Using Evidence to Kick the “Ick” Factor

Posted by The Evidence Blog on January 22, 2013

Desperate times sometimes call for desperate measures, especially in the world of medicine. And throughout history, some of those measures have had a pretty high “ick” factor.

If you lived in China at the turn of the 11th century and wanted to avoid contracting smallpox, you’d have been advised to blow small pox scabs up your nose. (While the technique had a 0.5% to 2.0% mortality rate, it was considerably less than the 20% to 30% mortality rate of the disease itself!) In 1921, maggots were introduced at Johns Hopkins University as a highly successful treatment for open wounds. (Eighty-three years later, the FDA approved maggots as a therapy for deep wound healing)

And in 1958, a team of Colorado researchers reported that an unusual procedure known as fecal bacteriotherapy held promise for fighting a stubborn gastrointestinal infection caused by an unknown organism, now known to be Clostridium difficile (C. difficile). Also referred to as a fecal transplant, the procedure involves the infusion of a healthy donor’s feces into an infected patient’s gut to restore a normal balance of healthy bacteria.

Why would anyone embrace such an “icky” procedure?

C. difficile infection (CDI) is typically caused by taking antibiotics that wipe the gut clean of its normal bacterial balance. Without normal levels of healthy bacteria, the gut becomes susceptible to C. difficile, especially in the hospital setting. Characterized by profuse diarrhea, uncontrollable vomiting, and high fever, the infection often does not respond to antibiotics. CDIs kill approximately 14,000 people annually in the United States, they are very difficult to treat, and the failure rates for antibiotic therapy are high. In addition, the treatment costs are estimated to exceed $1 billion a year.

In recent years, a more deadly, antibiotic-resistant form of the bacteria has emerged. The antibiotics approved to treat the infection are expensive and, as importantly, are not effective in up to 25% of patients. Additionally, the estimated efficacy of antibiotic therapy for a first recurrence is 60%, which declines further in patients with multiple recurrences.

It appears that widespread use of fecal bacteriotherapy has been limited by a paucity of evidence-based data rather than by patient preference. Although patients find the concept unappealing, they are open to considering the procedure to treat recurrent CDI, especially when recommended by a physician. Clinical uptake of the procedure by the medical community has been most hampered by a lack of clinical data showing that the procedure is more effective than antibiotics—the current care standard.

Until now, that is. A recent study published in the New England Journal of Medicine reports that among 42 patients with multiple recurrences of CDI, the infusion of donor feces was significantly more effective for the treatment of recurrent CDI than the use of vancomycin or vancomycin with bowel lavage. Success was defined as the resolution of diarrhea associated with CDI without relapse after 10 weeks. Overall, fecal infusions resulted in a cure rate of 94% (15 of 16 patients). Among those who received vancomycin or vancomycin plus lavage, the cure rates were 31% and 23%, respectively. Because so many people in the control group experienced relapses of CDI, the trial was stopped early. Of note is that among 18 patients who relapsed and subsequently underwent transfusion, 15 were also cured (83%).

Importantly, there was no significant difference in adverse events among the study groups, except for mild gastrointestinal symptoms in the transplant group on the infusion day. After 2 weeks, the patients who had undergone the transfusion developed an increased diversity of fecal bacteria similar to their donors.

As a company focused on evidence-based medicine, we’re pleased to see this type of rigorous research applied to a most “icky” procedure. The medical literature continues to emphasize the importance of evidence-based research as one way to reduce the volume of unnecessary procedures, medications, and other treatments that drive up costs without improving patient care. This is an example of how rigorous evidence for a controversial and “icky” procedure—if implemented in appropriate patients at the right time—may diminish pain and suffering, reduce costs, and improve patient outcomes.

Topics: Hayes Blog

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