Patient/provider panels will discuss the partnering process among CF patients, families, and providers for sustaining daily CF care to achieve realistic and mutually acceptable nutrition goals through collaboration and education.
Compare and contrast divergent goals from provider and patient/family viewpoints to promote optimal nutrition therapy,
Employ strategies to improve patient/family adherence to mutually agreed upon treatment plans,
Identify and utilize a variety of resources within the online CF community for positive nutrition messaging for CF patients and families.
Increasing numbers of people with CF now benefit from disease-altering small molecule therapies that target the basic defect in CFTR. Use of these therapies has been extended recently using a combination of in vitro laboratory experiments to identify responsive mutations, and clinical trials to determine effect on clinical outcomes. This plenary will summarize how recent advances may allow in the near future over 90% of individuals with CF to benefit from therapies that target the basic defect in CFTR, including even those with the rarest disease causing mutations.
Define that although CF is caused by a number of different mechanisms, mutations can be grouped by the nature of the defect and responsiveness to small molecule therapeutics.
Recall how laboratory experiments can and are being used to determine which rare disease-causing mutations are responsive to therapies.
Recall how recent advances may allow in the near future over 90% of individuals with CF to benefit from therapies that target the basic defect in CFTR.
Plenary 2 will review the progression of CF lung disease and the option of lung transplant to treat advanced disease. The presentation will highlight the challenges associated with each phase of the transplant journey, including the referral to a transplant center, waiting for a transplant, undergoing the transplant procedure, and the care requirements and life after transplant.
Discuss the progression of CF lung disease and the difficulties in predicting survival.
Apply knowledge of lung transplant outcomes and the referral process to inform the lung transplant discussion with patients and families.
Use an understanding of challenges before and after transplant to support patients and families through the process and work with transplant centers to optimize transplant outcomes.
This plenary session will focus on ways people with CF and their families make a difference by partnering with the larger community as advocates, by sharing their expertise on living life with CF with their care center teams, and by supporting with each other through virtual events and peer mentoring.
Identify ways that people with CF and their families have affected change through advocacy.
Explain how shared humanity and shared expertise can enhance care center visits.
Describe new programs to allow adults with CF to connect with each other.
Dr. Drucy Borowitz, M.D., Vice President, Community Partnerships, Cystic Fibrosis Foundation
The recently solved structures of full-length CFTR provide a template on which to evaluate electrophysiological and biochemical studies of this channel forming membrane protein. This symposium will discuss the cryo-electron microscopy structures of CFTR and their use for the interpretation of channel function studies and disease-causing mutations. Additionally, the symposium will explore the application of these structures for the development of new therapeutics for common and rare CF-causing alleles.
Examine the current structural and functional data to explore CFTR channel function.
Apply the existing structural and functional data to understand the molecular defects caused by CF-associated mutations.
Assess how structural and functional studies can be leveraged for future therapeutic development.
This session will review guidelines for CF therapy, the implementation of guidelines into clinical practice, assessment of CF lung disease, and challenges in the treatment of advanced CF lung disease.
Define the recommendations for the use of CFTR modulators in the treatment of CF.
Describe the management of patients with severe CF lung disease including the variation in inpatient treatment of CF pulmonary exacerbations and factors important in decisions to refer for lung transplant.
Define the status of multiple breath washout as a tool to guide clinical management of CF lung disease.
Cori Daines, M.D., Professor and Division Chief, University of Arizona--Banner University Med Cent
This session will focus on the utilization of CF animal models for investigation into CF disease manifestations of diabetes, airway clearance, altered intestinal stem cell proliferation and gastrointestinal cancer risk. Studies of CF ferrets, pigs, mice and ex vivo applications (cell or organoid culture) investigate the specific roles of CFTR in beta cell function, mucociliary clearance of the airways, Wnt signaling of stem cells and intestinal tumor suppression. These topics are discussed within the context of the utility and limitations of these animal models for understanding CF disease and its treatment.
Explain the utility of the CF ferret model in understanding the ways that CFTR affects the biology of pancreatic beta cells and its relevance to the major co-morbidity CF-related diabetes.
Describe the utility and limitations of the CF pig model for recapitulating CF airway disease and the outcomes that might alter current treatment of failed airway clearance.
Discuss the contributions and limitations of the CF mouse model for understanding intestinal hyperproliferation and the increased risk of gastrointestinal cancer in CF patients.
An interactive session designed to review the role of various services important in contributing to the nutritional management of individuals with cystic fibrosis and how these services are integrated to provide optimal care.
Review the role of various services in their contribution to the nutritional management of individuals with cystic fibrosis.
Describe how specific centers integrate these services to provide nutritional care.
Discuss goals for establishing future innovative ways to integrate these services.
This symposium is designed to promote discussion of airway clearance techniques for people with cystic fibrosis based on airway physiology and methods of choice. The first talk begins with an overview of lung physiology and mucociliary clearance, followed by a discussion of airway clearance techniques that do not require a device, such as percussion and vibration, active cycle of breathing techniques, autogenic drainage, and exercise. We will then discuss airway clearance techniques that require a device, such as PEP, Oscillating PEP, HFCWO/HFCC, and IPV. The final talk will discuss personalizing airway clearance techniques for people with cystic fibrosis based on their lung pathology.
Discuss lung physiology as it relates to mucociliary clearance in people with CF.
Discuss the underlying physiological mechanism of action for ACTs.
Discuss personalizing ACTs based on the person with CF's lung pathology and the ACTs mode of action.