Novel therapeutics for primary biliary cholangitis: Toward a disease-stage-based approach

Hani S. Mousa, Marco Carbone, Federica Malinverno, Vincenzo Ronca, M. Eric Gershwin, Pietro Invernizzi

Research output: Contribution to journalReview article

Abstract

Primary biliary cholangitis (PBC; previously “primary biliary cirrhosis”) is a cholestatic, putatively autoimmune-mediated liver disease with a clear female preponderance affecting the intrahepatic small and medium-size bile ducts and resulting in bile duct destruction, ductopenia and portal fibrosis that progresses slowly to biliary cirrhosis. Despite suboptimal response in one third of patients treated with ursodeoxycholic acid (UDCA), this remains the only FDA-approved agent for this disease. In this review, we cover recent advances in research that have yielded numerous agents currently at different stages of the drug pipeline, some of which are expected to be approved in the near future. We also discuss accumulating evidence supporting the use of older agents (fibrates and glucocorticoids) as an adjunctive therapy to UDCA in non-responsive patients. We suggest that with the imminent expansion of the therapeutic armamentarium for PBC, a more comprehensive approach – ideally taking into account not only biochemical markers of disease stage – is needed to better select patients in whom these strategies might be most useful. Studies are also needed to compare the relative efficacy of different proposed second-line treatments not only against UDCA monotherapy.

Original languageEnglish
Pages (from-to)870-876
Number of pages7
JournalAutoimmunity Reviews
Volume15
Issue number9
DOIs
Publication statusPublished - Sep 1 2016

Fingerprint

Ursodeoxycholic Acid
Cholangitis
Biliary Liver Cirrhosis
Bile Ducts
Fibric Acids
Glucocorticoids
Liver Diseases
Fibrosis
Therapeutics
Biomarkers
Research
Pharmaceutical Preparations

Keywords

  • Biologics
  • Budesonide
  • Farnesoid X receptor
  • Primary biliary cholangitis
  • Ursodeoxycholic acid

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology

Cite this

Mousa, H. S., Carbone, M., Malinverno, F., Ronca, V., Gershwin, M. E., & Invernizzi, P. (2016). Novel therapeutics for primary biliary cholangitis: Toward a disease-stage-based approach. Autoimmunity Reviews, 15(9), 870-876. https://doi.org/10.1016/j.autrev.2016.07.003

Novel therapeutics for primary biliary cholangitis : Toward a disease-stage-based approach. / Mousa, Hani S.; Carbone, Marco; Malinverno, Federica; Ronca, Vincenzo; Gershwin, M. Eric; Invernizzi, Pietro.

In: Autoimmunity Reviews, Vol. 15, No. 9, 01.09.2016, p. 870-876.

Research output: Contribution to journalReview article

Mousa, HS, Carbone, M, Malinverno, F, Ronca, V, Gershwin, ME & Invernizzi, P 2016, 'Novel therapeutics for primary biliary cholangitis: Toward a disease-stage-based approach', Autoimmunity Reviews, vol. 15, no. 9, pp. 870-876. https://doi.org/10.1016/j.autrev.2016.07.003
Mousa, Hani S. ; Carbone, Marco ; Malinverno, Federica ; Ronca, Vincenzo ; Gershwin, M. Eric ; Invernizzi, Pietro. / Novel therapeutics for primary biliary cholangitis : Toward a disease-stage-based approach. In: Autoimmunity Reviews. 2016 ; Vol. 15, No. 9. pp. 870-876.
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