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Thursday

This session will discuss recent developments in fluid, ion, and mucus transport, with an emphasis on the role of abnormal cells and gland function.

Learning Objectives:

  • Define the role of abnormal cell and gland secretions on mucus and fluid transport.
  • List the properties of mucin and mucus and how these may affect transport.
  • Recall the mechanisms by which mucins clear the airway, and implications of pathology.
Thursday

This workshop will initially discuss how insights into mechanisms regulating CFTR biosynthesis can help identify new targets for therapeutic intervention. A new tool for detecting plasma membrane CFTR, which could be used in high-throughput setting, will be also presented. The section will end with discussing how the newly resolved cryo-EM CFTR structures may enable structure-based drug design.

Learning Objectives:

  • Define folding and misfolding of CFTR.
  • Describe how the CFTR biosynthetic pathway can be targeted to correct several CFTR mutation classes.
  • Evaluate new approaches for assessing CFTR plasma membrane abundance and helping to design new CFTR modulators.

Note: Speaker Yunjie Huang's presentation was not recorded at his request. 

Thursday

This session will highlight recent research studying assessment and treatment of the gastrointestinal and liver manifestations of cystic fibrosis. The session will focus on: 1) assessment of common CF gastrointestinal problems, including development of innovative therapies; and 2) clinical research to better understand manifestations of and outcomes for CF-related liver disease.

Learning Objectives:

  • Recall the definition of CF-related liver disease and appraise the associations of CF-related liver disease with insulin resistance and mortality.
  • Examine methods for novel drug development and application in constipation in CF.
  • Assess esophageal impedance and gut microbiota dysbiosis in pediatric patients with CF.
Thursday

This workshop will examine the impact of mental health and treatment on lung function decline and risk of future exacerbations. In addition, the interpretations of conditional survival and reproducibility of FEVwill be described.

Learning Objectives:

  • Describe the impact of depression, mucolytics, and antibiotics on lung function decline in the US and Europe.
  • Identify a clinically meaningful change in FEVfor CF patients.
  • Review the interpretation of conditional survival and the factors that are associated with an increased risk of death before age 30.
Thursday

This session will feature presentations about recent quality improvement projects related to clinical research. Topics include improving the culture of research, patient barriers to research participation, and using TDN site metrics to set goals for improvement.

Learning Objectives:

  • Identify at least one quality improvement project that you can implement at your site over the next year.
  • Explain the importance of building a culture of research and the benefit it has for your research program and patients.
  • Describe how TDN metrics can be used as a tool to help drive quality improvement at your site.
Thursday

A leading cause of morbidity and mortality in CF patient populations is chronic respiratory infection. This workshop will present new research on pathogenesis of infections in CF, with a focus on bacterial pathogens in the CF respiratory tract. Abstracts presented will highlight bacterial adaptations to the host environment that promote enhanced virulence and interspecies interactions that influence pathogenesis in the airways.

Learning Objectives:

  • Discuss the mechanisms utilized by emerging and traditional CF pathogens to establish chronic respiratory disease.
  • Describe how adaptation to the CF airway environment leads to enhanced virulence of diverse bacterial pathogens.
  • Identify interactions occurring between bacterial species in the polymicrobial environment of the CF airways that influence pathogenesis of CF respiratory disease.
Thursday

The session will highlight strategies for assisting patients and families on a successful CF Journey. During the workshop, we will present ideas of what patients and families need throughout their lifespan to be successful across their lifespan.

Learning Objectives:

  • Identify and describe the elements of a successful CF journey across the life span.
  • Explain and summarize the importance of adherence in the CF journey.
  • Discuss how to communicate and integrate support with CF patients and families.
Thursday

In this session we will highlight new technologies and approaches that will be essential to move science ahead to a 'one time' cure for CF.

Learning Objectives:

  • Describe experimental strategies that improve and/or enhance CFTR expression.
  • Explain the advantages and disadvantages of gene altering technologies.
  • Identify the challenges that may hinder the success of genetic and/or cell-based approaches in the treatment of CF.
Thursday

In this session, recent evidence will be presented on current debatable topics in the areas of exercise, airway clearance and inhaled medications. Specifically, discussions pertaining to optimal modalities and methods for airway clearance will ensue along with the potential utility of field tests to quantify exercise capacity in the clinic. Attendees should be prepared to interact with the speakers and audience members to debate the pros and cons of the presented topics.

Learning Objectives:

  • Formulate a rationale for a sequence of respiratory treatments in obtaining desired clinical outcomes and patient adherence.
  • Discuss concerns and benefits of mobile high-frequency chest wall oscillation devices as compared to their standard counterparts.
  • Explain the potential role of exercise in facilitating airway clearance and distinguish between the information gained from formal aerobic exercise tests compared to clinical field tests.
Thursday

This workshop will highlight common transitions across the lifespan (school/educational planning, implementation of palliative care, and diagnosis of CFRD) and cross-cutting strategies that could be used in the context of disease transitions when providing care for patients and families (importance of shared decision making and the effectiveness of ACT in reducing anxiety and depressive symptoms).

Learning Objectives:

  • Identify common transitions and challenges that patients and families face across the lifespan.
  • Examine emotional responses to disease transitions.
  • Discuss interventions and strategies for supporting patients and families in navigating and managing disease transitions.

Note: Speaker Dio Kavalieratos' presentation was not recorded at her request. 

Thursday

As CF lungs at birth shows few signs of infection and inflammation, an emerging concept is that external challenges may precipitate the onset of infection and inflammation that persist into chronic lung disease. External challenges such as microaspiration of commensal upper airway bacteria or respiratory virus infections are potential external challenges that young CF lungs may not resolve as effectively as young non-CF lungs. This Symposium reviews the current knowledge of the status of the young CF lung and what the early challenges to the maintenance of airway health may be. By identifying external triggers of CF airway disease, strategies can be developed to minimize the risk of compromising the young CF.

Learning Objectives: 

  • Summarize the evolution of lung disease in the CF lung with age.
  • Appraise the changing microbiota as it evolves in the CF lung over time.
  • Evaluate the relative contribution of microaspiration versus respiratory virus infection. 

Note: Speaker Stephen Stick's presentation was not recorded at his request.

Thursday

This symposium will highlight several key aspects of cystic fibrosis related diabetes pathophysiology and management that are specific to different time periods during the life of individuals with CF. Specifically, speakers will address topics including glycemic abnormalities occurring in childhood that progress to CFRD, challenges in management of CFRD including hypoglycemia and barriers to obtaining expert medical care, and CFRD treatment regimens including novel future therapies.

Learning Objectives:

  • Discuss the early abnormalities in glycemic regulation that precede CF related diabetes.
  • Assess challenges in the management of CFRD including hypoglycemia and barriers to obtaining appropriate medical care.
  • Identify current treatment regimens for CFRD as well as novel developing therapies that may become available in the future.
Thursday

This is a new symposium at NACFC. The goal of this session is to review important publications over the last 2 years. The audience for this session is the entire cystic fibrosis team and the focus of the presentations will be on providing information in a way to help a patient that a member of the audience may see in clinic or the hospital after NACFC. The four topics will be presented by international experts in the field. Recent publications will be discussed for each topic: 1) Challenges in the diagnosis of CF infants following newborn screening; 2) Pulmonary exacerbation's and what we have learned from the STOP studies; 3) Mental health in CF and 4) an update on CFTR modulators. Attendees will be provided with a publication list for each of the topics.

Learning Objectives:

  • Summarize an approach to the challenge in cystic fibrosis infants with inconclusive diagnosis following newborn screening.
  • Describe the up to date approach to pulmonary exacerbations and for CFTR modulators.
  • Assess the current literature regarding mental heath in cystic fibrosis.
Thursday

The number of available model systems for laboratory study of CF continues to expand at a rapid pace. Current options range from genetically modified cell lines or animals to primary human cell models, each offering unique benefits. Many of these models have contributed to the development of current and emerging therapies for patients with CF. Moreover, the use of novel models at the interface of research and clinical care is expanding access to these medications. This symposium will focus on the use of CF-relevant model systems across the therapeutic pipeline from drug screening and development to personalized clinical approaches. Particular emphasis will be placed on the benefits and limitations of each discussed model, and how the choice of each model matches and empowers the study of the therapy in question.

Learning Objectives:

  • Demonstrate the wide range of complimentary CF-relevant model systems currently in use to study therapeutics.
  • Discuss the benefits and limitations of CF-relevant cell line, animal, and primary cell-based model systems relative to specific treatments or drugs.
  • Describe the utility of each unique model system at different stages of the drug development pipeline from discovery to the clinic.

Note: Speaker Christine Bear's presentation was not recorded at her request. 

Thursday

This session will review the objectives and preliminary findings of the International Working Group on Antimicrobial Resistance in CF.

Learning Objectives:

  • Define antimicrobial resistance in CF.
  • Describe the clinical relevance of antimicrobial resistance in CF.
  • Discuss the role of antimicrobial stewardship in CF.

Note: Speaker Ranjani Somayaji's presentation was not recorded at her request. 

Thursday

Overview of controversial therapies, including herbal products, nutritional supplements, salt rooms, Indrepta, vaping and other inhalants and medical/legalized marijuana.

Learning Objectives:

  • Define prevalence of CF patients' use of complimentary and alternative medicine and therapies and current practices with patients discussing their use of these products with care centers.
  • Summarize knowledge, risk perception, behaviors, education and perceived patient benefits about vaping, e-cigarettes, and other inhaled agents.
  • Review potential best practices regarding medical marijuana and transplant eligibility and palliative care uses.
Thursday

Rapidly advancing treatments of cystic fibrosis have increased the expected life span and quality of life trajectories for persons with CF. This symposium will: 1) Explore the potential for further enhancements of mental and physical health throughout the life-cycle through evidence-based recommendations for sleep, exercise, and nutrition. 2) Enable persons with CF and healthcare professionals to co-produce individual plans for healthy habits. Additionally, an adult CF patient will present his perspective on the impact that sleep, exercise, and nutrition has had on his life and well-being.

Learning Objectives:

  • Summarize current knowledge regarding the impact of sleep on health in cystic fibrosis and identify strategies for improving sleep using cognitive behavioral techniques.
  • Describe the benefits of engaging in a pulmonary rehabilitation program and utilizing individualized exercise and fitness regimens to optimize pulmonary and mental health outcomes.
  • Examine evolving nutrition research that goes beyond the FEV1/BMI association and high fat/high calorie diets. Discuss how dietary composition and personalized medical nutrition therapy can impact CF outcomes.
Thursday

This symposium will provide a current assessment of policy and economic trends that affect the cystic fibrosis coverage and access landscape. The symposium will include findings from a CF access to care study and a presentation on design and application of cost-effectiveness analyses in policy making. The session will culminate in a moderated discussion of how the CF provider community can use evidence to advocate for improved access to care and adequate, affordable coverage for patients.

Learning Objectives:

  • Summarize the current landscape of cost and coverage research and policies as they pertain to CF care.
  • Explain the role of cost effectiveness in precision medicine and its potential application in CF.
  • Interpret the findings of a national CF coverage and access study and discuss actions CFF leaders, CF clinicians, individuals with CF and families can take to prepare for pending changes in health costs, coverage, and access.

Note: Speaker Scott Grosse's presentation was not recorded at his request. 

Thursday

In this session, Dr. Lisa Saiman will address the challenges of infections in people with CF and discuss why infections remain a major focus of the CF community, even in the era of effective CFTR modulators. She will provide an overview of CF infections, describe ongoing studies to increase our understanding of CF microorganisms and their optimal treatment, and discuss current strategies to develop new anti-infective agents to treat even the most difficult infections.

Learning Objectives:

  • Identify ongoing challenges in the treatment of infections in people with CF, including the potential impact of CFTR modulators on infections.
  • Describe ongoing studies to improve our understanding of CF microorganisms including detection, diagnosis and optimal treatment.
  • Discuss strategies to develop new anti-infective therapies to treat people with CF.
Friday

This plenary will review the effect of CFTR dysfunction and CFTR restoration on both inflammation and mucociliary clearance in individuals with CF. Current and future therapeutic strategies to address inflammation and mucociliary clearance will be discussed, as well as how these strategies have potential to benefit both those patients using CFTR modulators and those not eligible for modulators.

Learning Objectives:

  • Identify current strategies to develop new therapies that treat inflammation in cystic fibrosis.
  • Identify current strategies to develop new therapies that improve mucociliary clearance in cystic fibrosis.
  • Describe the impact of CFTR modulators on inflammation and mucociliary clearance and why continued development of therapies is needed.