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Published: November 23, 2007
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 study released Thursday.
Among 248 children who received a lung transplant over an 11-year period, only one showed a clear benefit, but 167 were at a higher risk of dying after the procedure, Utah researchers reported in The New England Journal of Medicine.
Julian Allen of Children's Hospital of Philadelphia called the findings "startling" in an editorial in the same journal.
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 surgical procedure is already agonizing for many parents, he wrote with Gary Visner of Children's Hospital Boston, and the new finding "is sure to make an already difficult decision more difficult still."
But 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."
Cystic fibrosis is a devastating genetic defect that affects an estimated 30,000 Americans. It causes a buildup of mucus in many organs, particularly the lungs and the pancreas.
In the lungs, the mucus makes breathing progressively more difficult and leads to life-threatening infections.
A British study of 47 children showed that the procedure improved survival, but an earlier U.S. study by Theodore G. Liou and his colleagues at the University of Utah showed no benefit. Complications associated with the transplants, moreover, account for 12 percent of all deaths among CF 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, they concluded that only one of the 248 who received a transplant benefited from it and that four of the remaining 266 would have benefited had they received a transplant.
Of all the children, the researchers estimated that a transplant posed a significant risk of harm to 315. They also estimated 118 had an insignificant risk of harm but no obvious benefit from a transplant.
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