detection of lung disease in cystic fibrosis (CF), combined with aggressive
treatment in infants, may be the key to controlling the progression of the
disease, according to a recent study. New research shows that contrary to
previous scientific opinion, progressive lung damage in CF patients can begin as
early as infancy even though lung function shortly after diagnosis is
“We might be able to stop some of the lung
function reduction we’re identifying in the first months of life,” said Dr.
Sarath Ranganathan, Ph.D., consultant respiratory physician at the Royal
Children’s Hospital in Melbourne, and lead author of the paper, which appeared
in the second issue for December of the American Thoracic Society’s American
Journal of Respiratory and Critical Care Medicine.
know what’s going to work, but we have to target those patients in the first six
months of life if we’re going to be effective,” added Dr. Ranganathan.
Australian study, 68 infants with CF were compared to 49 infants without the
disease. The children were between six weeks and 30 months of age. Forced
expiratory volume (FEV) measurements were obtained for the children at baseline,
and 16 of the children with CF were measured again one year later. FEV was
equivalent for all children both with and without CF at baseline. But by six
months of age, the mean FEV score was significantly lower in infants with CF compared
to controls—and the deficit increased with each month of age. “This finding indicates that lung function declines
sooner than previously thought,” said Dr.
found that diminished lung function occurred even in the absence of clinical
symptoms and irrespective of CF genotype. But, notably, infants measured soon
after birth and within the first six months of life had normal lung function.
Newborn screening for CF has been in existence in Australia for nearly 30 years, but was introduced
only recently in the United
Kingdom and the United States. This finding validates the early-screening approach
taken in Australia and strongly suggests that
other nations should adopt similar approaches.
percent of those with CF can’t absorb the fat in their diet, and people with
this condition who are better nourished live longer,” said Dr. Ranganathan.
“[Those patients] should take manufactured pancreatic enzymes with every meal
that contains fat, so they can have a good high-fat diet with lots of calories,”
he added. “Although the patients in our study were well-nourished, they still
had diminished lung function. However, we can’t be certain whether or not fat
malabsorption that occurs prior to starting enzymes at diagnosis contributes to
diminished lung function later on in early childhood.”
While the FEV
tests were conducted according to standardized guidelines recently developed by
the American Thoracic Society and the European Respiratory Society, if better
tests for lung function in newborns could be developed, it might be possible to
determine that lung function is not in fact normal during the first six months,
Dr. Ranganathan said. “Our paper really indicated that lung function is
diminished after the first six months, but appears to be normal in the first
six, which is good news and bad news,” he said. “It’s bad news because even with the best current
treatment we don’t seem able to prevent diminished lung function occurring in
later infancy. The good news is that if we can diagnose infants with this
condition early by newborn screening, we have shown for the first time that
there appears to be a window of opportunity in the first six months of life to
intervene to prevent diminished lung function.” The findings may inform
future research—and, it is hoped, clinical practices.
a result of this research, Dr. Ranganathan and colleagues have developed a
research team called The Australian Respiratory Early Surveillance Team for
Cystic Fibrosis (AREST-CF).
looking into the possibility of an intervention trial using macrolides as these
drugs have proved useful in older children and adults. Anticipating that we
would conduct the trial in one or two years’ time, we think we need to recruit
close to 80 patients in each arm of the trial,” said Dr. Ranganathan, who is
chair of the Melbourne wing. “Our main aim would be to
optimize what an appropriate study design for an intervention trial should be
so that we can move as quickly as possible from
research to treatment. We’d like to be very aggressive about treatments
from the very day an infant is diagnosed,” he added.
article, view: http://www.thoracic.org/sections/publications/press-releases/resources/ranganathan-cf-art.pdf
Source : News release from American Thoracic Society on December 5, 2008.