Lu M, Zhou Y, Haller IV, Romanelli RJ, VanWormer JJ, Rodriguez CV, Anderson H, Boscarino JA, Schmidt MA, Daida YG, Sahota A, Vincent J, Bowlus CL, Lindor K, Zhang T, Trudeau S, Li J, Rupp LB, Gordon SC; FOLD Investigators., Clin Gastroenterol Hepatol. pii: S1542-3565(17)31529-X. doi: 10.1016/j.cgh.2017.12.033. [Epub ahead of print], 2017 Dec 22
BACKGROUND & AIMS: There are few data from longitudinal studies of trends in primary biliary cholangitis (PBC) among patients under routine clinical care in the United States (US). We collected data from the Fibrotic Liver Disease consortium to investigate changes in incidence and prevalence of PBC and the effects of patient demographics, clinical features, and treatment on mortality.
METHODS: We collected demographic and clinical data for the general patient population as well as PBC patients receiving care from 11 health systems in different regions of the US (Northeast, Midwest, Northwest, and South) from January 1, 2003 through December 31, 2014. Annual percentage changes in PBC prevalence and incidence were estimated using join-point Poisson regression. Differences based on race, age, and sex were calculated with rate ratios. 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 geographic regions.
RESULTS: In our racially diverse cohort of 3488 patients with PBC (21% Hispanic, 8% African American, 7% Asian American), 70% had ever received UDCA. From 2006 through 2014, the prevalence of PBC increased from 21.7 to 39.2 per 100,000 persons. Adjusted annual percentage changes in prevalence differed among age groups (≤40 years, 41-50 years, 51-60 years, 61-70 years, and >70 years), ranging from 3.0% to 7.5% (P<.05). Incidence did not change significantly during the study period (3.7 vs 3.4 per 100,000 person-years in 2006 and 2014, respectively; P=.09). Ratios of prevalence for women vs men (3.9:1) and incidence for women vs men (3.2:1) were consistent over the study period. Among African Americans, the prevalence of PBC increased from 16.9 to 30.8 per 100,000 during the study period, and annual incidence ranged from 2.6 to 6.6 per 100,000 person-years. In adjusted analyses, increased level of alkaline phosphatase at baseline was associated with significantly higher mortality (adjusted hazard ratios [aHR]=1.24; 95% CI, 1.04-1.48 for patients with 1-2 times the upper limit of normal and aHR=2.27; 95% CI, 1.88-2.73 for patients with more than 3 times the upper limit of normal). UDCA treatment was associated with significantly reduced mortality (aHR=0.57; 95% CI, 0.52-0.64).
CONCLUSION: In an analysis of data from patients receiving routine clinical care in Fibrotic Liver Disease Consortium health systems, we found that prevalence of PBC increased from 2004 through 2014, despite steady incidence. Patient demographic and clinical characteristics, as well as UDCA treatment, affected mortality.