Pretreatment prediction of response to ursodeoxycholic acid in primary biliary cholangitis: development and validation of the UDCA Response Score

M. Carbone, A. Nardi, S. Flack, G. Carpino, N. Varvaropoulou, C. Gavrila, A. Spicer, J. Badrock, F. Bernuzzi, V. Cardinale, H.F. Ainsworth, M.A. Heneghan, D. Thorburn, A. Bathgate, R. Jones, J.M. Neuberger, P.M. Battezzati, M. Zuin, S. Taylor-Robinson, M.F. DonatoJ. Kirby, R. Mitchell-Thain, A. Floreani, F. Sampaziotis, L. Muratori, D. Alvaro, M. Marzioni, L. Miele, F. Marra, E. Giannini, E. Gaudio, V. Ronca, G. Bonato, L. Cristoferi, F. Malinverno, A. Gerussi, D.D. Stocken, H.J. Cordell, G.M. Hirschfield, G.J. Alexander, R.N. Sandford, D.E. Jones, P. Invernizzi, G.F. Mells, C. Thomas, M. Rahman, T. Yapp, L. Miele, A. Gasbarrini, A. Grieco

Research output: Contribution to journalArticlepeer-review


Background: Treatment 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. Methods: We 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. Findings: 2703 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
Original languageEnglish
Pages (from-to)626-634
Number of pages9
JournalThe Lancet Gastroenterology and Hepatology
Issue number9
Publication statusPublished - 2018


  • alanine aminotransferase
  • alkaline phosphatase
  • aspartate aminotransferase
  • bilirubin
  • creatinine
  • cytokeratin 7
  • ursodeoxycholic acid, adult
  • area under the curve
  • Article
  • cohort analysis
  • correlation analysis
  • face validity
  • female
  • follow up
  • histopathology
  • human
  • human tissue
  • immunohistochemistry
  • liver biopsy
  • liver transplantation
  • major clinical study
  • male
  • middle aged
  • observational study
  • platelet count
  • premedication
  • primary biliary cirrhosis
  • priority journal
  • protein expression
  • receiver operating characteristic
  • scoring system
  • sensitivity and specificity
  • statistical model
  • treatment response
  • UDCA response score
  • validation study


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