This session focuses on recent discoveries relating to (1) the molecular defects of CFTR mutants; (2) the identification, development, and characterization of novel CFTR modulators; and (3) therapeutic implications.
Identify molecular defects in CFTR mutants associated with disease
Summarize strategies to identify CFTR modulators
Discuss the advantages and limitations of single and combination therapies for distinct CFTR mutants.
S. Vamsee Raju, B.Pharm., Ph.D., Post Doctoral Fellow, Gregory Fleming Cystic Fibrosis Research Center
This session will highlight the recent and important advances in understanding the mechanisms underlying altered mucus clearance in the CF airway. These talks will focus on three aspects of mucus clearance; fluid secretion, ion transport, and mucus properties and composition. Additionally, the information presented will focus on inter-relationships between these facets of the mucus clearance apparatus.
Explain how modulation of cellular ion transport alters airway surface fluid properties.
Describe how mucins and mucus are altered in CF and contribute to its pathophysiology.
Discuss how changes in airway ion transport, fluid homeostasis, and mucin biosynthesis combine to affect the clearance of mucus.
This workshop will focus on emerging research models for investigating the pathogenesis and treatment of cystic fibrosis. In addition to new animal models including the CF rat and ferret, cellular models to be presented range from cell lines expressing multiple CFTR mutations to primary culture methods expanding human airway tissue, generating organotypic structures and investigating male reproductive tract biology. Emphasis is placed on the advantages these research methods offer for investigating the multiple manifestations of CF disease.
Define new methods to expand and differentiate affected CF patient tissues in vitro for the investigation of CF pathogenesis and therapeutics.
Explain how cell lines expressing CFTR with multiple mutations can be used for the design of new correctors and potentiators of CFTR.
Discuss the contribution of CF ferret and rat models to our understanding of CF airway disease including inflammation and embryological development of the male reproductive tract
This workshop will review the latest trends in respiratory microbiology based on US CF Registry data. This session will also focus on the long-term impact of successful Pseudomonas aeruginosa eradication on health outcomes and the chronic use of inhaled liposomal amikacin in the management of chronic Pseudomonas aeruginosa infection. The results of a large survey will also be presented examining antibiotic utilization to treat Mycobacterium abscessus infection. Lastly, the usefulness of biofilm antimicrobial susceptibility testing and microbiome analysis in the management of CF pulmonary exacerbations will be presented.
Describe recent trends in sputum microbiology for individuals with CF
Summarize recent studies focused on the management of airway infections with Pseudomonas aeruginosa and Mycobacterium abscessus
Appraise the usefulness of biofilm antimicrobial susceptibility testing and culture-independent microbiome analysis in the management of CF pulmonary exacerbations
Bradley S. Quon, M.D., M.Sc, M.B.A., Clinical Research Fellow, University of British Columbia
Jane L. Burns, M.D., Professor, Pediatrics/Infectious Diseases, Univ. of Washington School of Medicine
This workshop will help you understand the importance of having a Crisis Communication Plan; as well as give you examples of what other sites have experienced and learned while creating their plan. You will come away with tools, ideas and motivation to make your own Crisis Communication Plan. If a CF patient has a life-threatening event while enrolled in a clinical trial, having a Crisis Communication Plan can prevent a catastrophe.
List the reasons why it is important to have a Crisis Communication Plan.
Locate the Crisis Management Tools on the TDN website.
Apply the Crisis management tools on the TDN website to your site.
This workshop is intended to address the management of CF patients with advanced lung disease. The session will include a discussion of CFTR modulation in patients with advanced lung disease. A broad array of topics in lung transplantation will be reviewed. Transplantation will be discussed from pediatric and adult viewpoints as well as from an international perspective.
Describe options for treating CF patients with advanced lung disease.
Describe potential risks and benefits of lung transplantation for patients with CF.
Compare international perspectives on lung transplantation for patients with CF.
Mandatory newborn screening (NBS) for CF in the US and in other countries has brought attention to new challenges and considerations for healthcare providers. Education and coordination of care with primary providers for testing is just one of many issues that have been experienced by CF care centers. This workshop will explore the way one center addressed this issue. This workshop will also look at how caregivers have responded to carrier status identification; parental understanding of NBS and their psycho-educational needs. The potential for postpartum depression following NBS will be explored and the parent of a child diagnosed by NBS will share her experience and thoughts.
Discuss ways to improve CF newborn screening processes using an entry point data system
Identify caregivers response to newborn screening results and their psychoeducational needs
Discuss the effects of postpartum depression and the impact on maternal coping of a new diagnosis of CF
Palliative care should continue across the spectrum and lifetime of a cystic fibrosis patient. However, it is often a misunderstood and underutilized service. We will have a multidisciplinary panel present and explain what palliative care is, some innovative ways and tools to apply it to clinical practice, and reasons why it is important to screen for need and appropriately intervene with palliative care early in treatment and throughout the patient's life.
Define the difference between palliative care and hospice.
Employ attendees will be able to employ palliative care concepts and involve palliative care teams within their practice.
Define why it is necessary to utilize palliative service prior to end-of-life care.
Research on the importance of nutritional status on outcomes in cystic fibrosis has established a strong association between the two. In this session, we will explore the current state of infant feeding practices. The role of pancreatic enzyme replacement therapy on nutritional status in early childhood and on health care utilization will also be explored. Monitoring of nutritional status has been centered around attaining an optimal BMI, but over-reliance on this measure may lead to missed opportunities. These topics will inform development of nutritional intervention algorithms designed to improve nutritional status.
Compare 1. Compare choice of feeding method for infants with CF and recommend strategies to support optimal growth.
Manage 2. Manage pancreatic enzyme replacement therapy dosing to maximize growth and improve overall health status.
Criticize 3. Critique the effectiveness of BMI to identify nutritionally at-risk patients, and implement a strategy to identify at-risk patients to enter into a nutritional intervention algorithm.
Elizabeth H. Yen, M.D., Assistant Clinical Professor, University of California San Francisco