Nancy Reau, MD

Second-Line Treatments and Management of Symptoms in PBC

Video content above is prompted by the following:

There are several second-line therapy options, including the first approved option, OCA. When do you use OCA, and when do you evaluate if it successfully meets treatment goals?

Long-term treatment with OCA is associated with pruritus (77% of patients) and fatigue (33% of patients) (

), which are also symptoms of PBC. What strategies do you use to manage these symptoms in general?

How do you counsel patients who may consider starting OCA and are affected by pruritus prior to therapy

The off-label use of fibrates has been another treatment option for PBC. When and how do you use fibrates for treating PBC? What are some challenges and adverse effects of fibrates?

How do you assess the effectiveness of these therapies in managing the disease? Please comment on biochemical response, pruritus, potential adverse effects, etc.

What are the key indicators that suggest a need for a change in treatment strategy?

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Obeticholic acid (OCA) is a second-line therapy for primary biliary cholangitis (PBC), used when first-line treatments are inadequate. It functions by activating the farnesoid X receptor, reducing bile acid synthesis. Long-term OCA use can cause pruritus and fatigue, necessitating symptom management strategies. Patient counseling is crucial, especially for those with pre-existing pruritus. Fibrates, used off-label, offer an alternative treatment but present challenges like adverse effects. Effectiveness is assessed through biochemical response and symptom management. Treatment strategy changes are indicated by inadequate response or adverse effects.

The panel of experts cover second-line treatment options for primary biliary cholangitis (PBC), focusing on the use of obeticholic acid (OCA), its mechanism of action, management of adverse effects like pruritus and fatigue, the role of off-label fibrates, and strategies for assessing and adjusting therapy based on patient response and adverse effects.

Treatment Goals and First-Line Therapy in PBC

What are the treatment goals for patients with PBC?

How do treatment goals change based on the severity of the disease?

UDCA is recommended as the first-line pharmacological treatment for PBC. How does it work to treat the disease?

What are some challenges or limitations you encounter when using UDCA that may prompt a need to modify therapy?

How do you define biochemical response and when?

Primary Biliary Cholangitis (PBC) treatment aims to alleviate symptoms, prevent disease progression, and improve quality of life. Treatment goals evolve with disease severity, focusing on symptom management in early stages and preventing complications in advanced stages. Ursodeoxycholic acid (UDCA) is the first-line treatment, working by improving bile flow and reducing liver inflammation. It is indicated for patients with early-stage PBC. Challenges with UDCA include incomplete biochemical response, necessitating therapy modification. Biochemical response is defined by specific liver function tests, typically assessed after six months of treatment.

The panel of experts outline the primary treatment goals for patients with primary biliary cholangitis (PBC), detailing how goals adjust based on disease severity, the role of ursodeoxycholic acid (UDCA) as first-line therapy, and the challenges that may require therapy modifications based on biochemical response.

Differential Diagnosis of PBC

How do you differentiate PBC from other liver diseases with similar presentations?

Primary biliary cholangitis (PBC) is distinguished from other liver diseases through a combination of clinical, serological, and histological features. Clinically, PBC often presents with fatigue and pruritus, and is more common in middle-aged women. Serologically, the presence of antimitochondrial antibodies (AMAs) is a hallmark of PBC, found in approximately 95% of patients. Elevated alkaline phosphatase levels are also indicative. Histologically, PBC is characterized by non-suppurative destructive cholangitis and the destruction of small to medium-sized bile ducts, which helps differentiate it from other cholestatic liver diseases.

The panel of experts focus on strategies for distinguishing primary biliary cholangitis (PBC) from other liver diseases with similar clinical presentations, emphasizing key diagnostic markers and assessment techniques.

Disease Progression and Extrahepatic Manifestations of PBC

What are some major complications and comorbidities associated with PBC (eg, vitamin deficiency, osteoporosis, comorbid conditions such as MASH, autoimmune conditions, obesity)?

Primary biliary cholangitis (PBC) is associated with several complications and comorbidities. Vitamin deficiencies, particularly fat-soluble vitamins like A, D, E, and K, are common due to malabsorption. Osteoporosis is a significant concern, often resulting from chronic cholestasis and vitamin D deficiency. Metabolic-associated steatohepatitis (MASH) can co-occur, complicating liver health further. Autoimmune conditions, such as Sjögren's syndrome and thyroid disorders, frequently accompany PBC. Obesity exacerbates liver disease progression and complicates management. These factors necessitate comprehensive monitoring and management strategies to mitigate their impact on patient outcomes.

The panel of experts examine major complications and comorbidities associated with primary biliary cholangitis (PBC), including vitamin deficiencies, osteoporosis, and conditions like metabolic-associated steatohepatitis (MASH), autoimmune disorders, and obesity.

Nancy Reau, MD

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