Transition is a broad and complex topic within CF, including transitions between settings, to new care centers, and to new disease states. This session will focus on various transitions across the CF trajectory, encouraging the audience to consider the impact of transitions on the patient, family, and care team. The presenters will share unique examples of CF transition programs, discussing implementation and encouraging the audience to consider applicability to their own care centers.
Describe different types of transitions for individuals with CF and their families across the lifespan.
Discuss the psychosocial impact of various transitions on the patient, family, and care.
Evaluate strategies to create or improve transition procedures at individual care centers.
Kimberly Canter, Ph.D., Psychologist, Nemours/Alfred I. duPont Hospital for Children
This session will look at current and evolving treatment practices within CF care around the globe with an emphasis on nebulizer therapy and airway clearance. The session presents the opportunity to explore varying practices, the opinions of people with CF and current international research in order to support and/or challenge current practice around nebulizer therapy and airway clearance. Improving knowledge of the theory and practicalities of treatment, of the links between exercise and airway clearance and of understanding and managing the challenges that these burdensome treatments present is essential to improving clinical outcomes and the quality of life of people with CF.
Describe the physiological properties of ACTs and debate how exercise influences airway clearance.
Discuss clinical reasoning around the practicalities of airway clearance techniques and nebulised therapies, for example choice of technique/medication and the order of administration.
Identify the challenges of sustaining adherence to long term therapy and gain insights into the opinions of people with CF, innovative research and practices in supporting adherence.
Matthew Dartt, B.S., R.T., Assistant Director of Respiratory Therapy, UCLA Medical Center, Santa Monica
Tracey Daniels, M.Sc., B.Sc., Advanced Clinical Specialist physiotherapist, York Teaching Hospital NHS Foundation Trust
As our knowledge for CFTR structure has improved, searches for mutation-specific as well as mutation-independent tactics have begun to emerge and open the path to revolutionizing our approach in treating CF patients. The presenters will share preclinical data for innovative therapeutic strategies, and describe new biomarkers useful in clinical trials, focusing on the infant cohort.
Discuss the potential development and therapeutic applications of 2 mutation independent strategies aiming to repair the CFTR gene (gene therapy) or the airway tissue (stem cell therapy).
Discuss the rationale for triple combination therapeutic regimens for F508Del-CFTR.
Assess knowledge into novel biomarkers for clinical trials in cystic fibrosis.
The interplay between a CF patient's microbial communities and their health is complex. The first talk in this session will illustrate how the gut microbiome influences the rate of clinical exacerbations through changes in immune signaling. The next talk will describe how Pseudomonas aeruginosa undergoes adaptive evolution in the sinuses of CF patients, potentially seeding the lungs with pathoadapted strains. The last three talks will explore the microbial community structure and function in the CF lung -- first by coupling genomics and metabolomics to identify biomarkers of disease progression, then by exploring how targeting a specific metabolism of P. aeruginosa, nitrate respiration, can kill antibiotic-tolerant bacteria, and finally by demonstrating how the commonly used antibiotic tobramycin primarily targets non-dominant taxa in the CF lung.
Demonstrate that the activity of bacterial communities at multiple sites in the human body affect the health of CF patients.
Illustrate the utility of understanding the metabolic state of CF pathogens in order to identify biomarkers of disease progression and to develop novel treatments.
Assess the ecological impact of treatment with tobramycin.
This workshop highlights advancements in new and emerging technologies progressing towards a cure. Discussions will include development of tools/strategies focused on rare and nonsense CFTR variants with translatable relevance to all CFTR mutations.
Summarize molecular defects attributable to CFTR premature truncation codons, including the role of nonsense mediated decay and its potential as a therapeutic target.
Compare approaches for CFTR gene repair or replacement in primary airway epithelia.
Evaluate state-of-the-art directed differentiation of airway progenitor cells from pluripotent source material.
Note: Speaker Youngjin Kim's presentation was not recorded at his request.
This session will be a combination of both clinical and basic science research that focuses on identifying either new treatments or therapeutic targets in the management of GI and hepatic manifestations of cystic fibrosis.
Identify a potential new treatment target in CF-associated constipation.
Discuss the use of organoids and '-omics' to better understand the impact of CFTR dysfunction on the intestinal environment.
Review the utility of imaging modalities to access GI and Hepatic disease in patients with CF.
Note: Speakers Christabella Ng and Michael Coffey's presentations were not recorded at their request.
This session will focus on various CF pharmacotherapy issues facing practitioners today. The topics that will discussed are the standardization and improvement in the rate of Pseudomonas aeruginosa eradication, collaborative efforts of an integrated healthcare system CF team on medication delivery, analysis of tobramycin serum concentration monitoring strategies (central vs peripheral line collections), the impact of the addition of a dedicated-CF pharmacy technician to an ambulatory CF clinic team, and the evaluation of the impact of pharmacist-driven interventions on medication adherence and outcomes on CFTR modulator therapy.
Describe the process involved to improve the rate of eradication of Pseudomonas aeruginosa.
Explain the impact of dedicated ambulatory CF pharmacy technicians and pharmacists on CF medication delivery, adherence, etc.
Discuss the difference in tobramycin serum concentration monitoring strategies between peripheral vein and central-line collections.
To inform the development of innovative new therapies for CF lung disease, this workshop has three objectives. First, to assess the expression and function of CFTR in different populations of airway epithelial cells, including pulmonary ionocytes, rare cells that highly express the Cftr gene. Second, to appraise the role of CFTR-independent pathways for anion transport in mucociliary clearance, including SLC26A9. Third to discuss the organisation of mucus in bundles in normal airways and sheets in CF airways.
Evaluate the expression and function of CFTR in different types of airway epithelial cells.
Assess CFTR-independent mechanisms of mucociliary clearance.
Review the organisation of mucus in normal and cystic fibrosis airways.
Note: Speaker Pamela Millar-Büchner's presentation was not recorded at her request.