Lung transplants for cystic fibrosis may not be such a good option

Researchers in the United States say lung transplants do little to improve the outcomes for children with severe cystic fibrosis (CF) and often increases their risk of dying.

It seems despite costly and complex surgery only 5 of the 514 American children, age 18 and younger, on the waiting list for a transplant from 1992 through 2002 lived any longer as a result of the operation.

Of the 248 children who actually received new lungs in the 11-year period, only one patient showed a clear benefit from transplantation, while 162 (almost two-thirds), were at a higher risk of dying after the procedure.

The researchers were unable to determine the harm or benefit of transplantation in the remaining 85 children who received new lungs.

The revelation will be a blow to the many young sufferers of the disease and their families as lung transplantation is an accepted treatment for severe cases of the fatal genetic disease.

Dr. Theodore Liou and his team at the University of Utah in Salt Lake City say they were shocked at the finding.

They embarked on the study believing there would be a number of patients who would benefit along with some who would not, but it did not balance out.

Cystic fibrosis affects around 70,000 people worldwide; it is a hereditary disease that attacks multiple systems in the body, most dramatically affecting the lungs and gastrointestinal tract.

The disease creates an abnormally thick mucus that clogs the lungs and digestive system.

Relatively few studies have examined the effectiveness of lung transplants for helping treat the incurable disease but this study is the most extensive study of the issue to date.

Although the Cystic Fibrosis Foundation says that 50 percent of recipients are alive five years after surgery, complications from transplants are responsible for 12 percent of all deaths among people with CF.

There have also been suspicions that the operation may not be best for sufferers under 18 as the five-year survival rate of 33 percent for children is less than for adults or people who have received a transplant for another reason.

Dr. Liou says the implication is that most children with CF are going to be harmed by lung transplantation and there should not be the expectation that lung transplantation will prolong their lives.

Dr. Liou says the study emphasises the need for CF patients to follow a consistent regimen of conventional therapy and medical care to maximize their survival.

Although treatments can slow the disease, there is no cure as yet and life life expectancy is at present around 25 years, although advances in therapy now allow some CF patients to live into their 40s, 50s, and beyond.

Although it is unclear why, the risk of death after lung transplantation increases steadily as children with CF get older the researchers say it is possibly because their immune systems have been compromised by infections.

According to Liou, diabetes develops in most patients who receive new lungs and the physical toll the disease takes may account for a large proportion of the harm to patients after transplantation.

Children who already have diabetes when they’re transplanted have, says Liou in effect, already paid the physical costs of the disease before receiving new lungs and bear that added burden on their chances of survival.

Dr. Liou says although lung transplants don’t help most children with CF, it does not mean transplantation cannot benefit some children, but it is critical to identify the children who would benefit from transplants.

Currently, transplant candidates are selected because of low lung function, worsening symptoms of the disease, and other factors than may indicate a poor prognosis;the researchers say those criteria are not working.

The researchers say they were unable to answer the effect of transplantation on quality of life, and say more research needs to be done in that area.

The research is published in the New England Journal of Medicine.