Evidence-Based Treatment of Primary Biliary Cirrhosis

Abstract

The natural history of primary biliary cirrhosis (PBC) has improved greatly during the past two decades because of its diagnosis at earlier stages and the widespread use of ursodeoxycholic acid as first-line treatment. As a result, far fewer patients require liver transplantation and patients diagnosed with early-stage disease appear to have a normal life expectancy. The evidence-based treatment of PBC is reviewed here. The challenge of designing and executing high-quality clinical trials as well as lessons from past clinical trials are discussed. The rationales for the use of budesonide and new investigational therapies (PPAR and FXR agonists) as well as their therapeutic potential in light of recent available data are also reviewed. An algorithm of the management of PBC patients responding or not to ursodeoxycholic acid is proposed.

A multicenter, randomized, double-blind trial comparing the efficacy and safety of TUDCA and UDCA in Chinese patients with primary biliary cholangitis

“…Administration of ursodeoxycholic acid (UDCA) is the standard of care for PBC patients [3–5] and is recommended by major guidelines. [6,7] The primary therapeutic action of hydrophilic UDCA is to replace hydrophobic bile acids, thereby attenuating cholestasis and hepatocellular injury.…”

Progress in the Management of Patients with Cholestatic Liver Disease: Where Are We and Where Are We Going?

“…14 Studies have shown significant improvement of ALP and changes in liver histology in PBC patients treated with budesonide. [24][25][26][27] Hirschfield et al 27 found that in PBC patients with a poor response to UDCA, the combination of UDCA and budesonide for 36 months did not improve the histological progression, but the proportion of patients with normal ALP (35%) was significantly higher than that in the placebo group (9%, p=0.023). The clinical trials are not large enough, so larger trials and more complete clinical trial evidence are needed to confirm the benefits of budesonide in the treatment of PBC.…”

Bile Acids, Liver Cirrhosis, and Extrahepatic Vascular Dysfunction

“…We have known for about 40 years that UDCA has a “hepatoprotective” effect. There is good evidence for the beneficial effect of UDCA in PBC ( Poupon, 2014 ). Yet, the proof of a beneficial influence for other chronic liver diseases such as PSC or metabolic liver diseases is much lower or nonexisting ( Reardon et al, 2016 ; de Vries and Beuers, 2017 ).…”