Rifaximin or Saccharomyces boulardii in heart failure with reduced ejection fraction: Results from the randomized GutHeart trial

EBioMedicine. 2021 Aug:70:103511. doi: 10.1016/j.ebiom.2021.103511. Epub 2021 Jul 28.

Abstract

Background: The gut microbiota represents a potential treatment target in heart failure (HF) through microbial metabolites such as trimethylamine N-oxide (TMAO) and systemic inflammation. Treatment with the probiotic yeast Saccharomyces boulardii have been suggested to improve left ventricular ejection fraction (LVEF).

Methods: In a multicentre, prospective randomized open label, blinded end-point trial, we randomized patients with LVEF <40% and New York Heart Association functional class II or III, despite optimal medical therapy, to treatment (1:1:1) with the probiotic yeast Saccharomyces boulardii, the antibiotic rifaximin, or standard of care (SoC) only. The primary endpoint, the baseline-adjusted LVEF at three months, was assessed in an intention-to-treat analysis.

Findings: We enrolled a total of 151 patients. After three months' treatment, the LVEF did not differ significantly between the SoC arm and the rifaximin arm (mean difference was -1•2 percentage points; 95% CI -3•2 - 0•7; p=0•22) or between the SoC arm and the Saccharomyces boulardii arm (mean difference -0•2 percentage points; 95% CI -2•2 - 1•9; p=0•87). We observed no significant between-group differences in changes in microbiota diversity, TMAO, or C-reactive protein.

Interpretation: Three months' treatment with Saccharomyces boulardii or rifaximin on top of SoC had no significant effect on LVEF, microbiota diversity, or the measured biomarkers in our population with HF.

Funding: The trial was funded by the Norwegian Association for Public Health, the Blix foundation, Stein Erik Hagen's Foundation for Clinical Heart Research, Ada og Hagbart Waages humanitære og veldedige stiftelse, Alfasigma, and Biocodex.

Keywords: Antibiotics; Heart failure; Inflammation; Microbiota; Probiotics; Trimethylamine N-oxide.

Publication types

  • Clinical Trial, Phase II
  • Multicenter Study
  • Randomized Controlled Trial

MeSH terms

  • Aged
  • Anti-Bacterial Agents / therapeutic use*
  • Cardiac Output
  • Exercise Test
  • Female
  • Gastrointestinal Microbiome*
  • Heart Failure / drug therapy
  • Heart Failure / microbiology*
  • Heart Failure / physiopathology
  • Heart Failure / therapy
  • Humans
  • Male
  • Middle Aged
  • Probiotics / therapeutic use*
  • Rifaximin / therapeutic use*
  • Saccharomyces boulardii / pathogenicity*
  • Standard of Care

Substances

  • Anti-Bacterial Agents
  • Rifaximin