Fecal Microbial Transplantation May Treat Ulcerative Colitis

Laurie Barclay, MD

April 11, 2013

Fecal microbial transplantation (FMT) via enema may be effective, tolerable, and feasible for treating children with ulcerative colitis (UC), according to findings from a phase 1 pilot study published online March 29 and in the June issue of the Journal of Pediatric Gastroenterology and Nutrition. FMT involves infusion of human stool from a healthy adult donor into the patient's intestine.

"FMT has been proposed as a promising new treatment option for recurrent [Clostridium] difficile infection and possibly for ulcerative colitis as well," lead author Sachin Kunde, MD, MPH, from Spectrum Health Medical Group, Helen DeVos Children's Hospital in Grand Rapids, Michigan, said in a news release. "We believe that the procedure may restore 'abnormal' bacteria to 'normal' in patients with [UC]. Our short-term study looked at the safety and tolerability of FMT for these patients."

Ten children, aged 7 to 21 years, who had mild to moderate UC, received freshly prepared fecal enemas daily for 5 days. The investigators collected data on tolerability, adverse events, and disease activity during FMT and weekly for 4 weeks thereafter.

At baseline, pediatric UC activity index (PUCAI) ranged from 15 to 65. The investigators considered a reduction in PUCAI by more than 15 to be clinical response, and PUCAI lower than 10 to be clinical remission.

There were no serious adverse events. Self-limiting adverse events were mild cramping, fullness, flatulence, bloating, diarrhea, blood in the stool, and moderate fever. Although 1 child could not retain fecal enemas, average tolerated enema volume in the other 9 children was 165 mL/day.

Clinical response within 1 week occurred in 7 (78%) of the 9 children, including 3 (33%) who had clinical remission and 6 (67%) who maintained clinical response at 1 month. Compared with baseline, median PUCAI significantly improved after FMT (P = .03)

"Patients often face a tough choice between various medications that have significant side effects. Allowing the disease to progress can lead to surgical removal of their colon," Dr. Kunde said in the news release. "Our study showed that fecal enemas were feasible and well-tolerated by children with [UC]. Adverse events were mild to moderate, acceptable, self-limited, and manageable by patients."

He noted the significant challenge of raising awareness that FMT is a socially and medically acceptable treatment option. In this study, patients found FMT more tolerable because they had no visual, olfactory, or physical contact with fecal material other than receiving it as an enema.

"This study opens the doors for an innovative, inexpensive and natural alternative to improve outcomes of this debilitating disease with billions of dollars in health care cost," Dr. Kunde noted in the news release.

Limitations of this study include its open-label design, small sample size, and lack of fecal and mucosal microbial community profiling. The investigators recommend future studies with a larger sample size and longer follow-up period.

"We must further investigate standardization of FMT preparation, ideal donor selection, the best route of administration, and optimal duration or scheduling of FMT to induce and maintain a clinical response," Dr. Kunde concluded in the news release.

"[W]e are still in very early stages of this field and need more evidence in terms of scientifically robust multicenter clinical trials before we can offer this to patients on clinical basis. Caution must be taken that FMT should be offered by centers that follow regulatory guidelines around this new therapy and have facilities and resources available to perform the procedure."

This study was supported by a Helen DeVos Children's Hospital Foundation grant. The authors have disclosed no relevant financial relationships.

J Pediatr Gastroenterol Nutr. Published online March 29, 2013. Abstract