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PBC Predictive Biomarkers

Despite no consensus on response criteria, ALP and bilirubin have been considered the most important biomarkers in managing risk1-4

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  • This is not an all-inclusive listing. Adapted from Younossi ZM, Bernstein D, Shiffman ML, et al. Am J Gastroenterol. 2019;114(1):48-63.

Risk of progression is dependent on reduction of predictive biomarkers3,5

  • Prior to first-line treatment, patients may have ALP 4x-5x ULN and bilirubin above normal6,a
  • Levels of ALP and bilirubin measured at baseline and each year for 5 years are strongly associated with clinical outcomes (liver transplant or death)5,b

Elevations in ALP and bilirubin have been found to have a considerable impact on liver transplant or death7,c

Occurrence of liver transplant or death

Chart showing impact of elevated ALP and bilirubin on liver transplant or death Chart showing impact of elevated ALP and bilirubin on liver transplant or death

Elevations in ALP increase risk of poor outcomes7

ALP >2x ULN is associated with a 2.2x greater risk for liver transplant or death at 1 year compared to those who remained ≤2x ULN (P<.0001)

Higher bilirubin levels are associated with increased mortality and reduced transplant-free survival7

Bilirubin >1x ULN is associated with a 5.5x greater risk for liver transplant or death at 1 year (P<.0001)

  • a Data from a combined analysis of 3 randomized, controlled trials comparing UDCA (n=273) vs placebo (n=275) for up to 4 years.6
  • b Data from an international, multicentered meta-analysis of UDCA-treated and untreated patients diagnosed with PBC (N=4,845).5
  • c Patient data were obtained from the international Global PBC study database; specifically, this analysis included UDCA-treated patients who had ALP ≤2x ULN or bilirubin ≤1x ULN at 1 year follow-up (N=2,527) who were evaluated to identify patients within this group at risk for adverse outcome during follow-up (median follow-up of 7.11 years).7
  • ALP, alkaline phosphatase; AST, aspartate aminotransferase; PBC, primary biliary cholangitis; UDCA, ursodeoxycholic acid; ULN, upper limit of normal.

References:
1. Murillo Perez CF, Hirschfield GM, Corpechot C, et al; the GLOBAL PBC Study Group. Fibrosis stage is an independent predictor of outcome in primary biliary cholangitis despite biochemical treatment response. Aliment Pharmacol Ther. 2019;50(10):1127-1136. 2. Younossi ZM, Bernstein D, Shiffman ML, et al. Diagnosis and management of primary biliary cholangitis. Am J Gastroenterol. 2019;114(1):48-63. 3. Lindor KD, Bowlus CL, Boyer J, Levy C, Mayo M. Primary biliary cholangitis: 2018 practice guidance from the American Association for the Study of Liver Diseases. Hepatology. 2019;69(1):394-419. 4. European Association for the Study of the Liver. EASL clinical practice guidelines: management of cholestatic liver diseases. J Hepatol. 2009;51(2):237-267. 5. Lammers WJ, van Buuren HR, Hirschfield GM, et al; the Global PBC Study Group. Levels of alkaline phosphatase and bilirubin are surrogate end points of outcomes of patients with primary biliary cirrhosis: an international follow-up study. Gastroenterology. 2014;147(6):1338-1349. 6. Poupon RE, Lindor KD, Cauch-Dudek K, Dickson ER, Poupon R, Heathcote EJ. Combined analysis of randomized controlled trials of ursodeoxycholic acid in primary biliary cirrhosis. Gastroenterology. 1997;13(3):884-890. 7. Lammers WJ, Van Buuren HR, Ponsioen CY, et al. Elevation of alkaline phosphatase during follow-up is an early predictor of hyperbilirubinaemia and of clinical endpoints in primary biliary cholangitis – an international study. Poster presented at: 66th Annual Meeting of the AASLD; November 13-15, 2015; San Francisco, CA.