Patients at Arkansas Children’s Cystic Fibrosis Center benefit daily from the team’s involvement in leading-edge research in medications for CF.
“We recently completed participation in a study looking at alleviating the burden of care of CF patients receiving highly efficient modulators by discontinuing use of inhaled hypertonic saline and dornase alfa,” said Ariel Berlinski, M.D., pediatric pulmonologist, medical director of the department of Respiratory Care Services and director of the Cystic Fibrosis Center at Arkansas Children’s Hospital and professor of Pediatrics in the Division of Pulmonology at the University of Arkansas for Medical Sciences (UAMS). “The approval of highly efficient modulators has transformed the lives of patients with CF.”
Approximately 90% of patients seen in the Arkansas Children's Cystic Fibrosis Center have been offered highly efficient modulators. The team is also involved in conversations regarding new standards of care in this changing landscape of CF treatment. We are committed to collaborate with the CF community to reach the goal of finding a cure.
Additionally, the team has a unique interest in aerosol delivery and respiratory care and is actively studying the effects of real-life drug delivery practices through simulations in the Arkansas Children’s Cystic Fibrosis Center’s Aerosol Research Laboratory.
The process of medical innovation may on occasion be required to respond to changes in the larger cultural and technological environment. For example, the need for expanded virtual care options has presented unprecedented challenges for children with CF, among them the need to monitor treatment efficacy or communicate clearly using a new technology. The Arkansas Children's team has approached these challenges as an opportunity to improve care.
"We historically have a very large respiratory care department, with a strong emphasis on providing respiratory care support to outpatient clinics and the hospital," Dr. Berlinski said. "To extend the support we offer our patients, we developed a method that allows a patient located elsewhere to be coached by an experienced respiratory therapist while performing office-based spirometry. We also developed an internal process for the use of pediatric home spirometry, and we continue to evaluate its place in patient care."
As a pediatrics site for the Cystic Fibrosis Therapeutics Development Network, a national network dedicated to CF research, the center is committed to continued innovation.
Current and upcoming projects include:
Dr. Berlinski notes that much of the CF research underway at Arkansas Children’s focuses on real-world validation of therapies and practices that have been shown effective in strictly controlled laboratory settings but may not work as well — or simply may work differently — in the context of patients’ lives.
"The human factor is crucial in the delivery of medications," he said. "I'm interested in understanding the effects of using various therapies when patients don't always follow recommended use patterns."
For example, patients may choose to deploy two devices in tandem, affecting the portion of lung where an aerosol is deposited. Or patients may not shake an inhaler or may delay between shaking the inhaler and actuating it. All these human factors impact the actual effect of a treatment on a patient.
Arkansas Children’s high standards of comprehensive multidisciplinary care, leveraged by skilled, appropriate application, can be seen in the Cystic Fibrosis Foundation Patient Registry Annual Data Report from 2021, the most recent year available.
During that timeframe:
In the same data report, the forced expiratory volume in the first second (FEV1) for 13 to 17 year old Arkansas Children’s patients was 103.3%, while the median for pediatric programs was 97.5%.
Dr. Berlinski and others on the Arkansas Children's Cystic Fibrosis Center team never lose sight of which outcomes are most meaningful to patients. This focus is especially key because managing CF can be a burdensome process for patients and families. Patients with CF only take about half their prescribed medications, the Cystic Fibrosis Foundation estimates. Research based on structured interviews has found that care plans may be more successful when those plans have been developed as part of a dialogue between patients and trusted providers.
"We aim to incorporate families into all our working groups," Dr. Berlinski said. "Their input is crucial. Treatment plans that we as clinicians perceive as the right course of action may not be viewed the same way by those to whom we are delivering care. Our patients and families let us know about concerns they may have, which has helped us establish priorities. That is very valuable."
At Arkansas Children’s, patients can transition seamlessly to the adult care program at UAMS and benefit from close communication between the two programs.
"Personally, I think we've been successful in treating patients with CF when they are able to achieve their goals," Dr. Berlinski said. "Transitioning pediatric patients to adult care is a significant milestone for patients and families and for the CF team as well."