Current Strategies for the Long-term Assessment, Monitoring, and Management of Cystic Fibrosis Patients Treated with CFTR Modulator Therapy

The content for this activity is based on the satellite symposium, “Current Strategies for the Long-term Assessment, Monitoring, and Management for Cystic Fibrosis Patients Treated with CFTR Modulator Therapy” that was presented at the 39th European Cystic Fibrosis Society Conference on June 10, 2016. The emergence of novel targeted agents, that directly correct CFTR loss function alleles, has created new treatment opportunities for patients with cystic fibrosis with advanced disease. Knowledge of the role of these agents in the clinical setting is quickly evolving and will require physicians stay acquainted with the latest data as well as evidence-based treatment guidelines in order to achieve optimized cystic fibrosis patient care. Ideally, after diagnosis, a personalized approach would be adapted and tailored to the patient through genome-informed medicine. However, due to the relative recentness of genomic-based therapeutics, physicians may have a limited knowledge base regarding these new treatment options and how to best incorporate these agents into patient management plans. Although cystic fibrosis is still largely regarded as a pediatric disease, the median survival for patients is 35 years of age. Consequently, pediatric-to-adult cystic fibrosis care programs would allow suitable preparation time for this transition and develop a standardized group of self-care and management skills.

Target Audience

This activity has been designed to meet the educational needs of pulmonologists, pediatricians, nurses, and other healthcare clinicians responsible for the care of patients with cystic fibrosis.

Learning Objectives

Upon completion of this activity, participants should be better able to:

  1. Discuss the importance of accurate diagnosis, genomic evaluation, and early pharmacologic intervention.
  2. Analyze the current relevant and emerging clinical trial data.
  3. Develop strategies for the CFTR modulated treated patient for accurate life-long assessment, monitoring, and management.


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J. Stuart Elborn, MD (Chair)
Professor of Respiratory Medicine
Dean, School of Medicine, Dentistry and Biomedical Science
Queen’s University of Belfast
Belfast, Northern Ireland, United Kingdom

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Jane Davies, MD
Professor of Paediatric Respirology and Experimental Medicine
Imperial College London
Honorary Consultant in Paediatric Respiratory Medicine
Royal Brompton and Harefield NHS Foundation Trust
London, United Kingdom

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Marcus A. Mall, MD
Professor of Pediatrics
Chairman, Translational Lung Research Center Heidelberg (TLRC)
Director, Department Translational Pulmonology
Head, Division of Pediatric Pulmonology and Allergy and Cystic Fibrosis Center
University of Heidelberg
Director, German Center for Lung Research
Heidelberg, Germany

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Patrick A. Flume, MD
Professor of Medicine and Pediatrics
Director, Adult Cystic Fibrosis Program
Medical University of South Carolina
Charleston, South Carolina, United States

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Barry Plant, MD
Consultant Respiratory Physician
Director, Adult Cystic Fibrosis Centre
Cork University Hospital
Professor, Department of Medicine
Cork University Hospital
University College Cork
Wilton, Cork, Ireland


Disclosure of Conflicts of Interest

It is the policy of the Elsevier Office of Continuing Medical Education (EOCME) that all faculty, instructors, and planners disclose any real or apparent conflict of interest relating to the topics of this educational activity. 

The faculty reported the following financial relationships or relationships to products or devices they or their spouse/life partner have with commercial interests related to the content of this continuing education activity:

Name of Faculty or Presenter

Relationship Identified With:

J. Stuart Elborn, MD

Consultant/Advisor: Novartis Pharmaceuticals Corporation; Vertex Pharmaceuticals Incorporated

Grant/Research Support: Basilea Pharmaceutica Ltd.; Novartis Pharmaceuticals Corporation

Jane Davies, MD

Consultant/Advisor: Bayer Pharma AG; Chiesi Farmaceutici S.p.A.; Galapagos NV; Gilead Sciences, Inc.; Pharmaxis Ltd; PTC Therapeutics; Vertex Pharmaceuticals Incorporated

Marcus A. Mall, MD

Consultant/Advisor: Vertex Pharmaceuticals Incorporated

Patrick A. Flume, MD

Consultant/Advisor: Gilead Sciences, Inc.;  Insmed Incorporated; Novartis Pharmaceuticals Corporation; Pharmaxis Ltd;  Savara Inc; Vertex Pharmaceuticals Incorporated

Grant/Research Support:  Bayer Pharma AG;  Cystic Fibrosis Foundation; Gilead Sciences, Inc.;  Hill-Rom; Insmed Incorporated; National Institutes of Health; Nivalis Therapeutics; Novartis Pharmaceuticals Corporation; Pharmaxis Ltd;  Savara Inc; Vertex Pharmaceuticals Incorporated

Barry Plant, MDConsultant/Advisor:  Gilead Sciences, Inc.;  Novartis Pharmaceuticals Corporation;  Vertex Pharmaceuticals Incorporated

Planners, Managers, Reviewers: Sandy Breslow; Alison Kemp; Leah Johnson; Timothy Hayes, MD, PhD; Kristen Scollon; Nicole Brestowski, Jennifer Hodge and Bernard Abrams, MD, hereby state that neither they nor their spouse/life partner have any financial relationships to products or devices with any commercial interest related to the content of this activity of any amount during the past 12 months.

Peer Review

This CME activity has been peer-reviewed by the Journal of Cystic Fibrosis

Financial Support

This activity has been supported by an independent educational grant from Vertex Pharmaceuticals Incorporated.

Provider Information

Co-provided by the Elsevier Office of Continuing Medical Education, AcademicCME, and Dannemiller.

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CME Credit (Physicians)

The Elsevier Office of Continuing Medical Education is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

The Elsevier Office of Continuing Medical Education designates this enduring activity for a maximum of 1.5 AMA PRA Category 1 CreditsTM. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

The American Medical Association has an agreement of mutual recognition of Continuing Medical Education (CME) credits with the European Union of Medical Specialists (UEMS), the accreditation body for European countries.  Physicians interested in converting AMA PRA Category 1 Credit™ to UEMS-European Accreditation Council for Continuing Medical Education CME credits (ECMECs) should contact the UEMS at Additional information on the process of converting AMA PRA Category 1 Credits™ to ECMECs® can be found at:

CME Credit (Nurses)

Dannemiller is a provider approved by the California Board of Registered Nursing (CBRN), Provider Number 4229. This activity has been approved for 1.5 contact hours. CBRN credit is not accepted by the Michigan and Utah State licensing boards.

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Disclosure of Unlabeled Use: This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The Elsevier Office of Continuing Medical Education, AcademicCME, DanneMiller, and Vertex Pharmaceuticals Incorporated do not recommend the use of any agent outside of the labeled indications.

Disclaimer: Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patient’s conditions and possible contraindications on dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.

Course summary
Available credit: 
  • 1.50 CBRN Contact Hours
  • 1.50 AMA PRA Category 1 Credit(s)™
  • 1.50 Non-physician
Course opens: 
Course expires: 

Available Credit

  • 1.50 CBRN Contact Hours
  • 1.50 AMA PRA Category 1 Credit(s)™
  • 1.50 Non-physician
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