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Biochemical criteria at 1 year are not robust indicators of response to ursodeoxycholic acid in early primary biliary cirrhosis: results from a 29‐year cohort study
Author(s) -
Papastergiou V.,
Tsochatzis E. A.,
RodriquezPeralvarez M.,
Thalassinos E.,
Pieri G.,
Manousou P.,
Germani G.,
Rigamonti C.,
Arvaniti V.,
Karatapanis S.,
Burroughs A. K.
Publication year - 2013
Publication title -
alimentary pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 3.308
H-Index - 177
eISSN - 1365-2036
pISSN - 0269-2813
DOI - 10.1111/apt.12522
Subject(s) - ursodeoxycholic acid , medicine , primary biliary cirrhosis , gastroenterology , liver transplantation , biliary cirrhosis , cohort , cirrhosis , surrogate endpoint , transplantation , disease , autoimmune disease
Summary Background In primary biliary cirrhosis ( PBC ), biochemical criteria at 1 year are considered surrogates of response to ursodeoxycholic acid ( UDCA ). However, due to the slow natural history of PBC , evaluation at 1 year may be suboptimal to assess the therapeutic response, particularly in early disease. Aim To determine whether evaluation of biochemical criteria at 1 year is a reliable surrogate of UDCA response in early PBC . Methods We analysed the prospectively collected data of 215 patients (untreated = 129; UDCA ‐treated = 86) with early PBC (normal baseline bilirubin/albumin) and a median follow‐up of 8 years (range: 1–29.1). The 1‐year attainment rates of the Barcelona, Paris‐I, Paris‐ II and Toronto definitions, and their predictive relevance for a poor outcome (death, transplantation, complications of cirrhosis), were assessed either as a result of UDCA or no treatment. Independent associations with attaining each UDCA response definition were identified by multivariate analysis. Results Untreated patients displayed 1‐year biochemical features compatible with ‘treatment response’ at rates (Barcelona: 36.4%, Paris‐I: 66.7%, Toronto: 59.7%, Paris‐ II : 40.3%) similar to those obtained under UDCA . Depending on the definition, baseline ALP ≤3x ULN ( OR : 4.80–35.90), AST ≤2x ULN ( OR : 5.63–9.34) and early histological stage ( OR : 3.67–3.87) were the stronger predictors for attaining the criteria. UDCA treatment was associated with attaining Barcelona ( OR = 2.16) and Paris‐ II ( OR = 2.84), but not Paris‐I, and not Toronto definition when excluding late histological cases. Paris‐I criteria were significantly predictive of long‐term outcomes ( HR = 2.83) in untreated patients. Conclusions In early PBC , biochemical criteria at 1 year reflect severity of the disease rather than the therapeutic response to UDCA .