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Published on: April 6, 2022
Systematic review: microbial manipulation as therapy for primary sclerosing cholangitis
Damjana Bogatic1,2, Robert V Bryant1,2, Kate D Lynch2,3
1Department of Gastroenterology, The Queen Elizabeth Hospital, Woodville, South Australia, Australia.
Background:
Primary sclerosing cholangitis (PSC) is a progressive liver disease with poor prognosis and no effective therapies to prevent progression. An aetiopathological link between PSC and gastrointestinal microbial dysbiosis has been suggested.
Aim:
To evaluate all potential medical therapies which may exert their effect in PSC by modulation of the gut-liver axis.
Methods:
We conducted a comprehensive scoping review of PubMed and Cochrane Library, including all articles evaluating an intervention aimed at manipulating the gastrointestinal microbiome in PSC.
Results:
A wide range of therapies proposed altering the gastrointestinal microbiome for the treatment of PSC. In particular, these considered antibiotics including vancomycin, metronidazole, rifaximin, minocycline and azithromycin. However, few therapies have been investigated in randomised, placebo-controlled trials. Vancomycin has been the most widely studied antibiotic, with improvement in alkaline phosphatase reported in two randomised controlled trials, but with no data on disease progression. Unlike antibiotics, strategies such as faecal microbiota transplantation and dietary therapy can improve microbial diversity. However, since these have only been tested in small numbers of patients, robust efficacy data are currently lacking.
Conclusions:
The gut-liver axis is increasingly considered a potential target for the treatment of PSC. However, no therapies have been demonstrated to improve transplant-free survival. Innovative and well-designed clinical trials of microbiome-targeted therapies with long-term follow-up are required for this orphan disease.
Insights
Microbiome-targeted therapies show promise for primary sclerosing cholangitis (PSC), a progressive liver disease. While some antibiotics improve liver enzymes, robust data on disease progression and transplant-free survival are lacking, necessitating further research.
Area of Science:
- Gastroenterology and Hepatology
- Microbiome Research
- Drug Discovery
Background:
- Primary sclerosing cholangitis (PSC) is a progressive, incurable liver disease.
- A potential link exists between PSC and gut microbial dysbiosis.
- Current therapies for PSC are limited, highlighting the need for novel treatment strategies.
Purpose of the Study:
- To review medical therapies targeting the gut-liver axis in PSC.
- To evaluate interventions that modulate the gastrointestinal microbiome for PSC treatment.
Main Methods:
- A comprehensive scoping review of PubMed and Cochrane Library databases was performed.
- Studies investigating interventions for manipulating the gastrointestinal microbiome in PSC were included.
Main Results:
- Various therapies, including antibiotics (vancomycin, metronidazole, rifaximin, minocycline, azithromycin), aim to alter the gut microbiome in PSC.
- Vancomycin showed alkaline phosphatase improvement in two RCTs but lacked disease progression data.
- Fecal microbiota transplantation and dietary therapy may enhance microbial diversity but require more robust efficacy data due to small patient numbers.
Conclusions:
- The gut-liver axis presents a promising therapeutic target for PSC.
- No current microbiome-targeted therapies have proven to improve transplant-free survival in PSC.
- Well-designed clinical trials with long-term follow-up are essential for evaluating microbiome-targeted therapies in PSC.
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