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Survival Outcomes

Based on the Global PBC Study Group, ALP levels between 1x-1.67x ULN had a similar risk of liver transplant or death as patients with levels up to 3x ULN1,2,a

≤1x ULN was the optimal threshold for ALP in a subgroup analysis of UDCA-treated patients with ALP ≤1.67x ULN and normal bilirubin at 1 year

Survival rate estimates

Graph showing survival rate for different levels of ALP

  • Adapted from Murillo Perez CF, Harms MH, Lindor KD, et al. Am J Gastroenterol. 2020;115(7):1066-1074.

When ALP was >1x ULN,

the risk of liver transplant or death was 44% greater

than if ALP was ≤1x ULN (P=.03)

  • Adapted from Murillo Perez CF, Harms MH, Lindor KD, et al. Am J Gastroenterol. 2020;115(7):1066-1074.

Bilirubin that surpasses 0.6x ULN was found to significantly increase risk1,2,c

The optimal threshold for bilirubin was found to be ≤0.6x ULN in this analysis of the Global PBC Study Group

Survival rate estimates

Graph showing survival rate for different levels of bilirubin

  • Adapted from Murillo Perez CF, Harms MH, Lindor KD, et al. Am J Gastroenterol. 2020;115(7):1066-1074.

When bilirubin was >0.6x ULN,

>2x more patientsd had a liver transplant or died within 10 years

than if bilirubin was ≤0.6x ULN

second small table
  • Adapted from Murillo Perez CF, Harms MH, Lindor KD, et al. Am J Gastroenterol. 2020;115(7):1066-1074.

When bilirubin is already high, normalizing ALP was found to reduce the risk significantly1,2,c

Even when bilirubin was >0.6x ULN, a significant survival benefit was observed when ALP was normalized (ie, ≤1x ULN) compared to ALP 1x-1.67x ULN

Survival rate estimates

Graph showing survival rate for different levels of ALP and bilirubin

  • Adapted from Murillo Perez CF, Harms MH, Lindor KD, et al. Am J Gastroenterol. 2020;115(7):1066-1074.

~5x more patientse had a liver transplant or died within 10 years

when ALP was between 1x-1.67x ULN and bilirubin was >0.6x ULN (26%), than those with ALP ≤1x ULN and bilirubin ≤0.6x ULN (~5%)

  • Adapted from Murillo Perez CF, Harms MH, Lindor KD, et al. Am J Gastroenterol. 2020;115(7):1066-1074.

Fibrosis stage, as well as biomarkers, should be considered in PBC risk stratification3,f

Fibrosis stage categorized as early (stage 1/2) vs advanced (stage 3/4) was an independent predictor of transplant-free survival, despite biochemical response in another Global PBC Study Group analysis

  • Patients with an advanced fibrosis stage had worse transplant-free survival rates despite treatment responseg
Transplant-free survival rate estimatesh

Graph showing transplant-free survival rate for different ALP levels and stages of fibrosis

  • Adapted from Murillo Perez CF, Hirschfield GM, Corpechot C, et al. Aliment Pharmacol Ther. 2019;50(10):1127-1136.

Patients with abnormal ALP and early fibrosis stage had similar transplant-free survival rates to those with normal ALP and advanced fibrosis stage (P=.12)

  • Adapted from Murillo Perez CF, Hirschfield GM, Corpechot C, et al. Aliment Pharmacol Ther. 2019;50(10):1127-1136.
  • a The Global PBC Study Group conducted an analysis including UDCA-treated and untreated patients diagnosed with PBC (N=3,059) with bilirubin levels ≤1x ULN at baseline or 1 year. The study explored optimal thresholds of bilirubin and ALP to predict liver transplantation or death. The association of ALP with survival was assessed in a subgroup of UDCA-treated patients with ALP ≤1.67x ULN and normal bilirubin at 1 year.1
  • b Survival was defined as an absence of liver transplant and all-cause mortality.1
  • c The Global PBC Study Group conducted an analysis including UDCA-treated and untreated patients diagnosed with PBC (N=3,059) with bilirubin levels ≤1x ULN at baseline or 1 year. The study explored optimal thresholds of bilirubin and ALP to predict liver transplantation or death.1
  • d Calculation based on percent rates of liver transplant or death extrapolated from survival estimate curve at 10 years.1,2
  • e Calculation based on data extrapolated from survival estimate curve at 10 years.1,2
  • f Data from the Global PBC Study Group were used, and this analysis included UDCA‐treated patients in whom a liver biopsy was performed at study entry in order to evaluate the utility of baseline fibrosis stage in predicting long-term outcomes in the context of biochemical risk stratification. Biopsies conducted in the 24 months prior to study entry and up to 12 months after study entry were considered baseline.3
  • g Biochemical treatment response after 1 year of UDCA therapy was defined by Toronto and Paris II criteria.3
  • h After 1 year of UDCA treatment.3
  • ALP, alkaline phosphatase; PBC, primary biliary cholangitis; UDCA, ursodeoxycholic acid; ULN, upper limit of normal.

References:
1. Murillo Perez CF, Harms MH, Lindor KD, et al; the GLOBAL PBC Study Group. Goals of treatment for improved survival in primary biliary cholangitis: treatment target should be bilirubin within the normal range and normalization of alkaline phosphatase. Am J Gastroenterol. 2020;115(7):1066-1074. 2. Data on file: Intercept Pharmaceuticals, Inc. 3. 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.