Lung transplants no help for cystic fibrosis: study

BOSTON (Reuters) - Children with severe cystic fibrosis are seldom helped by a lung transplant, researchers said on Wednesday in findings they said stunned them.

Only five of the 514 U.S. children on the waiting list for a transplant from 1992 through 2002 lived longer as a clear result of the expensive and complicated operation, Dr. Theodore Liou of the University of Utah in Salt Lake City and colleagues found.

"We were stunned, literally," Liou said in a telephone interview.

"We went into this thinking there would be a number of patients who would benefit and a number who would not, and it would balance out. This study showed it wasn't close to being equal," said Liou, whose findings were reported in the New England Journal of Medicine.

Transplantation is an accepted treatment for severe cases of the fatal genetic disease, which affects about 70,000 people worldwide and creates an abnormally thick mucus that clogs the lungs and digestive system.

But few studies have assessed the effectiveness of lung transplants for helping treat the incurable disease.

Although the Cystic Fibrosis Foundation says that 50 percent of recipients are alive five years after surgery, complications from transplants are responsible for 12 percent of all deaths among people with CF.

"A basic tenet regarding lung transplantation is that it extends life," Dr. Julian Allen of Children's Hospital of Philadelphia and Dr. Gary Visner of Children's Hospital in Boston wrote in a commentary.

"Most workers in transplant centers have encountered patients in whom the results of transplantation seem nothing short of miraculous," they wrote.

However there have been hints that the operation may not be best for sufferers under 18. The five-year survival rate of 33 percent for children is less than for adults or people who have received a transplant for another reason, Allen and Visner said.

But the duo also cautioned that because there has been a change in the rules that determine who gets a new set of lungs, waiting times for the sickest patients have shortened. Thus the Liou findings may no longer apply.

Liou disagreed. The new system of ranking patients will not help people who are unlikely to benefit from a transplant in the first place, he said.

"I think it's going to make people think twice about lung transplants," he said.

Because of improvements in treatment, children who might have died in their mid-teens in the 1970s now survive until their mid-30s.

"The results underscore that sustained, multidisciplinary care rather than lung transplantation is central to longevity in children with cystic fibrosis," the Liou team concluded.

(Reporting by Gene Emery; Editing by Maggie Fox and Vicki Allen)