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.

Mousa, H., Carbone, M., Malinverno, F., Ronca, V., Gershwin, M., Invernizzi, P. (2016). Novel therapeutics for primary biliary cholangitis: Toward a disease-stage-based approach. AUTOIMMUNITY REVIEWS, 15(9), 870-876 [10.1016/j.autrev.2016.07.003].

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

CARBONE, MARCO
Secondo
;
MALINVERNO, FEDERICA;INVERNIZZI, PIETRO
Ultimo
2016

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.
Articolo in rivista - Review Essay
Biologics; Budesonide; Farnesoid X receptor; Primary biliary cholangitis; Ursodeoxycholic acid;
English
870
876
7
Mousa, H., Carbone, M., Malinverno, F., Ronca, V., Gershwin, M., Invernizzi, P. (2016). Novel therapeutics for primary biliary cholangitis: Toward a disease-stage-based approach. AUTOIMMUNITY REVIEWS, 15(9), 870-876 [10.1016/j.autrev.2016.07.003].
Mousa, H; Carbone, M; Malinverno, F; Ronca, V; Gershwin, M; Invernizzi, P
File in questo prodotto:
Non ci sono file associati a questo prodotto.

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10281/141264
Citazioni
  • Scopus 29
  • ???jsp.display-item.citation.isi??? 28
Social impact