Epidemiologia, estratificação de risco e tratamento da colangite biliar primária: estudo multicêntrico brasileiro

Abstract

Primary biliary cholangitis (PBC) is a chronic cholestatic liver disease in which antimitochondrial antibodies (AMA) are the diagnostic hallmark. Little is known about this disease in highly admixed populations. The purpose of this study was to evaluate clinical features and outcomes of PBC in Brazil. 562 patients (95% females, mean age 51 ± 11 years) with PBC were included. Mean follow-up was 6.2 ± 5.3 years. 32% had cirrhosis, 7% underwent liver transplantation and 3% died of liver-related causes. Concurrent autoimmune diseases and overlap with autoimmune hepatitis occurred, respectively, in 18.9% and 14%. Subjects with AMA-negative PBC were significantly younger (52.2 ± 14 vs. 59.6 ± 11 years, p = 0.001) and had their first symptom at an earlier age (43.2 ± 13 vs. 49.5 ± 12 years, p = 0.005) in comparison to patients with AMA positive PBC. Lower Immunoglobulin M (272.2 ± 183 vs. 383.2 ± 378 mg/dL, p = 0.01) and triglycerides (107.6 ± 59.8 vs.129.3 ± 75.7 mg/dL, p = 0.025) and higher bilirubin (3.8 ± 13.5 vs. 1.8 ± 3.4 mg/dL, p = 0.02) levels were also observed in this subgroup. 96% of the patients were treated with ursodeoxycholic acid (UDCA) and 12% required add-on therapy with fibrate, either bezafibrate, fenofibrate or ciprofibrate. Response to UDCA and to UDCA/fibrates therapy varied from 39%-67% and 42-61%, respectively, according to different validated criteria. Ciprofibrate appears to be at least as effective as bezafibrate. After 6 months of treatment with UDCA, the absence of response could properly identify patients who could benefit from early addition of second-line therapies, especially among those with advanced disease or high baseline liver enzyme levels. Advanced histological stages and non-adherence to treatment were associated with primary non-response to UDCA, while lower baseline alkaline phosphatase and aspartate aminotransferase (AST) levels correlated with better responses to both UDCA and UDCA/fibrates.