Table 1

Commonly referenced criteria for prognosis based on laboratory indices134

CriteriaTreatment response criteriaSample sizeResults
Barcelona criteria13 Response to treatment defined by ALP decrease >40% of baseline values or normal levels after 1 year of treatment192 patients (181 women)8.9% died or fulfilled criteria for liver transplantation
Observed survival higher than that predicted by Mayo model and lower than control population (P<0.001)
61% responded to treatment
Survival of responders was significantly higher than that predicted by Mayo model and similar to that estimated for control population (P=0.15)
Paris I criteria14 Treatment response defined as:
  • ALP <3xULN and

  • Aspartate transaminase (AST) <2xULN and

  • Bilirubin <1 mg/dL

292 patients10-year transplant-free survival rate of 90% (95% CI 81% to 95%), compared with 51% (95% CI 38% to 64%) for those who did not (P<0.001)
Paris II criteria15 Early stage PBC defined by normal bilirubin and albumin at baseline
Response treatment criteria: ALP and AST ≤1.5×ULN with normal bilirubin level
165 patients;
average follow-up 7 years
All adverse events observed in non-responders (P<0.001)
Mayo282 Response defined as ALP <2 xULN at 6 months180 patientsAfter 6 months of UDCA therapy, patients with serum alkaline phosphatase levels less than twice normal (P<0.04) were more likely to respond favourably to treatment over a 2-year period
Mayo16 Response defined as ALP <2 xULN at 1 year73 patients;
median 2 years follow-up
Patients with ALP ≥2×ULN had a 2-fold greater likelihood of developing endpoints compared with patients with lower values (23% vs 11%) (P<0.05).
Patients with bilirubin >1 mg/dL were four times more likely to develop endpoints compared with those with lower values (33% vs 8%) (P=0.02)
Patients with ALP ≤1.67×ULN and bilirubin ≤1 mg/dL had the least likelihood of reaching adverse clinical endpoints
Toronto criteria18 ALP <1.67xULN at 2 years of treatment with UDCA69 patients with follow-up liver biopsy performed approximately 10 years after initial histological diagnosisHistological progression in stage of fibrosis observed in paired liver biopsies was associated with absence of biochemical response at 2 years: ALP >1.67xULN, P=0.001, OR 12.14, 95% CI 2.69 to 54.74 when defined as an increase in one stage
ALP >1.76× ULN, P=0.03, OR 5.07, 95% CI 1.17 to 21.95 when defined as an increase in two stages
Ductopenia (>50% loss) predicted histological progression (P=0.012) and biochemical response to UDCA (P=0.002)
Rotterdam criteria17 PBC classified as early (pre-treatment bilirubin and albumin values normal), moderately advanced (one level abnormal), or advanced (both values abnormal)
Biochemical response defined by normalisation of abnormal bilirubin and/or albumin values
375 patients;
median follow-up time 9.7 years
Prognosis for early PBC comparable to Dutch population and better than predicted by Mayo risk score
Survival of responders better than that of non-responders (according to Paris and Rotterdam criteria; P<0.001) Prognosis of early PBC comparable for responders and non-responders
Prognosis of responders significantly better in those with (moderately) advanced disease