Lung transplants in youths harmful, study finds.
Reported November 22, 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 today.
Among 248 children who received a lung transplant over an 11-year period, only one clearly benefited while 167 were at a higher risk of dying after the procedure, Utah researchers reported in the New England Journal of Medicine.
Dr. Julian Allen of Children’s Hospital of Philadelphia called the findings “startling” in an editorial in the journal. He said the study made 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 agonizing for many parents, he wrote in the editorial with Dr. Gary Visner of Children’s Hospital Boston, and the new finding “is sure to make an already difficult decision more difficult still.”
But Dr. Vaughn Starnes of Childrens Hospital Los Angeles said he “vehemently disagrees” with the report’s conclusions. He believed the researchers got bad results because they lumped together data from hospitals that specialize in transplants in children and those that specialize in adults and treat a few children.
“Our children are deriving a significant benefit in survival,” Starnes said. “We have patients who are alive 12 to 14 years out after the procedure.”
Cystic fibrosis is a genetic defect that affects about 30,000 Americans. It causes a buildup of mucus in many organs, particularly the lung and the pancreas. In the lungs, the mucus makes breathing progressively more difficult and leads to life-threatening infections.
A half-century ago, most children with the disorder died in their first year or two of life. By the 1970s, most died in their teens. Today, the median life expectancy is 36 years, according to the Cystic Fibrosis Foundation.
Studies of adults have shown that lung transplants can prolong life and produce what surgeons have called “near miraculous” improvements in daily living. But previous studies of children have been mixed.
Better knowledge about efficacy is crucial for a procedure that costs as much as $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 led by Dr. Theodore G. Liou at the University of Utah showed no benefit. Complications associated with the transplants, moreover, account for 12% of all deaths among cystic fibrosis patients.
To provide a more definitive understanding of benefits and risks, Liou’s team expanded its study to 514 children on the U.S. waiting list for lung transplants from 1992 through 2002. Of those, 248 actually underwent the procedure, with 33% of them surviving for at least five years.
Analyzing at least 26 different health variables, researchers concluded that only one of the 248 who actually received a transplant benefited from it and that four of the remaining 266 would have benefited had they received a transplant.
Of all the 514 children, the researchers estimated that a transplant posed a significant risk of harm to 315. They also estimated that 118 had an insignificant risk of harm but no obvious benefit from a transplant.
They did not directly assess quality of life.
The study results predate a 2005 change that awards donor lungs to the sickest patients rather than those who have been waiting the longest.
The change may lead to a higher success rate for the procedure, said Dr. Preston Campbell of the Johns Hopkins Hospital, who is also executive vice president for medicine at the Cystic Fibrosis Foundation, which funded the study.