Medical Treatment of Vanishing Bile Duct Syndrome in Adults
Medical management of vanishing bile duct syndrome (VBDS) should be aimed at treating both the underlying disease and symptoms or complications of long-standing cholestasis. For the treatment of pruritus, the most specific symptom of VBDS, anion exchange resins represent the first-line therapy and only for patients resistant or intolerant to cholestyramine, rifampicin or opioid antagonists should be considered. The main complications of chronic cholestasis are metabolic bone disease, hyperlipidemia and malnutrition. In patients with VBDS adequately supplemented with calcium and vitamin D the prevalence of bone disease is similar to that expected in an age- and sex-matched population. Only in a minority of patients hyperlipidemia and malnutrition should be treated. Regarding the treatment of the underlying disease, data on meaningful numbers of patients are available only for primary biliary cirrhosis (PBC) and primary sclerosing cholangitis (PSC). For end-stage liver disease, when the majority of bile ducts had been already destroyed, there is no indication for medical treatment and the only therapeutic option is orthotopic liver transplantation (OLT). Therefore medical treatment should be started earlier during the course of disease. Results from clinical trials with immunosuppressive, anti-inflammatory and anti-fibrotic agents have been disappointing and the only drug currently indicated for such diseases is ursodeoxycholic acid (UDCA), even if there is no consensus on its effect on mortality.