Using frozen stool from healthy, unrelated donors was safe and effective in treating patients with serious, relapsing diarrhea caused by Clostridium difficile, according to a new pilot study published in Clinical Infectious Diseases and available online. Known as fecal microbiota transplantation, the treatment was equally effective whether given via a colonoscope or a nasogastric tube. The findings suggest approaches that may make this promising treatment more readily available to patients.
A growing concern, C. difficile causes 250,000 infections requiring hospitalization and 14,000 deaths in the U.S. each year, according to the Centers for Disease Control and Prevention. Infection incidence has tripled in the last 15 years and is responsible for at least $1 billion in excess U.S. medical costs per year.
In an initial feasibility study, Ilan Youngster, MD, and colleagues at Massachusetts General Hospital (MGH) treated 20 patients suffering from repeated episodes of C. difficile infection with fecal microbiota transplantation, which helps restore the normal balance of bacteria in the affected person's gut. Fourteen of the patients were cured after one administration of donated stool. Of the remaining six patients, five received a second administration, and four were cured, for an overall cure rate of 90 percent.
Unlike previous studies, which used fresh stools from donors, the team at MGH worked with frozen material from unrelated donors who had been recruited and screened well ahead of time, a "banking" approach that may help overcome current treatment barriers. "Establishing a repository of prescreened frozen donor stools could make this treatment available for a wider population," the authors noted, as donor recruitment and screening can be lengthy and costly. The Food and Drug Administration is currently determining how it will regulate fecal microbiota transplantation, including what steps clinicians must take to provide it to patients.
For their first administration, patients were assigned to one of two delivery methods, using either a colonoscope or a nasogastric tube, inserted in the nose and down into the stomach. Both appeared to be equally effective. Patients who required a second infusion were allowed to choose between the two methods, and all five chose the nasogastric tube.
"Colonoscopy is a significant procedure requiring sedation," said Elizabeth L. Hohmann, MD, of MGH and a study co-author, while the nasogastric tube method "is an outpatient office procedure."
The study's findings suggest the latter would be a "cost-effective, upper gastrointestinal route" for transplantation, Dr. Hohmann said, particularly for elderly or debilitated patients who may not tolerate a colonoscopy. The researchers are now studying placing the donated material into a frozen capsule, which patients would take orally.