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.
