Improving screening and treatments for cystic fibrosis

In 2015, for the first time, adults with cystic fibrosis (CF) outnumbered children with the disease. The lifespan for those diagnosed with the inherited disorder that causes severe damage to the lungs, digestive system and other organs in the body, is also increasing.

Improvements in screening and treatments mean people with cystic fibrosis are living longer — some into their 40s and 50s or even later, said Rebekah Brown, MD, a pediatric pulmonologist at Monroe Carell Jr. Children’s Hospital at Vanderbilt and director of the Cystic Fibrosis Center at Vanderbilt. Patients are also being diagnosed earlier.

“A lot of our patients are diagnosed as newborns during the newborn blood screening (required in Tennessee since 2008) before they ever have any symptoms of CF,” said Brown, assistant professor of Pediatrics. “It allows us to intervene and be proactive to decrease the rate of progression of the disease and maybe prevent some of the complications like malnutrition which used to make children with CF very sick in their first year of life.” The screening also detects those who are carriers, but who might not develop the disease themselves.

Cystic fibrosis affects a salt channel which lines tubes in the body such as the airways, digestive tract and sweat glands. In people with the disease, a defective gene causes the secretions to become sticky and thick, plugging up tubes, ducts and passageways, especially in the lungs and pancreas.

People with CF can manage their disease by following a regular treatment routine that includes airway clearance, medication, a fitness plan and nutritional therapies. Presently, there is no cure.

About 220 CF patients are treated at Children’s Hospital, coming from Middle Tennessee, southern Kentucky, northern Alabama, Louisiana and Arkansas. Another 270 are treated at Vanderbilt University Adult Hospital, where pediatric patients transition at age 18.

The Vanderbilt CF team, accredited by the Cystic Fibrosis Foundation, takes an interdisciplinary, collective approach to the disease process. The team includes a pediatric pulmonary medical provider (physician or nurse practitioner), dietitian, social worker, respiratory therapist, pharmacist, physical therapist and Child Life specialist, who provides psychosocial support to children with CF and their families.

– by Nancy Humphrey

 

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