A significant aspect of the study is that it is the first multi-center randomized controlled trial to be completed comparing two operations in children, said the lead author, Larry Moss, M.D., chief of pediatric surgery in the Department of Surgery.
"Surgery is one of the most dramatic and potentially life-altering events in a patient's medical life, yet a remarkable amount of surgical practice is based upon historical tradition rather than scientific evidence," Moss said. "The vast majority of research on surgical conditions involves a single group of surgeons reporting upon their personal experience at one institution."
"This report argues against conventional wisdom that randomized studies cannot be effectively utilized to compare treatments for acutely life-threatening conditions," he said. "It proves that despite the biases against surgical trials, they can be completed with appropriate commitment by surgeons and their institutions."
NEC is a severe inflammatory disease of the intestine afflicting 5,000 to 10,000 premature infants in the United States each year. In its most severe form NEC results in perforation of part of the intestine, which requires emergency surgery that can be life-saving.
For more than 25 years surgeons have used two radically different operations for these babies. The first and more aggressive, laparotomy and bowel resection, involves a large abdominal incision with removal of all affected intestine and creation of a stoma, which means bringing the end of the intestine through the abdomen to drain into a bag. The alternative option, peritoneal drainage, involves making a Ľ inch incision in the lower abdomen and placing a small drain allowing egress of stool and pus from the abdomen without removing any intestine.
The trial led by Moss included 117 premature infants at 15 leading pediatric academic medical centers from the United States and Canada. When a baby developed perforated NEC at a study site, parents were counseled by the operating surgeons and offered enrollment into the trial. If they agreed, the operation their child received was assigned randomly.
"The study found that patient survival and other major outcomes for the two drastically different operations were virtually identical," he said. "After 30 years of debate over which procedure is best, the first true scientific experiment addressing this question suggests that the method of the surgery may not be the important aspect of treatment."
As a next step, Moss, working with six academic medical centers, is directing collection of clinical and biological information for a database to identify which babies with NEC are at greatest risk of perforation. The team is collaborating with other researchers who are developing promising new therapies that can be tested in this targeted group of patients.
"Since the study compared the most aggressive surgical treatment with the least invasive and found no difference, it appears unlikely that ongoing focus on the details of the operation is likely to improve survival in these patients," he said.
Moss also has developed and tested a set of clinical research reporting guidelines for surgery that revealed there was a lack of evidence for many surgical procedures in children. The tool was then validated on a large sample of several hundred studies. As a result of this work, the Journal of Pediatric Surgery has adopted the guidelines and is using them for all clinical research published in the journal.
NEJM Vol. xx: pp-pp (May 25, 2006)
Last reviewed: By John M. Grohol, Psy.D. on 21 Feb 2009
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