CHICAGO – A 10-year registry study aims to gather clinical and patient-reported outcomes on 4,000 adult and pediatric patients who undergo fecal microbiota transplant in the United States, officials of the American Gastroenterological Association announced during Digestive Disease Week®.
The AGA Fecal Microbiota Transplantation National Registry will be the first study to assess both short- and long-term patient outcomes associated with fecal microbiota transplant (FMT) in both adults and children, Colleen Kelly, MD, said in an video interview. Most subjects will have received FMT for recurrent or refractory Clostridium difficile infections – the only indication for which Food and Drug Administration currently allows independent clinician action. But the investigational uses of FMT are expanding rapidly, and patients who undergo the procedure during any registered study will be eligible for enrollment, said Dr. Kelly, co-chair of the study’s steering committee.
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The study’s primary objectives are short- and long-term safety outcomes, said Dr. Kelly of Brown University, Providence, R.I. While generally considered quite safe, short-term adverse events have been reported with FMT, and some of them have been serious – including one death from aspiration pneumonia in a patient who received donor stool via nasogastric tube (Clin Infect Dis. 2015 Mar;61[1]:136-7). Other adverse events are usually self-limited but can include low-grade fever, abdominal pain, distention, bloating, and diarrhea.
Researchers seek to illuminate many of the unknowns associated with this relatively new procedure. Scientists are only now beginning to unravel the myriad ways the human microbiome promotes both health and disease. Specific alterations, for example, have been associated with obesity and other conditions; there is concern that transplanting a new microbial population could induce a disease phenotype in a recipient who might not have otherwise been at risk.
With the planned cohort size and follow-up period, the study should be able to detect any unanticipated adverse events that occur in more than 1% of the population, Dr. Kelly said. It will include a comparator group of patients with recurrent or refractory C. difficile infection from a large insurance claims database to allow comparison between patients treated with FMT and those treated with antibiotics only.
The registry study also aims to discover which method or methods of transplant material delivery are best, she said. Right now, there are a number of methods (colonoscopy/sigmoidoscopy, enema, upper gastrointestinal endoscopy, nasogastric or nasoduodenal tube, and capsules), and no consensus on which is the best. As indications for FMT expand, there may be no single best method. The approach will probably be matched to the disorder being treated, and the study may help illuminate this as well.
For the first 2 years after a transplant, clinicians will follow patients and enter data into the registry. After that, an electronic patient-reported outcomes system will automatically contact the patient annually for follow-up information by email or text message. When patients enter their data, they can access educational material that will help keep them up-to-date on potential adverse events.
The study will also include a biobank of stool samples obtained during the procedures, hosted by the American Gut Project and the Microbiome Initiative at the University of California, San Diego. This arm of the project will analyze the microbiome of 3,000 stool samples from recipients, both before and after their transplant, as well as the corresponding donors whose material was used in the fecal transplant.
The registry study, a project of the AGA Center for Gut Microbiome Research and Education, is funded by a $3.3 million grant from the National Institute of Allergy and Infectious Diseases. It will be conducted in partnership with the Crohn’s and Colitis Foundation, Infectious Diseases Society, and the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition.
The registry study currently is accepting applications. Physicians who perform FMT for C. difficile infections, and centers that conduct FMT research for other potential indications, can fill out a short survey to indicate their interest.
Digestive Disease Week® is jointly sponsored by the American Association for the Study of Liver Diseases (AASLD), the American Gastroenterological Association (AGA) Institute, the American Society for Gastrointestinal Endoscopy (ASGE) and the Society for Surgery of the Alimentary Tract (SSAT).
This article was updated June 8, 2017.
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