As reported by The Hartford Courant, May 25, 2006.

Study Resolves Preemie Debate

Two Options Yield Equal Results

By Hilary Waldman

When micro-preemie Jake Hoyt's intestines failed on the 11th day of his life, doctors at Connecticut Children's Medical Center took their best guess about how to save the East Haddam boy from one of the most insidious killers of premature babies.

On Jan. 4, 2004, Dr. Richard Weiss inserted a straw-size drain in Jake's gut, allowing pus and stool to drain from the section of his intestine that, for some reason, had ruptured.

Like many pediatric surgeons, Weiss believed that the drain was a safer approach than surgically removing the dead section of intestine in a baby as small as Jake, who was born four months ahead of schedule at 1-pound, 10-ounces.

As it turned out, Weiss was tossing a coin.

An article published today in the New England Journal of Medicine suggests that the survival rate for newborns with the intestinal disease is the same whether they are treated with surgery or a drain. And the track record for both procedures is not good.

"The real message is that once the disease has progressed to this point, we're really just rearranging the deck chairs on the Titanic," said Dr. R. Lawrence Moss, surgeon-in-chief at Yale-New Haven Children's Hospital and the lead author of the article.

Moss led a team of researchers at 15 pediatric medical centers in a groundbreaking study aimed at taking the guesswork out of treating babies with the intestinal disease necrotizing enterocolitis, the most common surgical emergency among premature infants.

An estimated 5,000 to 10,000 babies in neonatal intensive care units across the country suffer from the condition each year, and one-quarter to one-half die.

Many babies recover after a few days on antibiotics. But the intestine tears in about the same number of newborns, requiring quick action by a pediatric surgeon.

For 30 years, doctors have argued over the best approach - drain or surgery - with the choice largely governed by tradition or physician preference. But there was no strong scientific proof that one approach was better.

The new study, believed to be the first of its kind in the nation, provided a head-to-head comparison. Over the last four years, when a baby with NEC was rushed to the operating room at a participating hospital, the surgeon was handed a sealed envelope. Half of the randomly distributed envelopes instructed surgeons to insert a drain. The other half called for surgical removal of the dead length of intestine.

Moss said he had hoped that a clear winner would emerge. But he is not disappointed by the results.

Beyond the lingering uncertainty for babies with NEC, the study showed for the first time that it is possible to ethically take a more scientific approach to evaluating medical treatment in children.

Because of a reluctance to experiment on kids, many surgical decisions for children continue to be based on good faith belief and observation, rather than air-tight evidence, Moss said.

Doctors still don't know, for example, whether every child needs surgery for appendicitis, if certain children with complications from pneumonia do better with surgery or just antibiotics and if all babies born with a hole in the diaphragm must be put on a heart/lung bypass machine to survive.

"We don't have answers to any of these questions," Moss said.

"If you can do [a head-to-head clinical trial] in a group of premature babies who need emergency surgery because they're dying, I would argue you could do it for any question."

With about 500,000 premature babies born in the United States each year and with that number climbing, Moss said the study also should send researchers running back to the drawing board in search of better answers for babies with NEC.

"We are completely unable to predict which babies are going to go on to [have their intestines] perforate and which will get better after a few days on antibiotics," Moss said.

In search of clues, researchers at the 15 participating institutions have started a data base documenting the progress of each baby who displays NEC symptoms in their neonatal intensive care units. Once enough information is collected, they will look for patterns that they hope will help them make better predictions in the future. They also will look for genetic markers to see if having a bowel perforation may be as preordained as is having blue or brown eyes.

If the researchers are able to predict an intestinal tear, doctors may be able to treat susceptible babies with new therapies such as growth factors or beneficial bacteria known as pro-biotics, which look promising in laboratory tests.

Doctors who treat premature babies agree that NEC is one of the last frontiers in the field of saving premature babies.

Over the last 30 years, improvements in medicine and technology have saved babies such as Jake Hoyt, who was born at 24 weeks gestation, just a little more than half the 40 weeks it takes for a fetus to become a full-term baby.

Brain damage or death from bleeding in the brain, blindness caused by high doses of oxygen in the neonatal intensive care unit, and lung damage from aggressive use of ventilators and from simple underdevelopment are complications that have been significantly reduced.

"I've been practicing neonatology for 25 years, and [NEC] is the one area we haven't made much progress in," said Dr. Victor Herson, medical director of the neonatal intensive care unit at Connecticut Children's Medical Center, which did not participate in the study.

For Jake Hoyt, the surgeon's gamble on the drain paid off. About a month after it was inserted, Jake's bowel began to function normally. He tolerated breast milk and started gaining weight. After 102 days in the hospital, his parents took him home on April 6, 2004 - nine days before his due date. Jake weighed 5 pounds, 7 ounces and was 19 inches long.

Today, Jake appears to have beaten the odds. At 2, he loves to run around and have conversations in three- and four-word sentences. He enjoys books, pretending to drive his grandfather's tractor and using tools to "repair" anything he can get his hands on.

Jake's mother, Rene Roselle Hoyt, who just earned a doctorate in special education, said she respects the outcome of the clinical trial. But she's glad Jake got the drain.

"I appreciate having been given the drain because the outcomes would have been so much more complex in terms of feeding him" if he had had the surgery, she said. "The doctors did a great job, and we were just blessed."

Dr. Naveed Hussain, medical director of the neonatal intensive care unit at the University of Connecticut Health Center, said he wished the trial had come up with a clear answer. But he said its immediate value may be giving doctors a clear conscience.

Hussain said surgeons at the health center frequently choose the drain for tiny babies such as Jake who may be too frail to tolerate the anesthesia, blood loss and long recovery necessary for surgery. But they often wonder if they did the right thing. The health center also did not participate in Moss' trial.

"This is helpful because you always wondered if you just put in a drain, some people think that was second-class treatment," Hussain said. "This confirms that if you don't want to open the baby up, you can still do the drain and get good results."