Major clinical events, signs and severity assessment scores related to actual survival in patients who died from primary biliary cirrhosis. A long-term historical cohort study

BACKGROUND/AIMS: One of the prognostic methods for survival in primary bili ary cirrhosis (PBC) is the Mayo model, with a time-scale limited to 7 years . The aim of our study was to assess how major clinical events, signs, seve ral severity assessment methods and Mayo survival probabilities fit in with actual patient survival, by using yearly observations until 0.5 years befo re patient death from PBC. METHODOLOGY: Data of 32 patients dying from PBC were collected prior to dea th at -0.5, -1, -2 etc. years (median: -5 years, range: -16 to -0.5 years). Major events registered were: first occurrence of ascites, upper gastroint estinal bleeding or manifest hepatic encephalopathy and signs, first observ ation of spider naevi or purpura. Severity assessment methods applied (all with scores and classes) were: Mayo (M), Child-Campbell (C), Pugh-Child (P) , Pugh-Child-PBC (PP), 'Child-Pugh' (CP), and Ascites Nutritional State-Chi ld (ANS). Fifty percent survival estimates were calculated from Mayo scores . Severity assessment method variables were: ascites (C, P, PP, CP, ANS), e ncephalopathy (C, P, PP, CP), nutritional state (C, ANS), edema (M), age (M ), serum albumin (M, C, P, PP, CP), bilirubin (C, M, P, PP, CP), and prothr ombin time (M, P, PP, CP). RESULTS: In 27 out of 32 patients a major event occurred, always between -6 and -0.5 years (median: -1 year) and, never between -16 and -7 years (p<0. 0001). A sign was first observed in 30/32 between -14 and -0.5 years (media n: -2 years). Compared to the total population, a sign, and even more so, a n event indicated a shorter survival (p=0.004 and p=0.0002, respectively). The median 50% estimated survival (predicted by the Mayo model) fitted the actual survival from -6 to -0.5 years (r=-0.7, p<0.0001), but not from -16 to -7 years (r=-0.1, p=0.4). All -6 to -0.5-year severity scores correlated (p<0.0001) both with actual survival (M, C, P, PP and CP r=0.7; ANS r=0.5) and with estimated M 50% survival (C, P, PP, CP r=-0.9; ANS r=-0.6; M scor e: -0.99), but none with actual survival from -16 to -7 years, except for M , slightly (r=-0.3, p=0.04). A nomogram for mean C, CP, M and ANS scores re lated to actual survival was constructed for the -6 to -0.5-year period. Th e C and CP classes A, B, and C did not appear to distinguish sufficiently i nto actual survival, whereas the M classes did. CONCLUSIONS: The occurrence of a major event appeared to exclude survival o ver 6 years. In these final 6 years, Child-Campbell, Mayo and Pugh scores c orrelated equally well with actual survival and better than Ascites/Nutriti onal State score. In our PBC patients, Campbell was an excellent alternativ e for Pugh; for Pugh, the original Child-Turcotte variable limits were full y sufficient.