Lung transplants no help for cystic fibrosis: study

Lung transplants no help for cystic fibrosis: study
An empty operating theatre is seen at the University hospital in western German city of Bonn March 16, 2006. Children with severe cystic fibrosis are seldom helped by a lung transplant, researchers said on Wednesday in findings they said stunned them. (Ina Fassbender/Reuters)BOSTON (Reuters) - Children with severe cystic fibrosis areseldom helped by a lung transplant, researchers said onWednesday in findings they said stunned them.
Only five of the 514 U.S. children on the waiting list fora transplant from 1992 through 2002 lived longer as a clearresult of the expensive and complicated operation, Dr. TheodoreLiou of the University of Utah in Salt Lake City and colleaguesfound.
"We were stunned, literally," Liou said in a telephoneinterview.
"We went into this thinking there would be a number ofpatients who would benefit and a number who would not, and itwould balance out. This study showed it wasn't close to beingequal," said Liou, whose findings were reported in the NewEngland Journal of Medicine.
Transplantation is an accepted treatment for severe casesof the fatal genetic disease, which affects about 70,000 peopleworldwide and creates an abnormally thick mucus that clogs thelungs and digestive system.
But few studies have assessed the effectiveness of lungtransplants for helping treat the incurable disease.
Although the Cystic Fibrosis Foundation says that 50percent of recipients are alive five years after surgery,complications from transplants are responsible for 12 percentof all deaths among people with CF.
"A basic tenet regarding lung transplantation is that itextends life," Dr. Julian Allen of Children's Hospital ofPhiladelphia and Dr. Gary Visner of Children's Hospital inBoston wrote in a commentary.
"Most workers in transplant centers have encounteredpatients in whom the results of transplantation seem nothingshort of miraculous," they wrote.
However there have been hints that the operation may not bebest for sufferers under 18. The five-year survival rate of 33percent for children is less than for adults or people who havereceived a transplant for another reason, Allen and Visnersaid.
But the duo also cautioned that because there has been achange in the rules that determine who gets a new set of lungs,waiting times for the sickest patients have shortened. Thus theLiou findings may no longer apply.
Liou disagreed. The new system of ranking patients will nothelp people who are unlikely to benefit from a transplant inthe first place, he said.
"I think it's going to make people think twice about lungtransplants," he said.
Because of improvements in treatment, children who mighthave died in their mid-teens in the 1970s now survive untiltheir mid-30s.
"The results underscore that sustained, multidisciplinarycare rather than lung transplantation is central to longevityin children with cystic fibrosis," the Liou team concluded.
(Reporting by Gene Emery; Editing by Maggie Fox and VickiAllen)

Lung Transplants May Not Help Most Cystic Fibrosis Children

Lung Transplants May Not Help Most Cystic Fibrosis Children
WEDNESDAY, Nov. 21 (HealthDay News) -- Hardly any of the childrenwho receive lung transplants because of severe damage caused by cysticfibrosis benefit from the risky operation, a study concludes.
It is a controversial conclusion, made more controversial because thetransplant rules have been changed since the study was done, said Dr.Theodore G. Liou, associate professor of internal medicine at theUniversity of Utah, and lead author of a report in the Nov. 22 NewEngland Journal of Medicine.
CF is a hereditary disease in which the lungs and digestive tractbecome clogged with mucus. People with CF die at a relatively young age.Previous studies have shown some survival benefit for adult cysticfibrosis sufferers who received lung transplants as a last resort, Liousaid.
The new report is one of very few looking at such transplants inchildren, he said. "In 2005, we published one [study] showing nodifference in outcome between patients who were transplanted and those whowere not, although they were equally sick as far as we could tell," Liousaid. "That got us into a lot of trouble, because people didn't like theresults."
The new study looked at 514 children with cystic fibrosis who were onthe waiting list for lung transplants from 1992 to 2002. A total of 248 ofthe children did undergo the procedure during the study period.
The researchers found a significant estimated benefit for only five ofthose who had transplants, with "a significant risk of harm" associatedwith lung transplantation for 315 of the young patients, meaning thatother treatment would have benefited them more.
The process has changed since the study was conducted, partly in waysthat work against transplantation, Liou noted. Children selected for lungtransplants now are first put on an intensive course of treatment intendedto strengthen them for the surgery, he said, and "conventional treatmentshave gotten to be very good," he added.
The rules for actual performance of a transplant have also changed,said Dr. Julian L. Allen, chief of the division of pulmonary medicine atthe cystic fibrosis center at the Children's Hospital of Philadelphia. Heis also co-author of an accompanying editorial in the journal.
Until 2005, all children awaiting a lung transplant were placed on asingle list, receiving organs as they became available. Now, the decisionto transplant includes consideration of the patient's condition, withsicker children getting the operation sooner, Allen said.
"In some cases, children who were put on intensive therapy weredeferred, because they got better," he said. "There was something aboutthe children in that group who didn't get transplanted that made them getbetter."
The bottom line, Liou said, is that a decision that has always beencomplex has gotten even more so. "Maybe people will pay attention and tryto be more careful about selecting patients for lung transplants," hesaid.
"You need to be careful about who you refer," Allen said. "You need tosee if the results in this study hold true under the current rules. Also,the quality of life after a transplant has to be looked at. We need futurestudies that evaluate the quality of life."
One complicating factor with younger recipients of lung transplants isthe need to be sure that they adhere to the strict regimen needed toprevent rejection of the organ, Allen added. The better success rate witholder cystic fibrosis recipients is due partly to their better ability tofollow instructions, he said.
More information
There's more on cystic fibrosis at the Cystic FibrosisFoundation.

Lung transplant rarely prolongs life in CF

Lung transplant rarely prolongs life in CF
NEW YORK (Reuters Health) - Lung transplantation inchildren with cystic fibrosis (CF) is not likely to prolonglife and may do more harm than good, according to a look backat essentially the entire U.S. pediatric experience with lungtransplantation for cystic fibrosis for the period 1992 through2002.
According to a report in November 22 issue of The NewEngland Journal of Medicine, a total of 248 of the 514 childrenwith cystic fibrosis who were on the waiting list during the10-year-period underwent lung transplantation.
"Children undergoing lung transplant did much worse thanexpected," Dr. Theodore G. Liou of the University of Utah, SaltLake City, told Reuters Health.
"We fully expected, at the start of the study, that wewould find a group that did well and others that did not do sowell. Unfortunately, we found that the group that most likelydid well with transplant was very small, while the group thatmost likely did poorly (worse than if not transplanted) wasquite large," Liou said.
Specifically, analyses showed that only five children had asignificant estimated benefit associated with lungtransplantation. The majority of children -- 315 to be exact --were at significant risk for harm from lung transplant, whilefor 194 children, the procedure was not clearly harmful orbeneficial.
"Our paper essentially points out how difficult it is tofind the patients most likely to die soon and thus most likelyto have a survival increase by transplantation," Liou said.
"One possible reason why there doesn't seem to be muchsurvival benefit would be that conventional therapy for cysticfibrosis has gotten so good that children with cystic fibrosisare simply no longer dying," Liou suggested. "Therefore, alifesaving operation is not only unneeded but not possible.Improvements in cystic fibrosis care have been steady andremarkably effective."
"We are not trying to eliminate the procedure or take awaythe last bit of hope for desperately ill children with CF,"Liou emphasized. "We are trying very hard to look critically atthis therapy and identify how we could make it better."
Liou also noted that the data used for the study are notsufficient to make a statement about quality of life forchildren who have a lung transplant and how it compares toquality of life without one.
"We think that some sort of study to collect the rightinformation to allow simultaneous assessment of the effects onsurvival and quality of life in the same patient is needed,"Liou said.
SOURCE: The New England Journal of Medicine, November 22,2007.