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Lung transplants for children questioned

Lung transplants - a treatment of last resort for cystic fibrosis - are rarely beneficial to children with that condition and are often harmful, according to a new study.

Among 248 children who received a lung transplant over 11 years, one showed a clear benefit while 167 were at a higher risk of dying after the procedure, Utah researchers reported in the new issue of The New England Journal of Medicine.

In an editorial in the same journal, Dr. Julian Allen of The Children’s Hospital of Philadelphia called the findings “startling.” He said the study makes it clear that “although transplantation may improve quality of life, it may not improve survival.”

Deciding whether to choose the expensive and time-consuming surgery is agonizing for many parents, he wrote with Dr. Gary Visner of Children’s Hospital Boston, adding that the new finding “is sure to make an already difficult decision more difficult still.”

But Dr. Vaughn Starnes of Children’s Hospital Los Angeles said he “vehemently disagrees” with the report’s conclusions.

He said the researchers obtained bad results because they lumped together data from hospitals that specialize in transplants in children and those that specialize in adults and treat only a few children.

“Our children are deriving a significant benefit in survival,” he said. “We have patients who are alive 12 to 14 years out after the procedure.”

A half-century ago, most children with the disorder died in their first year or two. By the 1970s, most died in their teens. Today, the median life expectancy is 36 years, according to the Cystic Fibrosis Foundation, which paid for the study. Within a few years there will be more adults with the disease than children.

Studies in adults have shown that lung transplants can prolong life and produce what surgeons have called “near-miraculous” improvements in daily living. But previous studies in children have been mixed.

Better knowledge about efficacy is crucial for a procedure that costs up to $400,000 during the first year and an additional $80,000 a year for the rest of the patient’s life.

A British study of 47 children showed that the procedure improved survival, but an earlier U.S. study by Dr. Theodore Liou and his colleagues at the University of Utah showed no benefit. Complications associated with the transplants, moreover, accounted for 12 percent of all deaths among cystic-fibrosis patients.

To provide a more definitive understanding of benefits and risks, Liou’s team studied the 514 children who were on the U.S. waiting list for lung transplants from 1992 through 2002. Of those, 248 underwent the procedure, with 33 percent of them surviving for at least five years.

Analyzing at least 26 health variables, researchers concluded that only one who received a transplant benefited from it and that four of the remaining 266 would have benefited had they received a transplant.

They did not directly assess quality of life.

The study results were obtained before a 2005 change in lung-allocation criteria that now awards donor lungs to the sickest patients rather than those who have been waiting the longest.

The change might lead to a higher success rate for the procedure, said Dr. Preston Campbell of the Johns Hopkins Hospital.

Campbell, executive vice president for medicine at the Cystic Fibrosis Foundation, added that he envisioned a time such calculations of survival and benefit will be unnecessary. “The goal of the foundation is to reach a day when no child will need a lung transplant,” he said.

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