PMID- 26901615
OWN - NLM
STAT- MEDLINE
DCOM- 20161011
LR  - 20161230
IS  - 1744-7623 (Electronic)
IS  - 1472-8214 (Linking)
VI  - 21
IP  - 1
DP  - 2016
TI  - Emerging drugs for the treatment of Primary Biliary Cholangitis.
PG  - 39-56
AB  - 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.
FAU - Ali, Ahmad H
AU  - Ali AH
FAU - Tabibian, James H
AU  - Tabibian JH
FAU - Carey, Elizabeth J
AU  - Carey EJ
FAU - Lindor, Keith D
AU  - Lindor KD
LA  - eng
PT  - Journal Article
PT  - Review
PL  - England
TA  - Expert Opin Emerg Drugs
JT  - Expert opinion on emerging drugs
JID - 101135662
RN  - 0 (Autoantibodies)
RN  - 724L30Y2QR (Ursodeoxycholic Acid)
RN  - EC 3.1.3.1 (Alkaline Phosphatase)
SB  - IM
MH  - Alkaline Phosphatase/blood
MH  - Animals
MH  - Autoantibodies/immunology
MH  - Cholangitis/*drug therapy/immunology/physiopathology
MH  - Chronic Disease
MH  - Disease Progression
MH  - *Drug Design
MH  - Female
MH  - Genome-Wide Association Study
MH  - Humans
MH  - Liver Cirrhosis, Biliary/*drug therapy/immunology/physiopathology
MH  - Male
MH  - Mitochondria/immunology
MH  - Ursodeoxycholic Acid/therapeutic use
EDAT- 2016/02/24 06:00
MHDA- 2016/10/12 06:00
CRDT- 2016/02/23 06:00
PHST- 2016/02/23 06:00 [entrez]
PHST- 2016/02/24 06:00 [pubmed]
PHST- 2016/10/12 06:00 [medline]
AID - 10.1517/14728214.2016.1150999 [doi]
PST - ppublish
SO  - Expert Opin Emerg Drugs. 2016;21(1):39-56. doi: 10.1517/14728214.2016.1150999.