Primary Biliary Cholangitis: Promising Emerging Innovative Therapies and Their Impact on GLOBE Scores

Abstract

Primary biliary cholangitis (PBC), previously referred to as primary biliary cirrhosis, is an autoimmune disorder leading to the destruction of intra-hepatic bile ducts. If untreated, progressive bile duct damage and cholestasis can lead to ductopenia and result in cirrhosis. Ursodiol, the first drug approved for PBC, has changed the natural history of this disease and improved patient outcomes. Subsequently, several new prediction models incorporating a response to ursodiol were developed. These include the GLOBE score, which was shown to predict long-term outcomes in patients with PBC. In 2016, obeticholic acid (OCA) became the second drug to be approved by the FDA, predominantly based on improvement in alkaline phosphatase (ALP) levels. This trial has subsequently influenced the design of clinical trials. Several drugs are currently being evaluated as therapeutic options for PBC, with improvement in ALP being a main endpoint. In this review, we will discuss the impact of new therapies on GLOBE scores in patients with PBC.

Transforming Screening, Risk Stratification, and Treatment Optimization in Chronic Liver Disease Through Data Science and translational Innovation

“…Still, stiffness alone lacks perfect accuracy, so combining with prognostic algorithms like GLOBE and UK-PBC may further optimize prognostic utility. 49 Selecting appropriate patients is equally important when delivering localized therapies like RFA for HCC. 50 RFA induces tumor necrosis through thermal ablation, but recurrence is common.…”

Fibrates and Fibrate-induced Liver Injury in Primary Biliary Cholangitis

“…In 2021, a formal contraindication was issued against the use of OCA in patients with Child-Pugh class B or C cirrhosis, citing 25 reported cases of liver injury as a direct result of use between 2016 and 2021. 29 OCA has been shown to significantly improve the biochemical markers of PBC that can correlate with prolonged liver transplantfree survival, allowing nearly 50% of patients with incomplete biochemical response to UDCA alone to achieve a complete response. For those who cannot afford OCA, or cannot tolerate it, or are too advanced in their disease process to use it safely, alternative treatments are needed.…”

PPAR agonists in Primary Biliary Cholangitis: A Systematic Review and Meta-Analysis of Randomized Controlled Trials

“…[7] The only medications approved by the Food and Drug Administration (FDA) are ursodeoxycholic acid (UDCA) and obeticholic acid (OCA), as an adjunctive agent to UDCA or monotherapy in UDCA-intolerant patients. [8,9] However, 40% of patients do not respond adequately to UDCA and OCA frequently leads to pruritus and treatment discontinuation. [7,10] On the other hand, PPAR ligands are receiving much interest due to positive results obtained in clinical trials, especially regarding reduction in pruritus.…”