Gut microbial profiling as a therapeutic and diagnostic target for managing primary biliary cholangitis.

Annarosa Floreani, Sara De Martin, Tsukasa Ikeura, Kazuichi Okazaki, Merrill Eric Gershwin

Research output: Contribution to journalReview articlepeer-review

1 Scopus citations


Introduction: Microbial antigens present in the intestine has been suggested as possible triggers of primary biliary cholangitis (PBC) and it has been demonstrated that the gut microbiome is modified in PBC patients. On this basis, the modulation of the gut microbiome has been proposed as a pharmacological target for PBC management. To provide a state-of-the-art analysis of the preclinical and clinical evidence on this topic, a systematic review of literature in PubMed, Scopus, and Science Direct was conducted (inclusive dates: 2000–2020). Area covered: In particular, several strategies for microbiome modulation have been investigated in both experimental and clinical studies, i.e. dietary interventions, and the administration of probiotics and prebiotics and drugs. Moreover, clinical evidence point to two drugs approved for PBC, i.e. ursodeoxycholic and obeticholic acids, as gut flora modulators. Accordingly, fecal microbiota transplantation is also under evaluation for PBC treatment. On the other hand, typical alterations of the microbiome have been observed in PBC patients, although their diagnostic impact remains to be better evaluated. Expert opinion: The addition of gut microbiome manipulation to standard pharmacological treatments is one important challenge for PBC therapy in the near future. Further studies are needed to ascertain whether microbiome profiling could be considered a diagnostic strategy in PBC.

Original languageEnglish (US)
Pages (from-to)507-514
Number of pages8
JournalExpert Opinion on Orphan Drugs
Issue number12
StatePublished - 2020
Externally publishedYes


  • bile acids
  • metabolomic
  • metagenomics
  • microbial profiling
  • microbiome
  • microbiota
  • obeticholic acid
  • PBC
  • UDCA

ASJC Scopus subject areas

  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
  • Health Policy
  • Pharmacology (medical)


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