FMT Safe But Not Statistically Significant in Reducing Infections in AML, Allogeneic HSCT

By Leah Sherwood - Last Updated: July 28, 2023

Fecal microbiota transplantation (FMT) was shown to be safe and to partially improve the imbalance of gut bacteria in patients with acute myeloid leukemia (AML) and recipients of allogeneic hematopoietic stem cell transplantation (HSCT), according to a phase II study.

However, the technique did not decrease the rate of infections in these patients, according to the researchers, led by first author Armin Rashidi, MD, PhD, of the Department of Medicine at the University of Minnesota in Minneapolis. The study’s senior author is Christopher Staley, PhD, of the Department of Surgery at the University of Minnesota.

“In allogeneic [HSCT] recipients and patients with AML, third-party FMT was safe and ameliorated intestinal dysbiosis, but did not decrease infections,” they wrote in the paper, which was published in the Journal of Clinical Oncology.

Phase II Study

In the randomized, double-blind, placebo-controlled study, Dr. Rashidi and colleagues compared the outcome of FMT in patients with AML receiving induction chemotherapy (26 patients) and HSCT recipients (74 patients). Patients were randomly assigned 2:1 to received oral encapsulated FMT or placebo. The primary endpoint of the study was to determine the number of infections that occurred within four months after the patients received their respective treatments, and patients were monitored for nine months.

In the HSCT cohort, the four-month infection density was .74 and .91 events per 100 patient-days in the FMT and placebo arms, respectively (infection rate ratio, .83; 95% CI, .48-1.42; P=.49). In the AML cohort, the four-month infection density was .93 in the FMT arm and 1.25 in the placebo arm, with an infection rate ratio of .74 (95% CI, .32-1.71; P=.48).

The study concluded that FMT was safe, and patients who received FMT had a lower infection rate compared with those who received the placebo. However, the difference in infection rates between the treatment and placebo groups was not statistically significant.

Dr. Rashidi and colleagues wrote that the “novel findings from this trial will inform future development of FMT trials.”


Rashidi A, Ebadi M, Rehman TU, et al. Randomized double-blind phase II trial of fecal microbiota transplantation versus placebo in allogeneic hematopoietic cell transplantation and AML. J Clin Oncol. 2023. doi:10.1200/JCO.22.02366

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