Management, outcomes and survival of an Australian IgG4-SC cohort: The MOSAIC study.

BACKGROUND AND AIMS: IgG4 sclerosing cholangitis (IgG4-SC) is the biliary component of the multisystem IgG4-related disease. We aimed to investigate the clinical features, demographics, treatment response and outcomes of IgG4-SC in a large Australian cohort. METHODS: We conducted nationwide retrospective cohort via the Australian Liver Association Clinical Trials Network (ALA-CRN). 39 sites were invited to participate. IgG4-SC was defined by the clinical diagnostic criteria established by the Japanese Biliary Association in 2012. Data were collected on patient demographic, clinical and laboratory information, presenting features, response to therapy and clinical outcomes. RESULTS: 67 patients meet inclusion criteria from 22 sites. 76% were male with mean age of 63.3 ± 14.5 years and a median IgG4 level of 3.6 g/L [0.09-67.1]. The most frequent presenting symptom was jaundice (62%) and abdominal pain (42%) and Type 1 biliary stricturing (52%) at the distal common bile duct was the most frequent biliary tract finding. Prednisolone was used as a primary treatment in 61 (91%) and partial or complete response occurred in 95% of subjects. Relapse was common (42%) in those who ceased medical therapy. After a median follow up of 3.9 years there was one hepatocellular carcinoma and no cholangiocarcinomas. CONCLUSIONS: Our study confirms the preponderance of IgG4-SC in males and highlights the steroid response nature of this condition although relapse is common after steroid cessation. Progression to malignancy was uncommon.

The benign course of liver disease in adults with cystic fibrosis and the effect of ursodeoxycholic acid.

BACKGROUND AND AIMS: The life expectancy of patients with cystic fibrosis (CF) has been increasing and the associated liver disease has emerged as a significant medical issue. Our aim was to describe the clinical features, course and effect of ursodeoxycholic acid (UDCA) on liver disease in an adult CF population. STUDY: From 1983 to 2005, 278 patients with CF were followed up at the Alfred Hospital, an adult tertiary referral centre. Twenty-seven patients (9.7%) satisfied the criteria for liver disease and their clinico-pathological features were assessed. The effect of UDCA on hepatobiliary symptoms and biochemical parameters was determined. RESULTS: The mean age at liver disease diagnosis was 23 years (range 8-47 years). Portal hypertension was present in 18 (67%) patients. During a median follow-up of 7 years (range 1.5-15), variceal haemorrhage occurred in two patients and ascites in three (one spontaneously). Nine (33%) patients died and five (19%) underwent lung transplantation. There was no encephalopathy, liver transplantation or liver-related deaths. UDCA was taken by 22 patients and was associated with a significant improvement in hepatobiliary symptoms [11/22 (50%) in the pre-UDCA period vs 1/22 (4%) in the post-UDCA period; P=0.0003] and a significant reduction in aspartate aminotransferase (P=0.005); alanine aminotransferase (P<0.001); gamma-glutamyltranspeptidase (P=0.021); and alkaline phosphatase (P<0.001). CONCLUSIONS: Liver disease in adults with CF is commonly complicated by portal hypertension, but morbidity and mortality associated with this in our small patient population were low. UDCA is associated with improvement in hepatobiliary symptoms and liver function tests.