From the Guidelines
Primary biliary cholangitis (PBC) patients who have undergone liver transplantation should be given ursodeoxycholic acid (UDCA) lifelong to prevent recurrence, as recommended by the most recent guidelines 1. The dosage of UDCA should be 10 to 15 mg/kg/day in two divided doses, as this has been associated with a lower risk of PBC recurrence, as well as a reduction in the long-term risk of graft loss, liver-related death, and all-cause death 1. Key aspects of PBC management include:
- Monitoring for complications like osteoporosis, fat-soluble vitamin deficiencies, and pruritus
- Regular liver function tests every 3-6 months to monitor disease progression and treatment response
- Specialist hepatology referral for optimal management It is also important to note that the choice of immunosuppressive regimen should consider medication adherence, as this is one of the most important risks for acute rejection 1. Additionally, mental health should be screened, and psychological therapies should be provided to patients experiencing common feelings such as reactive depression or anxiety, and physical activity has been shown to improve quality of life 1. Overall, the management of PBC should prioritize a comprehensive approach that addresses the physical and psychological aspects of the disease, with a focus on preventing recurrence and improving quality of life.
From the FDA Drug Label
OCALIVA is a prescription medicine used to treat primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have not responded well enough to UDCA, or alone in adults who cannot tolerate UDCA. It is not known if taking OCALIVA will improve your chance of survival or improve your symptoms of PBC. The answer to PBC is that obeticholic acid (OCALIVA) is used to treat primary biliary cholangitis (PBC) in adults who have not responded well enough to ursodeoxycholic acid (UDCA), or alone in adults who cannot tolerate UDCA 2.
- Key points:
- Obeticholic acid (OCALIVA) is used to treat PBC.
- OCALIVA is used in combination with UDCA or alone in adults who cannot tolerate UDCA.
- It is not known if OCALIVA will improve survival or symptoms of PBC. Note that hepatic decompensation and failure have been reported with OCALIVA treatment in PBC patients with cirrhosis 2.
From the Research
Definition and Diagnosis of PBC
- Primary biliary cholangitis (PBC) is an autoimmune disease of the liver characterized by destruction and inflammation of the intrahepatic bile ducts 3.
- The disease is often discovered through abnormal alkaline phosphatase (ALP) activity, and is confirmed when anti-mitochondrial antibodies (AMA) are present 3.
- Diagnosis of PBC is based on the presence of AMA or specific antinuclear antibodies, and a cholestatic biochemical profile, while biopsy is recommended only in selected cases 4.
Treatment of PBC
- Ursodeoxycholic acid (UDCA) is the first-line therapy for PBC, which has been proven to normalize serum markers of liver dysfunction, halt histologic disease progression, and lead to a prolongation of transplant-free survival 5.
- However, 30-40% of patients do not respond to UDCA, and obeticholic acid (OCA) is the only registered agent for second-line treatment in UDCA-non responders 5.
- OCA has been shown to improve surrogate markers of prognosis in PBC, and has proven to be effective in improving hepatic bile acid excretion in patients with PBC 6, 7.
- Other treatment options, such as bezafibrate and budesonide, may be considered in patients with inadequate response to UDCA 6.
Management and Prognosis of PBC
- PBC can progress to cirrhosis and end-stage liver disease, and hepatocellular carcinoma (HCC) develops in up to 3.5% of PBC patients 3.
- Risk stratification is crucial in PBC management, and tools such as bile acid dosage and liver biopsy may provide relevant information for risk stratification and prediction of UDCA response 4.
- Personalized approaches to therapy may be facilitated by a better understanding of PBC pathogenesis and the development of new treatments 4.