Epidemiologic trends and treatment survival benefits in a US cohort of patients with primary biliary cholangitis (PBC).
Lu M, Zhou YR, Haller I, Romanelli R, VanWormer JJ, Rodriguez CV, Anderson H, Boscarino JA, Schmidt MA, Daida Y, Sahota AK, Vincent J, Bowlus CL, Lindor KD, Zhang T, Trudeau S, Li J, Rupp LB, Gordon SC, and Fibrotic Liver Dis FC. Epidemiologic trends and treatment survival benefits in a US cohort of patients with primary biliary cholangitis (PBC). Hepatology 2017; 66:157A.
Background and aims: We used data from the Fibrotic Liver Disease (FOLD) Consortium — an established PBC cohort of over 4000 PBC cases drawn from 14.5 million patientts — to study trends in PBC prevalence and incidence, and risk of all‐cause mortality, among PBC patients under routine care in the US. Methods: Annual percentage change (APC) in PBC incidence and prevalence was estimated with join‐ point Poisson regression. Differences associated with race, age, and gender were calculated with rate ratios (RR). Risk of all‐cause mortality was estimated using Cox regression with adjustment for patient characteristics and treatment with ursodeoxycholic acid (UDCA). Propensity scores were used to adjust for treatment selection bias. Analyses were adjusted by study site. Results: Mean age of diagnosis in our racially‐diverse cohort of 3488 PBC patients (21% Hispanic; 8% African American; 7% Asian American) was 59 years. Mean follow‐up was 5 years. 70% received UDCA treatment. From 2006–2014, PBC prevalence increased from 21.7 to 39.2 per 100,000 persons. Adjusted APC (aAPC) differed by age, ranging from 3.0–7.5% (p<0.05). Incidence was 3.7 per 100,000 person‐years in 2006, and 3.4 in 2014, with no trend detected (p=0.09). Across time, the ratio of prevalence (3.8:1) and incidence (3.2:1) between women and men was consistent African Americans had lower prevalence (0.8:1) and incidence (0.6:1) than Whites. In adjusted analyses (Fig), men, patients with elevated ALP, those with a AST/ALT ratio >1.1 (a marker of cirrhosis), and UDCA‐untreated patients were at higher risk of mortality (p<0.05). Specifically, UDCA treatment reduced risk of mortality (adjusted hazard ratio [aHR]=0.52 [0.5–0.6]); AST/ALT ratio >1.1 increased risk of mortality (aHR=2.6 [2.3–2.9]) Conclusions: In US patients under routine clinical care, PBC prevalence rose while incidence remained steady between 2006–2014. Elevated AST/ALT ratios were associated with increased all‐cause mortality; UDCA treatment reduced all‐cause mortality.