32nd Annual North American Cystic Fibrosis Conference

Oct 18, 2018 ‐ Oct 20, 2018



Sessions

W25--GMS: In Vivo Models of CFW25--GMS: In Vivo Models of CF

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W25--GMS: In Vivo Models of CF

Oct 20, 2018 10:30am ‐ Oct 20, 2018 11:50am

This session will discuss recent advances in the study of animal models of CF.

Learning Objectives:

  • Compose insights into the pathogenesis of cystic fibrosis by examining different animal models.
  • Discuss how animal models are used to test novel therapies for cystic fibrosis.
  • Compare cystic fibrosis disease manifestations in different animal species.

Workshop Chair(s): Workshop Speaker(s):

W26--INF/MIC: Microbial Ecology of CF Airways

Oct 20, 2018 10:30am ‐ Oct 20, 2018 11:50am

It has become increasingly clear that polymicrobial interactions, environmental conditions and evolutionary adaptations are critical to the progression of CF disease. Yet, a lack of ecological perspective has limited the translation of bench research into effective therapeutic strategies. For example, Pseudomonas does not colonize the airways alone - how do other community members and the infection environment impact Pseudomonas physiology? Recently, culture-independent approaches (genomics, transcriptomics and metabolomic) have shed light on CF infection communities, though many outstanding questions remain. In this workshop we will explore the ecology of CF airway infection communities, with the ultimate goal of devising more effective clinical therapies. Talks from scientists studying clinical samples and also recreating polymicrobial communities in the lab will educate the audience about new tools and hypotheses for relating the CF infection ecosystem to disease progression.

Learning Objectives:

  • Compare and contrast the presented approaches for studying polymicrobial communities in the CF airways that will help us identify evolutionary and ecological instabilities.
  • Identify missing datasets that are critical for managing CF as an ecosystem.
  • Exchange information about polymicrobial infection ecology amongst microbiologists, clinicians, and bioinformaticians.

Workshop Chair(s): Workshop Speaker(s):

W27--NRS-SW/PSYCH: Innovative Approaches to Building a Successful Team

Oct 20, 2018 10:30am ‐ Oct 20, 2018 11:50am

This session will focus on the integration of the different rolls of the Cystic Fibrosis care team members, and how they work with the entire team to provide high quality care to each patient.

Learning Objectives:

  • Discuss the importance of each team member's roll on the multidisciplinary team.
  • Identify characteristics of a high functioning CF team.
  • Describe strategies to integrate each individual discipline into CF clinic.

Workshop Chair(s): Workshop Speaker(s):

W28--NT: Novel & Emerging Targets for Those in Need

Oct 20, 2018 10:30am ‐ Oct 20, 2018 11:50am

During this session we will explore novel and emerging approaches to overcome the basic physiological consequences of CFTR deficiency that do not directly target mutant CFTR proteins. These approaches have the potential to reduce disease burden in people with cystic fibrosis who currently are not responsive to CFTR modulator therapies. Considered will be potential therapeutic effects of recently discovered compounds targeting the epithelial sodium channel, sodium-hydrogen exchanger 3, the intracellular scaffold protein phosphoinositide 3-kinase γ, and VPAC (vasoactive intestinal peptide and pituitary adenylate cyclase activating peptide) receptors. In addition, a new pre-mRNA editing technique to remove disease-associated mutations by exon 15 skipping will be presented.

Learning Objectives:

  • Analyze how compounds inhibiting the epithelial sodium channel and sodium-hydrogen exchanger 3 reverse the effects of CFTR deficiency on airway surface liquid hydration and mucus clearance.
  • Describe how polypeptides that either inhibit the intracellular scaffold protein phosphoinositide 3-kinase γ or have agonistic effects at VPAC (vasoactive intestinal peptide and pituitary adenylate cyclase activating peptide) receptors increase channel function of F508del CFTR and reduce cystic fibrosis disease manifestations in mouse models.
  • Discuss a novel pre-mRNA editing technique to induce exon 15 skipping to remove an array of deleterious CFTR mutations in order to restore CFTR channel function.

Note: Speakers Patrick Moore, Alessandra Murabito, Valerie Chappe's presentations were not recorded at their request.

Workshop Chair(s): Workshop Speaker(s):

W29--PT&RT-CLIN: The Role of the PT/RT for Patients Who Are Pre & Post Lung Transplant

Oct 20, 2018 10:30am ‐ Oct 20, 2018 11:50am

This session will look at current and evolving practices in the pre and post lung transplant patient with cystic fibrosis with an emphasis on physical therapy/pulmonary rehab and airway clearance. Exercise and ACTs have become effective treatment options for patients on the waiting list for lung transplantation, and can improve quality of life. Exercise is also necessary to ensure patients are strong enough to recover from surgery. Improving education on exercise and airway clearance is essential to improve the quality of life in these patients.

Learning Objectives:

  • Define the importance of exercise and airway clearance techniques in patients with cystic fibrosis pre-lung transplant and post lung transplant wanting to improve their quality of life and prior level of function.
  • Describe challenges before and after lung transplant to support patients with cystic fibrosis that are utilizing physical therapy/pulmonary rehab.
  • Discuss the impact that physical therapy/pulmonary rehab and airway clearance can have on recovery of patients with cystic fibrosis who have had a lung transplant.

Workshop Chair(s):
  • Matthew Bowman, D.P.T., P.T., C.C.S., Physical Therapist
  • Matthew Dartt, B.S., R.T., Assistant Director of Respiratory Therapy, UCLA Medical Center, Santa Monica
Workshop Speaker(s):

W30--SW/PSYCH-NTR-NRS: Feeding & Eating Disorders

Oct 20, 2018 10:30am ‐ Oct 20, 2018 11:50am

One of the most concerning symptoms of cystic fibrosis for children are failure to thrive and malnutrition. Patients with cystic fibrosis must follow a strict diet that is implemented by their parents or self-implemented. The nutritional focus for survival establishes child feeding as a primary treatment goal for parents, and children with CF require 20 to 50% more calories per day and a higher fat diet than a healthy child. Treatment regimens that specify a therapeutic diet may place the child at increased risk for feeding problems or disordered eating behaviors. This session will focus on recent research on feeding and disordered eating in children and adults.

Learning Objectives:

  • Describe the unique risk factors for feeding problems in patients with cystic fibrosis.
  • Determine the impact of behavioral-nutrition intervention on families of children with cystic fibrosis.
  • Describe the relation between cystic fibrosis and the risk for negative body image.

Workshop Chair(s): Workshop Speaker(s):

W31--U&C-CLIN: Hot Topics in Health Policy

Oct 20, 2018 10:30am ‐ Oct 20, 2018 11:50am

The content of this workshop session with be based on abstracts submitted by researchers focusing on cost of care, access and insurance issues, and utilization of healthcare resources for CF patients in both the U.S. and abroad.

Learning Objectives:

  • Describe trends in cost of CF Care.
  • Identify patterns of health care utilization for CF patients with government-funded insurance (i.e. Medicaid, Medicare, Universal).
  • Discuss access to care challenges for CF patients.

Note: Speaker Kate Skolnik's presentation was not recorded at her request.

Workshop Chair(s): Workshop Speaker(s):
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