31st Annual North American Cystic Fibrosis Conference

Nov 2, 2017 ‐ Nov 4, 2017


The North American Cystic Fibrosis Conference (NACFC) provides a collaborative and educational forum for CF professionals to help advance CF research and care. This annual meeting brings together scientists, clinicians, and caregivers from around the world to discuss and share ideas on the latest advances in CF research, care, and drug development and to exchange ideas about ways to improve the health and quality of life for people with CF.


Sessions

W18: NT: Innovative Approaches to CF Therapy

Nov 3, 2017 2:00pm ‐ Nov 3, 2017 3:35pm

Identification: W18

During this workshop, novel approaches to treating CF lung disease will be presented and discussed. This session will also focus on outcome measures that reflect response to therapy in CF.

  1. Identify novel mechanisms of action in which CFTR can be activated.
  2. Define the role of other transport processes in clearing the CF airway surface liquid.
  3. Identify novel assays and biomarkers to measure improvement in CF lung disease and to assess long-term effects of therapeutic agents.

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

W19: PT&RT: Emerging Clinical Tools in PT/RT: What Are We Measuring?

Nov 3, 2017 2:00pm ‐ Nov 3, 2017 3:20pm

Identification: W19

This session will describe emerging clinical tools and outcome measures that may help to guide clinical decision-making and improve quality of care. Additionally, we will review the benefits of an established clinical tool (CPET) and discuss application in cystic fibrosis care.

Learning Objectives:

  1. Describe methods of measuring multi-dimensional wellness and adherence to therapy.
  2. Prepare an overview of how to collect reliable measures of the lung clearance index (LCI) and describe potential clinical applications of the LCI in young children with cystic fibrosis.
  3. Explain how a formal cardiopulmonary exercise test can provide outcomes to guide clinical decision making.

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

W20: SW/PSYCH: Mental Health Outcomes & Research

Nov 3, 2017 2:00pm ‐ Nov 3, 2017 3:20pm

Identification: W20

This workshop focuses on new research findings related to the CFF Mental Health Initiative. Presentations include prevalence data on depression and anxiety at a pediatric hospital in Italy, longitudinal results at the Buffalo CF Center on mental health outcomes, a model implementation program in a large adult Center, the effects of telehealth interventions (including Acceptance and Commitment Therapy) on mental health outcomes, and results of an internet survey of 84 CF Centers funded with a Mental Health Coordinator.

Learning Objectives:

  1. Summarize data on implementation of the mental health guidelines.
  2. Summarize about the outcomes of interventions to address depression and anxiety.
  3. Summarize about the impact of the 1st year of dissemination at 84 CF Centers. .

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

W21: APP&D: CF Inflammation: Mechanisms, Treatment & Prevention

Nov 4, 2017 10:30am ‐ Nov 4, 2017 11:50am

Identification: W21

This session explores how maladaptive responses of inflammatory cells and mediators may contribute to CF airway disease, and how new interventions targeting these responses could be therapeutic for people with CF.

Learning Objectives:

  1. Describe how pharmacological correction of CFTR modulates inflammatory signaling; discuss the role of IL-17 T cells in P. aeruginosa infection.
  2. Explain the impact of the CF airway milieu on transcriptional regulation of PMNs; learn the interplay between pannexin 1 and inflammatory responses of macrophages.
  3. Discuss metabolic differences between macrophages derived from different regions of the lung.

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

W22: CFTR: CFTR Theratyping: Insights from Matching Modulators & Mutations

Nov 4, 2017 10:30am ‐ Nov 4, 2017 11:50am

Identification: W22

There are nearly 2,000 mutations in the CFTR gene, and only a small fraction of these have been tested for responses to approved drugs. CFTR modulators are small-molecule compounds that act upon CFTR by correcting a folding defect (VX-809) or by potentiating a gating defect (VX-770). Patients with gating mutations, like G551D, display very favorable responses to the drug, ivacaftor (VX-770). The far more common F508del mutation results in a protein that has a folding defect. 50% of CF patients have 2 copies of F508del and display modest responses to lumacaftor/ivacaftor (VX-809/VX-770). It is unknown if patients with rare mutations will respond to these CFTR modulators. In this workshop, we will discuss theratyping, which involves grouping patients who are likely to respond favorably to the same CFTR-modulating therapies (VX-809, VX-770, and novel compounds as they become available) based on their CFTR mutations. We will also discuss screening tools that may assist with theratyping.

Learning Objectives:

  1. Describe biomarkers and screening tools to identify compounds with improved potency and efficacy for F508del CFTR.
  2. Explain how molecular and functional phenotypes of rare CFTR mutations will assist with identifying targets for drug therapies.
  3. Discuss pre-clinical evidence for novel compounds that increase protein expression and function of mutant CFTR.

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

W23: CLIN: EpidemiologyW23: CLIN: Epidemiology

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W23: CLIN: Epidemiology

Nov 4, 2017 10:30am ‐ Nov 4, 2017 11:50am

Identification: W23

Workshop highlighting important epidemiologic research into pulmonary exacerbations in CF, and identifying important predictors of survival among patients with CF.

Learning Objectives:

  1. Describe implications of viral infections for CF patients hospitalized with pulmonary exacerbation.
  2. Demonstrate understanding of treatment approaches to pulmonary exacerbations in CF.
  3. Summarize predictors of survival among patients with CF, including the effects of pulmonary exacerbations.

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

W24: CLIN: Gastroenterology, Hepatology & Nutrition

Nov 4, 2017 10:30am ‐ Nov 4, 2017 11:50am

Identification: W24

From guts to glory, this session covers the breath of GI and Nutrition problems faced by individuals with Cystic Fibrosis; evaluating new innovations including Relizorb and the effects of ivacaftor/lumacaftor on SIBO, assessing newer approaches to old problems such as vitamin D deficiency and infant feeding, and exploring the etiology of the challenging problem of chronic pancreatitis.

Learning Objectives:

  1. Discuss outcome of in-line pancreatic exocrine replacement therapy in people with CF receiving enteral tube feeds.
  2. Discuss the evolving role of probiotic therapy in CF.
  3. Evaluate the role of CFTR in chronic pancreatitis.

Workshop Chair(s):
  • Meghana N. Sathe, M.D., Co-director of Pediatric CF Center and TDN, Univ. of Texas Southwestern Medical School
  • Adam Stein, M.D., MD, Northwestern University
Workshop Speaker(s):

W25: CLIN: Quality Improvement in Clinical Research

Nov 4, 2017 10:30am ‐ Nov 4, 2017 11:50am

Identification: W25

This session will discuss specific Quality Improvement projects and Best Practices related to the clinical research process. It will feature recent projects that have been conducted at research sites over the past year.

Learning Objectives:

  1. Identify at least one Best Practice that you can implement at your site.
  2. Describe one potential Quality Improvement project that could benefit your current program.
  3. Explain how improving the Culture of Research at your site translates into increased interest and knowledge about participation in clinical trials.

Workshop Chair(s):
  • Zoe Davies, CPNP, CCRC, Pulmonary Nurse Practitioner, Stanford CF Center
  • Colleen E. Dunn, M.S., R.R.T., CCRC, Translational Research Manager
Workshop Speaker(s):

W26: GMS: CF Model Systems: New Insights Through Cell & Animal Models

Nov 4, 2017 10:30am ‐ Nov 4, 2017 11:50am

Identification: W26

This workshop will focus on a range of experimental models available and under development for studies in CF. We will focus on the utility of each model, from cell culture to large animals, with particular emphasis on how each might be used to add value to the field.

Learning Objectives:

  1. Summarize newly developed in vitro models of CF.
  2. Summarize new animal models for in vivo studies of CF disease.
  3. Identify new outcome measures from in vitro and in vivo models alike.

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

W27: INF/MIC: Microbial Ecology of CF Airways

Nov 4, 2017 10:30am ‐ Nov 4, 2017 11:50am

Identification: W27

The recent surge in both culture-dependent and culture-independent surveys of the lung microbiota underscore the polymicrobial nature of CF lung disease. The challenge now is to understand how interactions between microbial community members and their local environment contribute to disease states. The purpose of this workshop is to highlight recent advances in this area, and to better understand the complex interplay between bacterial communities and the host airway.

Learning Objectives:

  1. Distinguish the structure and function of microbial communities and how their temporal dynamics are linked to disease state.
  2. Describe the contributions of interspecies bacterial interactions to pathogenesis. 3
  3. Discuss recent in vitro and in situ studies of bacterial pathogens and their interactions with host cells.

Workshop Chair(s):
  • Andrea Hahn, M.D., M.S., Assistant Professor of Pediatrics, Children's National Medical Center
  • Ryan Hunter, Ph.D., University Of Minnesota
Workshop Speaker(s):
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