and bilirubin were significantly associated with developing decompensating events, whereas the prompt and deferred/no treatments were not. We obtained similar results in early PBC patients defined without histological examination. Conclusions: We showed that deferred/no treatment for early PBC patients did not affect the outcomes. This study provides a rationale for a future prospective, randomized study.