title: How the concept of biochemical response influenced the management of primary biliary cholangitis over time.
abstract: BACKGROUND
  Criteria assessing biochemical response to ursodeoxycholic acid (UDCA) are established risk stratification tools in primary biliary cholangitis (PBC). We aimed to evaluate to what extent liver tests influenced patient management during a three decade period, and whether this changed over time.
  
  
  METHODS
  851 Dutch PBC patients diagnosed between 1988 and 2012 were reviewed to assess patient management in relation to liver test results during UDCA treatment. To do so, biochemical response at one year was analysed retrospectively according to Paris-1 criteria.
  
  
  RESULTS
  Response was assessable for 687/851 (81%) patients; 157/687 non-responders. During a follow-up of 8.8 years (IQR 4.8-13.9), 141 died and 30 underwent liver transplantation. Transplant-free survival of non-responders (60%) was significantly worse compared with responders (87%) (p < 0.0001). Management was modified in 46/157 (29%) non-responders. The most frequent change observed, noted in 26/46 patients, was an increase in UDCA dosage. Subsequently, 9/26 (35%) non-responders became responders within the next two years. Steroid treatment was started in one patient; 19 patients were referred to a tertiary centre. No trend towards more frequent changes in management over time was observed (p = 0.10).
  
  
  CONCLUSIONS
  Changes in medical management occurred in a minority of non-responders. This can largely be explained by the lack of accepted response criteria and of established second-line treatments for PBC. Nevertheless, the observation that response-guided management did not increase over time suggests that awareness of the concept of biochemical response requires further attention,particularly since new treatment options for PBC will soon become available.
authors: Department of Gastroenterology and Hepatology, Erasmus University Medical Center Rotterdam, the Netherlands.
authors: W J
authors: WJ
authors: Lammers
authors: M
authors: M
authors: Leeman
authors: C I J
authors: CI
authors: Ponsioen
authors: K
authors: K
authors: Boonstra
authors: K J
authors: KJ
authors: van Erpecum
authors: F H J
authors: FH
authors: Wolfhagen
authors: J Ph
authors: JP
authors: Kuyvenhoven
authors: J M
authors: JM
authors: Vrolijk
authors: J P H
authors: JP
authors: Drenth
authors: E M
authors: EM
authors: Witteman
authors: C M J
authors: CM
authors: van Nieuwkerk
authors: B W
authors: BW
authors: van der Spek
authors: B J M
authors: BJ
authors: Witteman
authors: G W
authors: GW
authors: Erkelens
authors: M A M T
authors: MA
authors: Verhagen
authors: S A C
authors: SA
authors: van Tuyl
authors: A C
authors: AC
authors: Poen
authors: J T
authors: JT
authors: Brouwer
authors: F
authors: F
authors: Ter Borg
authors: G H
authors: GH
authors: Koek
authors: T J M
authors: TJ
authors: van Ditzhuijsen
authors: B E
authors: BE
authors: Hansen
mesh_terms: D000328:Adult; D000368:Aged; D000469:Alkaline Phosphatase; D001219:Aspartate Aminotransferases / Q000097:blood; D001663:Bilirubin / Q000097:blood; D002756:Cholagogues and Choleretics / Q000627:therapeutic use*; D019468:Disease Management; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008105:Liver Cirrhosis, Biliary / Q000097:blood / Q000188:drug therapy*; D016031:Liver Transplantation / Q000706:statistics & numerical data; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D012709:Serum Albumin / Q000378:metabolism; D016896:Treatment Outcome; D014580:Ursodeoxycholic Acid / Q000627:therapeutic use*
chemical_list: D002756:Cholagogues and Choleretics; D012709:Serum Albumin; D014580:Ursodeoxycholic Acid; D001219:Aspartate Aminotransferases; D000469:Alkaline Phosphatase; D001663:Bilirubin