Public Health and Primary CareReview Article

Cystic Fibrosis: A Novel Pharmacologic Approach to Cystic Fibrosis Transmembrane Regulator Modulation Therapy

Deborah Virant-Young, PharmD; Justin Thomas, DO; Sarah Woiderski, DO; Michelle Powers, OMS III; Joseph Carlier, OMS III; James McCarty, OMS III; Tyler Kupchick, OMS III; and Anthony Larder, OMS III
Notes and Affiliations
Notes and Affiliations

Accepted: June 8, 2015

Published: September 1, 2015

J Osteopath Med; 115(9): 546-555

Therapy for cystic fibrosis (CF) has progressed during the past several decades. Much of this progress is because of advances in genetic testing to precisely identify the underlying cause of CF transmembrane regulator (CFTR) dysfunction. However, with more than 1900 mutations that can produce a faulty CFTR, the management of CF can remain a challenge. Several innovative drugs recently approved by the Food and Drug Administration, termed genetic modulators, target the underlying disease by modulating the CFTR defect. This review provides physicians with an established simple classification scheme to guide their use of these drugs. The treatment challenge of 1900 CFTR mutations has been simplified into 6 physiologic classes, each paired with an available therapy to offer patients the most functional improvement. Drug therapy monitoring, adverse effects, and indications for discontinuation must also be considered.

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