WASHINGTON -- A trio of researchers is calling for a paradigm shift in how preterm infants with a devastating and often deadly disease are treated.
Nearly a half-million babies are born prematurely each year in the U.S., putting them at considerable risk of developing necrotizing enterocolitis (NEC). NEC is a syndrome in which the GI tract becomes infectedand inflamed causing destruction of the bowel. If the infant survives, he or she faces multiple surgeries and possible long-term impairment. The economic impact of treatment alone is reportedly as high as $1 billion a year in the U.S.
Writing in JAMA Pediatrics, Dan Merenstein, MD, of Georgetown University Medical Center, Andi L. Shane, MD, MPH, MSc, of Emory University School of Medicine and Girish Deshpande, FRACP, MSc, of University of Sydney’s Nepean Hospital, say ample evidence exists that probiotics can help prevent NEC in preterm infants and should become a part of standard care. Probiotics are used as a standard of care in many hospitals in Australia, New Zealand, Finland, Italy and Japan.
In 2010, a study review by the American Academy of Pediatrics acknowledged the evidence that probiotics prevent NEC, but stated, “more studies are needed.”
The authors say that recommendation conflicts with a conclusion by Cochrane Reviews, an international network that provides evidence-based reviews of current research. In 2011, Cochrane Reviews identified 16 clinical trials examining the use of probiotics to prevent NEC and concluded, “…our updated review of available evidence supports a change in practice.”
The authors ask, “How much more evidence is needed…?”
Merenstein, Shane and Deshpande advocate for more research, but say “regulatory roadblocks” have hindered clinical trials with probiotics. They say it is not feasible to conduct studies in the U.S. due to requirements for data from populations in addition to the one of interest.
“Although probiotics products are sold at every supermarket and are present on hospital formularies, the [U.S. Food and Drug Administration] demands that probiotic research that makes a health claim obtain an investigational new drug (IND) application,” they write.
The authors contrast the regulatory requirements to study probiotics with a practice in which stool is transplanted into patients with Clostridium difficile infections. “Research involving stools does not require an IND,” the authors write.
“We propose that the probiotic administration to prevent NEC be studied in a comparative effectiveness design…” without the need for an IND, the authors say. “The other option is to continue with the standard of care, in which no new products are provided; something we believe is ethically unacceptable.”
Merenstein has served as an expert witness for General Mills, Nestle Healthcare and Bayer Healthcare on matters related to probiotics. Deshpande has received research grant support from Fresenius Kabi and Baxter Health Care for investigator initiated intravenous lipid randomized trials. Shane has received research grant support from The Gerber Foundation. All authors have received travel and conference support from the International Scientific Association for Probiotics and Prebiotics.
About Georgetown University Medical Center
Georgetown University Medical Center is an internationally recognized academic medical center with a three-part mission of research, teaching and patient care (through MedStar Health). GUMC’s mission is carried out with a strong emphasis on public service and a dedication to the Catholic, Jesuit principle of cura personalis – or “care of the whole person.” The Medical Center includes the School of Medicine and the School of Nursing & Health Studies, both nationally ranked; Georgetown Lombardi Comprehensive Cancer Center, designated as a comprehensive cancer center by the National Cancer Institute; and the Biomedical Graduate Research Organization (BGRO), which accounts for the majority of externally funded research at GUMC including a Clinical Translation and Science Award from the National Institutes of Health.