Emerging drugs for the treatment of Primary Biliary Cholangitis

Ahmad H. Ali, James H. Tabibian, Elizabeth J. Carey, Keith Lindor

Research output: Contribution to journalArticle

9 Citations (Scopus)

Abstract

Introduction: Primary biliary cholangitis (PBC) is an autoimmune chronic disease of the liver that can progress to cirrhosis and hepatocellular carcinoma. It affects approximately 1 in 4,000 with a 10:1 female to male ratio. The diagnosis of PBC can be made based on serum antimitochondrial antibodies (AMA) in a patient with abnormally high serum alkaline phosphatase after ruling out other causes of cholestasis and biliary obstruction. Genome-wide association studies have revealed several human leukocyte antigen (HLA) and non-HLA risk loci in PBC, and complex environmental-host immunogenetic interactions are believed to underlie the etiopathogenesis of the disease. Fatigue and pruritus are the most common and often problematic symptoms; although often mild, these can be severe and life-alternating in a subset of patients. Ursodeoxycholic acid (UDCA) is the only drug approved by the United States Food and Drug Administration for the treatment of PBC. Clinical trials have shown that UDCA significantly improves transplant-free survival. However, nearly 40% of PBC patients do not respond adequately to PBC and are at higher risk for serious complications when compared to PBC patients with complete response to UDCA. Areas Covered: Here we provide a detailed discussion regarding novel therapeutic agents and potential areas for further investigation in PBC-related research. Expert Opinion: Results of ongoing clinical trials and emerging treatment paradigms for PBC will likely further improve medical management of this disorder in the near future.

Original languageEnglish (US)
Pages (from-to)39-56
Number of pages18
JournalExpert Opinion on Emerging Drugs
Volume21
Issue number1
DOIs
StatePublished - Jan 2 2016

Fingerprint

Cholangitis
Pharmaceutical Preparations
Ursodeoxycholic Acid
Therapeutics
HLA Antigens
Clinical Trials
Immunogenetics
Genome-Wide Association Study
Cholestasis
Expert Testimony
United States Food and Drug Administration
Pruritus
Serum
Autoimmune Diseases
Fatigue
Alkaline Phosphatase
Hepatocellular Carcinoma
Fibrosis
Chronic Disease
Transplants

Keywords

  • bile duct disorders
  • cholestasis
  • outcomes
  • pharmacotherapy
  • Primary biliary cholangitis
  • transplantation
  • ursodeoxycholic acid

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)

Cite this

Emerging drugs for the treatment of Primary Biliary Cholangitis. / Ali, Ahmad H.; Tabibian, James H.; Carey, Elizabeth J.; Lindor, Keith.

In: Expert Opinion on Emerging Drugs, Vol. 21, No. 1, 02.01.2016, p. 39-56.

Research output: Contribution to journalArticle

Ali, Ahmad H. ; Tabibian, James H. ; Carey, Elizabeth J. ; Lindor, Keith. / Emerging drugs for the treatment of Primary Biliary Cholangitis. In: Expert Opinion on Emerging Drugs. 2016 ; Vol. 21, No. 1. pp. 39-56.
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