New treatment options are expected to add decades to the lives of people with cystic fibrosis (CF), but those living with this genetic disease still must deal with burdensome issues like pain, fatigue and many other distressing symptoms.
Now, a new five-year, $3.1 million grant from the National Heart, Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH) will help Emory researchers undertake a novel study combining biological, social and clinical research methods to better understand the underlying processes of these symptoms — and identify opportunities to improve quality of life.
According to the Cystic Fibrosis Foundation, close to 40,000 adults and children in the United States live with CF, which primarily affects the lungs, digestive system and other organs. New therapies have increased median life expectancy from 30 years in 1990 to 59 years today. Dio Kavalieratos, PhD, director of research for the Emory Palliative Care Center and the study’s principal investigator, sees this change in outlook as an opportunity to rethink how clinics help people with CF live day-to-day.
“Our goal is to develop a nuanced, person-centered understanding of cystic fibrosis symptoms,” Kavalieratos says. “Looking side by side at how CF affects someone metabolically, physically, mentally, and in daily life will give us new targets to prevent and treat symptoms. To our knowledge, this is one of the first studies to integrate sophisticated metabolomic analyses with qualitative research methods — to compare what is happening at the molecular level with a person’s lived experience managing and adjusting to burdensome symptoms.”
This groundbreaking research initiative, titled “Patient-centered Understanding of Quality of Life, Symptoms and Health Equity in Cystic Fibrosis (PULSE – CF),” will be led by Kavalieratos in collaboration with partners from Emory University, Augusta University, the University of North Carolina at Chapel Hill, the Georgia Cystic Fibrosis Research and Translation Center, and patient stakeholders. The research team will triangulate three different types of data to learn more about the relationship between symptoms and quality of life. This includes:
- Reports by people with CF about the symptoms they experience regularly
- Evidence of inflammation or other biological processes in blood and breath samples
- Interviews with participants about their health care and other factors of daily life that might affect their health
The research team will look for patterns or clusters of symptoms, such as whether people with cough also tend to have fatigue and pain. They will compare characteristics of people who share common symptoms and use advanced machine learning techniques to investigate whether groups of symptoms co-occur with biological processes like inflammation. Finally, the group will interview all participants about their experience getting care for CF to identify opportunities to improve symptom management and individualize treatment for people with CF.
The study aims to enroll 140 adults living with CF over the next several years. Cade Hovater, a patient investigator who helped plan the study, says people with CF often believe they have no better option but to endure pain and other symptoms.
“Often, as people living with CF, we have been told that pain, fatigue and other quality of life issues are just part of living with cystic fibrosis. However, over the past decade we saw research into CF mental health lead to changes in how mental health needs are addressed in CF clinics. I am hopeful that PULSE-CF will provide evidence to improve care for the pain, fatigue and other symptoms that often go undertreated or untreated.”