Study Quantifies Dramatic Improvements in Lung Function Through More Aggressive Treatment of Cystic Fibrosis

Researchers at Cincinnati Children’s Say Study Reaffirms Therapeutic Approach Used by Increasing Number of Treatment Centers

Wednesday, September 17, 2008

Earlier treatment with mucous-thinning medication, enhanced nutrition and aggressive infection control were associated with young children battling cystic fibrosis having on average normal lung function with no decline, according to a study by researchers at Cincinnati Children’s Hospital Medical Center posted online in the Journal of Pediatrics. 

Focusing on children ages 6 to 12 treated at the Cincinnati Children’s Cystic Fibrosis Center the researchers said their data suggest increasingly aggressive treatment regimens – used by a growing number of centers across the country – enhance outcomes for children with CF. The study also supports information coming from other medical centers and the Cystic Fibrosis Foundation on the benefits of improved nutrition and treatment with dornase alfa, an inhalant used to reduce the thickness of mucous that causes chronic lung infection and damage in children with CF, said Gary McPhail, MD, a physician and researcher in the division of Pulmonary Medicine at Cincinnati Children’s.

"We believe this to be the first peer-reviewed data that shows essentially zero decline in lung function in kids with CF between ages 6 to 12, and that there may be other treatment centers seeing similar results,” said Dr. McPhail, lead author of the study and assistant professor of Pediatrics at the University of Cincinnati College of Medicine. “Our study underscores the importance of being very aggressive with treatment at an early age to have a significant benefit for children, which is an approach advocated by the CF Foundation."

The researchers studied the health outcomes of two different groups of children with CF – those born between 1985-1992 and 1993-2000. The children born later received more aggressive treatment at an earlier age because of new therapies and research indicating the approach would be more effective. This included an enhanced level of nutritional intervention, infection control, more frequent doctor visits and the start of treatment with dornase alfa.

The researchers noted that children treated at Cincinnati Children’s for cystic fibrosis are frequently prescribed dornase alfa by age 6, even if there is no evidence of lung function decline. The medication has been used to treat CF since the early 1990s.

Average lung function values at age 6 for children born 1993-2000 were 12 percent higher than children born 1985-1992 (as determined through measurement of Forced Expiratory Volume at one second). Between the ages of 6 and 12, average lung function values for children born 1993-2000 were 16 percent higher than the older group, with the children born 1993-2000 showing essentially no decline in lung function, Dr. McPhail said.

Also dramatically improved among the group born 1993-2000 was overall nutritional status as determined by body mass index, or BMI. While children in the study born 1985-1992 had BMI at ages 3, 6 and 12 that fell below the average values for their age (under the 50^th percentile), BMI for children born 1993-2000 fell above average, or over the 50^th percentile. Children in the group of patients born later also had fewer chronic P aeruginosa infections, with 37.7 percent showing signs of infection compared to 65.2 percent in group born 1985-1992.
The researchers based their study on data from the medical records of 144 children treated for CF at Cincinnati Children’s (74 born 1985-1992 and 70 born 1993-2000).

Dr. McPhail said advancements in CF care in recent decades are because of ongoing research and new treatments advocated by the CF Foundation and more than 100 accredited CF centers across the country.”

“During the 1940s, when CF was first recognized as a genetic disease, average survival was to about 1 year of age, and by the 1960s it increased to about 12 years of age,” Dr. McPhail explained. “People with CF now have a median predicted survival into their upper 30s.”
CF causes children to produce thick mucous in their lungs that leads to chronic infection, damage and lung failure. The mucous forms because the liquid normally produced by the body to lubricate lung tissues does not contain sufficient water.

Other researchers contributing to the study include James D. Acton, MD, Matthew C. Fenchel, MS, Raouf S. Amin, MD, and Michael Seid, PhD, all of Cincinnati Children’s. Funding support for the research came from the Division of Pulmonary Medicine at Cincinnati Children's.

Cincinnati Children's is one of America’s top three children’s hospitals for general pediatrics and is highly ranked for its expertise in digestive diseases, respiratory diseases, cancer, neonatal care, heart care and neurosurgery, according to the annual ranking of best children's hospitals by U.S. News & World Report. One of the three largest children’s hospitals in the U.S., Cincinnati Children’s is affiliated with the University of Cincinnati College of Medicine and is one of the top two recipients of pediatric research grants from the National Institutes of Health. For its achievements in transforming healthcare, Cincinnati Children's is one of six U.S. hospitals since 2002 to be awarded the American Hospital Association-McKesson Quest for Quality Prize ® for leadership and innovation in quality, safety and commitment to patient care. The hospital is a national and international referral center for complex cases, so that children with the most difficult-to-treat diseases and conditions receive the most advanced care leading to better outcomes.