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Prognostic Factors for Transplant-Free Survival and Validation of Prognostic Models in Chinese Patients with Primary Biliary Cholangitis Receiving Ursodeoxycholic Acid.

ABSTRACT: We aimed to validate the prognostic models for primary biliary cholangitis (PBC) in Chinese patients receiving ursodeoxycholic acid (UCDA), and to compare their performances in predicting the long-term survival.Chinese patients with PBC from a tertiary center were identified via electronic search of hospital medical registry. Risk factors associated with adverse events (liver transplantation or death from liver-related causes including hepatocellular carcinoma (HCC) and liver decompensation) were determined. Transplant-free survival was defined as survival free of liver-related death or transplantation.Of the 144 patients, 41 (28.5%) had baseline cirrhosis. The median age at diagnosis was 57.8 years. During a median follow-up of 7.0 years, 40 patients died (21 liver-related; 19 non-liver-related), 12 developed HCC, and 10 underwent transplantations. The 5-, 10-, and 15-year transplant-free survival probabilities were 91.0%, 78.1%, and 58.9%, respectively. Independent risk factors for adverse events were increasing age (hazard ratio (HR) 1.05), cirrhosis (HR 8.53), and suboptimal treatment response (HR 3.06). Aspartate aminotransferase/platelet ratio index at 1 year (APRI-r1) in combination with treatment response optimized the risk stratification. The performances of the GLOBE, UK-PBC scores, Rotterdam criteria, and APRI-r1 were comparable in predicting adverse events. The area under receiver operating curves within 5, 10, and 15 years were as follows-GLOBE score: 0.83, 0.85, and 0.85, respectively; UK-PBC score: 0.89, 0.83, and 0.79, respectively; Rotterdam criteria: 0.82, 0.76, and 0.80, respectively; APRI-r1: 0.80, 0.83, and 0.77, respectively.The UK-PBC, GLOBE scores, Rotterdam criteria, and APRI-r1 had good and comparable prognostic prediction values for Chinese PBC patients receiving UCDA.

SUBMITTER: Cheung KS

PROVIDER: S-EPMC5518946 | biostudies-literature | 2017 Jun

REPOSITORIES: biostudies-literature

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Prognostic Factors for Transplant-Free Survival and Validation of Prognostic Models in Chinese Patients with Primary Biliary Cholangitis Receiving Ursodeoxycholic Acid.

Cheung Ka-Shing KS Seto Wai-Kay WK Fung James J Lai Ching-Lung CL Yuen Man-Fung MF

Clinical and translational gastroenterology 20170622 6

<h4>Objectives</h4>We aimed to validate the prognostic models for primary biliary cholangitis (PBC) in Chinese patients receiving ursodeoxycholic acid (UCDA), and to compare their performances in predicting the long-term survival.<h4>Methods</h4>Chinese patients with PBC from a tertiary center were identified via electronic search of hospital medical registry. Risk factors associated with adverse events (liver transplantation or death from liver-related causes including hepatocellular carcinoma ...[more]

PMID: 28640288

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Project description:BackgroundTreatment guidelines recommend a stepwise approach to primary biliary cholangitis: all patients begin treatment with ursodeoxycholic acid (UDCA) monotherapy and those with an inadequate biochemical response after 12 months are subsequently considered for second-line therapies. However, as a result, patients at the highest risk can wait the longest for effective treatment. We determined whether UDCA response can be accurately predicted using pretreatment clinical parameters.MethodsWe did logistic regression analysis of pretreatment variables in a discovery cohort of patients in the UK with primary biliary cholangitis to derive the best-fitting model of UDCA response, defined as alkaline phosphatase less than 1·67 times the upper limit of normal (ULN), measured after 12 months of treatment with UDCA. We validated the model in an external cohort of patients with primary biliary cholangitis and treated with UDCA in Italy. Additionally, we assessed correlations between model predictions and key histological features, such as biliary injury and fibrosis, on liver biopsy samples.Findings2703 participants diagnosed with primary biliary cholangitis between Jan 1, 1998, and May 31, 2015, were included in the UK-PBC cohort for derivation of the model. The following pretreatment parameters were associated with lower probability of UDCA response: higher alkaline phosphatase concentration (p<0·0001), higher total bilirubin concentration (p=0·0003), lower aminotransferase concentration (p=0·0012), younger age (p<0·0001), longer interval from diagnosis to the start of UDCA treatment (treatment time lag, p<0·0001), and worsening of alkaline phosphatase concentration from diagnosis (p<0·0001). Based on these variables, we derived a predictive score of UDCA response. In the external validation cohort, 460 patients diagnosed with primary biliary cholangitis were treated with UDCA, with follow-up data until May 31, 2016. In this validation cohort, the area under the receiver operating characteristic curve for the score was 0·83 (95% CI 0·79-0·87). In 20 liver biopsy samples from patients with primary biliary cholangitis, the UDCA response score was associated with ductular reaction (r=-0·556, p=0·0130) and intermediate hepatocytes (probability of response was 0·90 if intermediate hepatocytes were absent vs 0·51 if present).InterpretationWe have derived and externally validated a model based on pretreatment variables that accurately predicts UDCA response. Association with histological features provides face validity. This model provides a basis to explore alternative approaches to treatment stratification in patients with primary biliary cholangitis.FundingUK Medical Research Council and University of Milan-Bicocca.

Project description:IntroductionAbout one-third of primary biliary cholangitis (PBC) patients do not exhibit complete response to ursodeoxycholic acid (UDCA). Some of these patients were reported to benefit from the combination therapy of fibrates and UDCA, but more clinical evidence is required. In this study, we conducted a randomized, controlled trial on the safety and efficacy of fenofibrate in the treatment of patients with PBC.MethodsForty-eight PBC patients with incomplete response to UDCA were enrolled and randomly assigned to two groups (24 in the experiment group and 24 in the control group). For the experimental group, the patients were administered 13-15 mg/kg/day UDCA in combination with 200 mg/day fenofibrate. For the control group, the patients continued to receive UDCA at 13-15 mg/kg/day. The patients were followed up for at least 12 months. The serum levels of alkaline phosphatase (ALP), gamma-glutamyl transferase (γ-GT), aspartate aminotransferase (AST), and other biochemical parameters were measured at 3, 6, and 12 months during the trial to assess patient conditions.ResultsAt 12 months, 20.8% of the patients in the experimental group had all three indexes of serum ALP, γ-GT, and total bilirubin normalized, while 0% of patients in the control group reached the primary outcome (difference, 20.8 percentage points; 95% CI, 4.6-37.0). 54.2% of the patients had normal ALP levels in the experimental group and 4.2% in the control group (difference, 50 percentage points; 95% CI, 28.5-71.5). The experimental group had greater improvement of ALP (p < 0.001) and IgG (p = 0.026) than the control group. The biochemical indexes of the patients in the experimental group also significantly improved during the treatment of fenofibrate.ConclusionAddition of fenofibrate can improve biochemical indexes of PBC patients who had an incomplete response to UDCA. Reversible elevation of serum creatine and transaminases is observed in some patients.The trial was registered in the Chinese Clinical Trial Registry (ChiCTR) as ChiCTR1800020160 (protocol available online: http://www.chictr.org.cn/showproj.aspx?proj=32443).

Dataset Information

Prognostic Factors for Transplant-Free Survival and Validation of Prognostic Models in Chinese Patients with Primary Biliary Cholangitis Receiving Ursodeoxycholic Acid.

Publications

Prognostic Factors for Transplant-Free Survival and Validation of Prognostic Models in Chinese Patients with Primary Biliary Cholangitis Receiving Ursodeoxycholic Acid.

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