Five things to know about advancements in cystic fibrosis care

Early detection of cystic fibrosis allows children to start receiving treatment for the genetic disorder long before symptoms typically appear. That early intervention – along with new and more advanced treatments – is giving more people with cystic fibrosis longer, higher quality lives.

“I tell parents that cystic fibrosis will become just part of their life,” said Samuel Goldfarb, MD, co-director of the Minnesota Cystic Fibrosis Center at M Health Fairview and a professor of Pediatric Pulmonary and Sleep Medicine at the University of Minnesota Medical School. “Your child should grow up and do all the normal things, including playing sports and going to college if that’s what they want to do. But it is a chronic disease. We have great treatments for it, but we don’t have the cure for it yet and we’re continuing to work on that.”

Goldfarb shares five things to know about cystic fibrosis (CF), including treatment innovations and why he’s optimistic about the future of cystic fibrosis care.

1. Early intervention can lead to better outcomes.

Cystic fibrosis is an inherited condition that affects the lungs and gastrointestinal systems. It occurs in people who get defective copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene from both parents. People with just one defective gene don’t have cystic fibrosis but could pass it on to their children.

Babies are tested for cystic fibrosis with a heel prick blood test soon after birth. If the test detects a high level of a protein called IRT, the child will have genetic testing to identify gene mutations. They will be referred to a cystic fibrosis center for confirmation of the diagnosis which can include a sweat test. This screening process with rapid diagnosis is the best way to diagnose cystic fibrosis and get the child started on therapies. Newborns who start receiving treatment early tend to have better growth, healthier lungs, and longer lives.

Cystic fibrosis can cause mucus around the lungs, pancreas, and other organs to become thick and sticky. A regular part of cystic fibrosis care is getting the airways cleared to help with breathing and preventing lung infections. People with cystic fibrosis are also monitored for signs of malnutrition, poor growth, diabetes, sinus disease, and gastrointestinal complications.

2.  People with cystic fibrosis are living longer, healthier lives.

Thanks to early detection, ongoing management and newer therapies, care for people with cystic fibrosis has changed dramatically. In recent years, the number of hospitalizations for children with cystic fibrosis has gone down considerably. At the same time, life expectancy continues to rise.

“When I started, life expectancy was 30,” Goldfarb said. “Now it’s in the 50s or even longer depending on the year the person was born.”

Care advancements have even changed the way physicians provide care. Cystic fibrosis doctors used to serve as the patients’ primary care provider because the condition played such a major role in their health. Now some young people with cystic fibrosis have very few symptoms, so their condition is taking a backseat. They can see general pediatricians for routine care if that’s easier or closer to home.

3.  Research continues to bring new treatment options.

Doctors no longer treat only a person’s symptoms. They can also target the cause of the condition with therapies that correct the defective gene. And therapies are only getting better.

The medicines that a child with cystic fibrosis takes today might be completely different in 10 years because ongoing research is bringing better treatments, Goldfarb said. Currently, the University of Minnesota research is contributing to research on:

4. M Health Fairview has a long tradition of supporting people with cystic fibrosis.