Volume 8, Issue 5

CFTR Modulators In The Real World

In this issue:

The development of CFTR modulators has led to a new era in the treatment of cystic fibrosis (CF), changing the focus of therapy from managing the symptoms of CF to addressing the root cause of the disease.  But what do we know about the longer-term use of these agents in the real world? 

In this Issue, Dr. Gregory Sawicki, Associate Professor of Pediatrics at Harvard Medical School and Director of the Cystic Fibrosis Center at Boston Children's Hospital, reviews the recent publications describing the newer findings about the use of CFTR modulators in clinical practice.

Learning objectives:

  • Describe longer-term outcomes of ivacaftor therapy based on real-word patient registry data.
  • Identify non-pulmonary effects of longer-term ivacaftor therapy.
  • Describe real-world outcomes on initiating lumacaftor-ivacaftor therapy.



Gregory Sawicki, MD, MPH
Gregory Sawicki, MD, MPH

Director, Cystic Fibrosis Center
Boston Children's Hospital
Associate Professor of Pediatrics
Harvard Medical School
Boston, Massachusetts

Program Directors:

Peter J. Mogayzel, Jr., MD, PhD

Professor of Pediatrics
Director, Cystic Fibrosis Center
Johns Hopkins University School of Medicine
Baltimore, MD

Noah Lechtzin, MD, MHA

Director, Adult Cystic Fibrosis Program
Associate Professor of Medicine
Johns Hopkins University School of Medicine
Baltimore, MD

Suzanne Sullivan, RN, BSN

Senior Clinical Nurse
The Johns Hopkins University
Baltimore, MD

Length of activity:

1.0 hour Physicians
1.0 contact hour Nurses

Launch date: February 13, 2019
Expiration date: February 12, 2021