By Megan Rauscher
NEW YORK (Reuters Health) - Lung transplantation in children with cystic fibrosis (CF) is not likely to prolong life and may do more harm than good, according to a look back at essentially the entire U.S. pediatric experience with lung transplantation for cystic fibrosis for the period 1992 through 2002.
According to a report in November 22 issue of The New England Journal of Medicine, a total of 248 of the 514 children with cystic fibrosis who were on the waiting list during the 10-year-period underwent lung transplantation.
"Children undergoing lung transplant did much worse than expected," Dr. Theodore G. Liou of the University of Utah, Salt Lake City, told Reuters Health.
"We fully expected, at the start of the study, that we would find a group that did well and others that did not do so well. Unfortunately, we found that the group that most likely did well with transplant was very small, while the group that most likely did poorly (worse than if not transplanted) was quite large," Liou said.
Specifically, analyses showed that only five children had a significant estimated benefit associated with lung transplantation. The majority of children -- 315 to be exact -- were at significant risk for harm from lung transplant, while for 194 children, the procedure was not clearly harmful or beneficial.
"Our paper essentially points out how difficult it is to find the patients most likely to die soon and thus most likely to have a survival increase by transplantation," Liou said.
"One possible reason why there doesn't seem to be much survival benefit would be that conventional therapy for cystic fibrosis has gotten so good that children with cystic fibrosis are simply no longer dying," Liou suggested. "Therefore, a lifesaving operation is not only unneeded but not possible. Improvements in cystic fibrosis care have been steady and remarkably effective."
"We are not trying to eliminate the procedure or take away the last bit of hope for desperately ill children with CF," Liou emphasized. "We are trying very hard to look critically at this therapy and identify how we could make it better."
Liou also noted that the data used for the study are not sufficient to make a statement about quality of life for children who have a lung transplant and how it compares to quality of life without one.
"We think that some sort of study to collect the right information to allow simultaneous assessment of the effects on survival and quality of life in the same patient is needed," Liou said.
SOURCE: The New England Journal of Medicine, November 22, 2007.