UH Physicians Publish Editorial on Evolving Cystic Fibrosis Care Model

Innovations in Pulmonology, Critical Care & Sleep Medicine | Spring 2025

Alex H. Gifford, MD, FCCP

Katherine Bruening Wajda, MD

Alex H. Gifford, MD, FCCP,and Katherine Bruening Wajda, MD, published an editorial in the Journal of Cystic Fibrosis outlining a paradigm shift in care model recommendations for cystic fibrosis (CF).

Until recently, best-practice guidelines stated that people with CF should be evaluated by their multidisciplinary care team at least quarterly. These appointments have often been lengthy due to the need for people with CF to see multiple providers on the same day. However, the development of groundbreaking CF therapies and the expansion of telemedicine during and after COVID created opportunities to alleviate some of the burdens associated with frequent visits.

History of CF Care

In 1964, visionary physicians at University Hospitals Rainbow Babies & Children’s developed a multimodal treatment regimen for infants and children with CF that included aggressively supporting nutritional status, clearing mucus from the lungs, and treating lung infections with antibiotics. Their practices set the standard for CF care for decades.

“Eminent clinicians here in Cleveland laid the groundwork for a multidisciplinary approach to CF care that offered profound clinical benefits,” says Dr. Gifford, the Connie and Jim Brown Chair in Pediatric Pulmonary Survivorship at UH Rainbow Babies and Children’s and Director of the Adult Cystic Fibrosis Program at University Hospitals Cleveland Medical Center and an associate professor at Case Western Reserve University School of Medicine. “However, the mortality rate for CF remained high because we did not have therapies that could significantly improve the natural history of the disease.”

Breakthrough Therapies

The advent of small-molecule therapies targeting the defective CF transmembrane conductance regulator (CFTR) protein has altered the disease trajectory. Highly effective CFTR modulators, including ivacaftor (IVA) and elexacaftor-tezacaftor-ivacaftor (ETI) improve CFTR function for people with CF who have responsive gene variants, a phenomenon associated with improvements in lung function, nutritional status, quality of life and survival.

“These highly effective modular therapies have been game changers in slowing overall disease progression, with increasing numbers of people who are achieving stable disease,” says Dr. Bruening Wajda, Associate Program Director for the UH Adult Cystic Fibrosis Program and an assistant clinical professor at the School of Medicine. “Patients are getting a lot of their life back and have a newfound opportunity for flexibility in their treatment schedule.”

Survey of Patients and Caregivers

When the COVID pandemic forced the CF community to pivot to remote care, the timing aligned with the recent release of ETI. Stakeholders saw the potential to rethink the necessity of quarterly in-person visits. “For many patients, we have a disease that looks very different than it did when we followed the original care model,” Dr. Gifford says.

In 2022, the Cystic Fibrosis Foundation sponsored a broadly representative nationwide survey of patients, caregivers and providers that sought input on their priorities and preferences for care delivery. Some key themes were identified:

• The survey found broad support for flexibility and shared decision-making.
• 43 percent of adults thought routine visits should occur every 4 to 6 months, versus 34 percent who believed visits should continue to occur every 3 months.
• 49 percent of caregivers of children with CF preferred routine visits every 3 months.
• 90 percent of community respondents and 91 percent of providers believed telehealth should be a standard part of routine CF care.

“We continue to see patients at least twice a year in person, and the CF Foundation has offered guidance on incorporating telehealth,” Dr. Bruening Wajda says. “Once patients have established that they have stable disease progression from multiple organ systems, it is appropriate to make the determination whether to space out visits or split them between in-person and virtual.”

Longer, Healthier Lives

Roughly two-thirds of the country’s CF population are age 18 years or older, a flip in recent decades to a higher proportion of adults. With that inspiring change comes new facets of health care needs.

“CF care centers have traditionally served as medical homes for their patients,” Dr. Gifford says. “Some of the new care model recommendations would not have been considered in the past because individuals did not live long enough to need these services. Now, I have patients contemplating the thought of retirement.”

He explains that with increasing longevity due to CFTR modulators comes the onset of health concerns that CF providers may or may not feel comfortable managing on their own. The need to manage cardiovascular disease, chronic kidney disease, cancer, osteoporosis, and sexual and reproductive health (among other age-related conditions) highlights an important role for the primary care provider in CF care.

Looking Ahead to Increased Customization and Autonomy

The CF Foundation convened working groups to develop specific types of guidance for the CF care community. This guidance embraces a holistic approach to evaluating health stability.

“The new care model enshrines the principles of prioritizing disease severity and promotes self-efficacy,” Dr. Gifford says. “Sicker patients should be seen more frequently, but we can offer more flexibility to patients who are doing well.”

For more information, contact Dr. Gifford at Alex.Gifford@UHhospitals.org or Dr. Bruening Wajda at Katherine.Wajda@UHhospitals.org.

Contributing Experts:
Alex H. Gifford, MD, FCCP
Connie and Jim Brown Chair in Pediatric Pulmonary Survivorship
University Hospitals Rainbow Babies and Children’s Hospital
Director, Adult Cystic Fibrosis Program
Cystic Fibrosis Therapeutics
Leroy W. Matthews Cystic Fibrosis Center
University Hospitals Cleveland Medical Center
Associate Professor
Case Western Reserve University School of Medicine

Katherine Bruening Wajda, MD
Associate Program Director, Adult Cystic Fibrosis Program
University Hospitals Cleveland Medical Center
Assistant Clinical Professor
Case Western Reserve University School of Medicine