32nd Annual North American Cystic Fibrosis Conference

Oct 18, 2018 ‐ Oct 20, 2018



Sessions

S08--U&C: Cost, Coverage, Access: Preparing for a New Healthcare Landscape

Oct 18, 2018 2:00pm ‐ Oct 18, 2018 3:25pm

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. 

Symposium Chair(s): Speaker(s): Symposium Speaker(s):

S09--APP&D-NT-CLIN: The Spectrum of CF Muco-obstructive Airway Disease

Oct 19, 2018 10:30am ‐ Oct 19, 2018 11:55am

One of the fundamental pathophysiologic defects in CF is abnormal mucociliary clearance (MCC), a basic host defense mechanism. This deficiency in the CF lung leads to mucostasis, chronic bacterial infection and progressive inflammation, ultimately resulting in diminished lung function and shortened lifespan in patients with CF. As defective MCC is common among all patients with CF, this pathway represents a target for treatment that would be beneficial for all patients, regardless of genotype. Moreover, accumulating data suggests that mucus obstruction alone results in inflammation, regardless of the presence of bacteria. This symposium session will provide an overview of the mechanisms involved in mucociliary clearance and the therapeutic targets associated with them. 

Learning Objectives: 

  • Distinguish the pathophysiologic phenotypes of CF mucus obstruction, and compare and contrast with other muco-obstructive disorders. 
  • Discuss important therapeutic approaches that address abnormal mucociliary clearance in CF. 
  • Examine novel targets for therapy to slow the progression of CF lung disease.

Note: Speaker David Stoltz's presentation was not recorded at his request. 

Symposium Chair(s): Symposium Speaker(s):

S10--CFTR: Say Hello to CFTRs Little Friends

Oct 19, 2018 10:30am ‐ Oct 19, 2018 11:55am

Recent research has identified that both normal and mutant CFTR function can be modulated by endogenous and exogenous elements. This session will describe several studies focusing on how to understand current CFTR modulator compound interactions with CFTR as well as endogenous cellular mechanisms that regulate CFTR trafficking and function.

Learning Objectives:

  • Summarize the mechanism of action of existing small molecule therapies on CFTR and how they physically interact with CFTR.
  • Summarize how post-translation modification of CFTR (e.g., sumoylation) can significantly regulate CFTR maturation or degradation.
  • Describe how other endogenous proteins may alter CFTR trafficking and function through direct interactions.

Note: Speakers Christine Bear and Jue Chen's presentations were not recorded at their request. 

Symposium Chair(s): Symposium Speaker(s):

S11--CLIN: CF Liver Disease: Advances & Challenges

Oct 19, 2018 10:30am ‐ Oct 19, 2018 11:55am

The session is designed to provide insight into the current state of liver disease in CF. The session will include up to date information on the evaluation and management of CF associated liver disease. The symposium will include information relevant for clinicians, care team members and CF scientists. The focus of this symposium will be on the identification and evaluation of liver disease in CF, including unique aspects for adults with CF. Discussion of the management of advanced liver disease and therapeutic options including liver transplantation will be addressed.

Learning Objectives:

  • Define/describe the classification of liver involvement in CF and various theories of CF liver disease pathogenesis.
  • Discuss the implications of abnormal imaging findings and the role of elastography in CF.
  • Review the management of complications of portal hypertension in CF and role of liver transplantation.

Symposium Chair(s): Symposium Speaker(s):

S12--INF/MIC: Microbial Adaptation in the CF Airway

Oct 19, 2018 10:30am ‐ Oct 19, 2018 11:55am

This session focuses on the mechanism by which CF-related pathogens establish infection and persist in the host. The session will start with Staphylococcus aureus as one of the earliest and most prevalent microorganism in CF, which can persist in the airways for years or even decades. The second talk will focus on the fitness traits exhibited by Pseudomonas aeruginosa colony variants and the selective forces that underlie conversion and patho-adaptation during chronic infection. This will be followed by a talk describing how Burkholderia cenocepacia senses the amino acid-rich environment of the cystic fibrosis lung and elicits an adaptive response that is niche-specific according to nutritional differences. The final talk will describe microbiome methods and how they differ and limitations of each. This session will offer new advances in microbial pathogenesis and how the microbiome alters lung disease pathogenesis in CF.

Learning Objectives:

  • Describe microbial adaptation for Staphylococcus aureus, Pseudomonas aeruginosa and Burkholderia cenocepacia in CF.
  • Evaluate the microbiome approaches and how the study of microbiome in CF changed the way we think about pathogenesis in CF.
  • Characterize the most recent updates in the field of microbial pathogenesis in CF.

Symposium Chair(s): Symposium Speaker(s):

S13--NRS-CLIN: *The Changing Face of Pulmonary Exacerbation Treatment

Oct 19, 2018 10:30am ‐ Oct 19, 2018 11:55am

This session will provide an overview of the current trends in pulmonary exacerbation management highlighting the pros and cons of home versus hospital exacerbation treatment. In addition, models for both inpatient and outpatient exacerbation management from onset to resolution will be discussed.

Learning Objectives:

  • Describe the benefits, risks and outcome variation of treating pulmonary exacerbations in the home versus hospital setting, addressing the medical, social and economic implications of each.
  • Identify trends of home intravenous management, discussing a model of monitoring, and issues related to delivery devices, safety and drug stability.
  • Review the importance of following exacerbation to resolution and present a sample algorithm of standardized exacerbation identification and treatment.

Symposium Chair(s):
  • Stacy Bichl, A.P.N., CPNP, AE-C, Nurse Practitioner, Ann & Robert H. Lurie Children's Hospital of Chicago
  • Connie Richless, M.N., R.N., Coordinator Adult Program
Symposium Speaker(s):

S14--NT: *Progress & Promise of the CFTR Modulator Pipeline

Oct 19, 2018 10:30am ‐ Oct 19, 2018 11:55am

The drug discovery and development pipeline for CF has never been more active. In this session, we will begin by reviewing the state of play with small molecule CFTR modulators, currently clinically available in single and dual agent form and in trials as triple agents. Recognizing that patients with certain, rarer mutations are more difficult to treat, presenters will then look to the future, considering best ways of testing drugs for these groups and reviewing progress with splice and nonsense mutations.

Learning Objectives:

  • Review the current landscape of CFTR modulating agents and other drugs targeting the basic CFTR defect.
  • Illustrate the challenges inherent in drug development for patients with rare gene mutations.
  • Use two examples of rare mutation groups (splice mutations and premature truncation codons) to describe preclinical progress and anticipate routes to the clinic.

Note: Speaker Batsheva Kerem's presentation was not recorded at her request. 

Symposium Chair(s): Symposium Speaker(s):

S15--NTR-CLIN-SW/PSYCH: Multidisciplinary Approach to Nutrition

Oct 19, 2018 10:30am ‐ Oct 19, 2018 11:55am

This session will promote disciplines working together to care for patients with CF, especially those patients with complicated or complex needs. There will be one adult case presentation and one pediatric case presentation.

Learning Objectives:

  • Express the complicated and multifaceted nature of nutrition problems in children and adults with CF.
  • Categorize some of the main barriers to improving nutrition including medical and non medical.
  • Identify and summarize the roles of different professionals and members of the CF team in improving nutrition in CF patients: including dietitians, physicians, mental and behavioral health professional, social workers, and pharmacists.

Symposium Chair(s): Symposium Speaker(s):

S16--PT&RT-CLIN: The Multidisciplinary Care of the CF Patient With NTM

Oct 19, 2018 10:30am ‐ Oct 19, 2018 11:55am

Infection with nontuberculous mycobacteria is one of the most feared and poorly understood complications of CF lung disease. There is a good deal of uncertainty in identifying which patients will benefit from treatment, which antibiotics to use, proper dosing and monitoring, specific aspects of respiratory care, and how long to continue treatment. This Symposium will review the central aspects of the diagnosis and treatment of NTM in individuals with CF, based on the 2016 CFF/ECFC Guidelines. In particular, the role and coordination of the multidisciplinary team will be emphasized.

Learning Objectives:

  • Review the criteria for treatment of NTM in the context of CF lung disease.
  • Discuss the considerations for the selection and administration of antibiotics for the treatment of NTM disease.
  • Describe specific consideration for airway clearance, respiratory therapy and coordination of the multidisciplinary team in optimizing response to NTM treatment.

Symposium Chair(s): Symposium Speaker(s):

S17--CFTR: Challenging CFTR Mutations: Meeting Unmet Needs

Oct 20, 2018 2:30pm ‐ Oct 20, 2018 3:55pm

The care of CF patients who possess mutations responsive to available CFTR modulators has changed dramatically in recent years. If new modulators being tested in the clinic perform as hoped, the majority of patients with CF could be treated with at least one drug targeted to the underlying cause of their disease. However, a subset of rare variants will not share the benefit of these developments. In particular, patients with premature stop codons, splice defects, conductance mutations, and refractory folding mutants, such as N1303K, will remain an unmet need. To expand novel treatments to all patients, a deeper understanding of these variants is needed. In this session, we will explore current knowledge of these challenging molecular pathologies.

Learning Objectives:

  • Discuss the molecular and functional phenotypes of rare disease-causing variants (including premature stop codons, splicing mutations, and conductance mutations) and outline potential therapeutic strategies for correcting them in cystic fibrosis.
  • Describe development of new tools to understand the molecular and functional features of rare mutations, including those that could enable screening to identify novel therapeutics.
  • Define why challenging folding mutants, such as N1303K, are refractory to currently approved CFTR modulators.

Note: Speaker Batsheva Kerem's presentation was not recorded at her request.

Symposium Chair(s): Symposium Speaker(s):
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