Volume 9, Issue 3

Triple Combination CFTR Modulation: The Next Step in CF Therapy

In this issue:

Restoring function to the cystic fibrosis transmembrane conductance regulator (CFTR) protein has become a mainstay of CF therapy. It began with the 2012 approval of ivacaftor monotherapy, benefitting ~5% of US patients. Now, the recent approval of triple combination modulator — elexacaftor/tezacaftor/ivacaftor — promises to benefit close to 90% of the US CF population. Who are the targets of this therapy? What are the potential adverse effects? What do the data say? 

In this issue, Dr. Scott Sagel from the Breathing Institute at the Children’s Hospital Colorado, affiliated with the University of Colorado Anschutz Medical Campus, provides some answers. 

Learning objectives:

  • Evaluate the data used to support the approval of triple therapy for people with at least one copy of the F508del CFTR mutation. 
  • Describe the real-world clinical effectiveness of highly effective CFTR modulator therapy to modify CF disease progression. 

Author:

Scott Sagel, MD, PhD
Scott Sagel, MD, PhD

Professor of Pediatrics
Asher-Accurso Chair of Cystic Fibrosis
Director, Pediatric Cystic Fibrosis Center 
Breathing Institute, Children’s Hospital Colorado
University of Colorado Anschutz Medical Campus

Program Directors:

Noah Lechtzin, MD, MHA

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

Peter J. Mogayzel, Jr., MD, PhD, MBA

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

Donna Peeler, RN

Clinical Nurse 
Pediatric Clinic Coordinator 
Johns Hopkins Cystic Fibrosis Center 
Baltimore, MD

Length of activity:

1.0 hour Physicians
1.0 contact hour Nurses

Launch date: June 2, 2020
Expiration date: June 1, 2022