Studying tools to improve the quality of life for people with cystic fibrosis

Thanks to advances in treatments in recent years, people with cystic fibrosis (CF) are living longer than ever before. Along with this increasing longevity comes some ongoing challenges for patients — from adhering to their treatment plans to managing symptoms to optimize their quality of life. The Cystic Fibrosis Center, through the Division of Pulmonary Medicine at Boston Children’s Hospital, is making great strides in these areas, supporting patients with CF in innovative ways and helping to improve their health and well-being through new tools and approaches that can simplify their efforts and reduce stress.

“Boston Children’s Cystic Fibrosis Center is the largest in New England and one of the largest in the U.S.,” says Gregory Sawicki, MD, MPH, director of the center. “We are very involved in clinical research. In fact, in 2019, our clinical research team at Boston Children’s was the highest enrolling center in the country across all CF centers. We are currently participating in more than 30 clinical trials spanning early-phase treatment trials, phase 2 and 3 treatment trials, and observational and real-world studies. We also go beyond clinical trials to think more broadly about holistic care of patients,” he says.

What’s next: Improving adherence and symptom management

One of the ways Boston Children’s contributes to the well-being of patients is by serving as the sponsor and study management core of the Success with Therapies Research Consortium, which was founded in 2014 and is affiliated with the Cystic Fibrosis Foundation. Sawicki co-chairs the consortium, along with Kristin Riekert, PhD, from Johns Hopkins University.

“The mission of the consortium is to advance the clinical study of interventions to improve adherence and CF disease self-management to optimize health outcomes and quality of life,” Sawicki says.

Clinical investigations sponsored by Boston Children’s as part of the consortium’s work include: a tele-coaching pilot study that recently launched to increase accessibility to care for patients, using apps to improve adherence to care, and relying on electronic tools to improve nutrition and growth in people with CF. 

Exploring the CF consortium’s goals

The consortium studies, which closely involve CF patients and families, all share some common goals, including:

• providing validated measures of adherence and self-management specific for CF patients
• taking a patient-centered approach and empowering people with CF
• tailoring offerings to people’s unique needs and strengths
• creating initiatives that are feasible to implement
• leveraging collaborations with family, the CF care team, and the community

Boston Children’s takes a leadership role

Boston Children’s has eight staff members that contribute to these efforts, from sponsoring the work and securing funding to developing protocols and study materials, as well as facilitating the day-to-day study operations.

“We are now preparing to launch a qualitative interview study through the consortium and we will recruit participants out of Boston Children’s as the lead site,” Sawicki says.

Advice for specialists

For other physicians and specialists working with people with CF, he points out that it’s essential to partner with families to improve medication compliance and find strategies to help patients be proactive in caring for their health. “The goal for all providers should be to improve day-to-day adherence and cystic fibrosis disease self-management to optimize health outcomes and improve quality of life,” Sawicki stresses.

Learn more about the Cystic Fibrosis Center.