Answering the call to address cystic fibrosis treatment burden in the era of highly effective CFTR modulator therapy.

Background: We recognize an unprecedented opportunity to study the effects of withdrawing one or more chronic treatments in people with CF (PwCF) who benefit greatly from CFTR modulator therapy, but feasibility and acceptance of such a study within the community is unknown.
Methods: We surveyed PwCF, their families, and their acquaintances between November 16, 2018, and December 2, 2018, and CF clinicians between December 19, 2018, and January 2, 2019, about treatment withdrawal research. We sought feedback from these groups about their level of interest in this research, the consistency with which they were taking modulator and non-modulator treatments, the ways in which they conceptualized health changes, and what chronic non-modulator treatments they were most interested in stopping. We also asked for stakeholder perspectives on the design of a treatment withdrawal trial, but we intend to report these perspectives elsewhere.
Results: Eighty percent (541/675) of CF community respondents and 95% (206/218) of CF clinicians said that a trial of treatment simplification should be performed in the context of highly effective modulator therapy. Most current CFTR modulator users (292/359, 81%) have not stopped another chronic treatment. Worsening lung function by spirometry or increased daily symptoms were important health indicators. PwCF, their families, and/or their acquaintances ranked airway clearance techniques and inhaled antibiotics as the most burdensome treatments.
Conclusions: There is considerable support among the CF community and CF clinicians in the U.S. for controlled trials to assess the safety and impact of treatment simplification in patients taking highly effective modulator therapy.
Competing Interests: Conflict of Interest Statement Dr. Alex H. Gifford has received grant funding from the Cystic Fibrosis Foundation. Dr. Nicole Mayer-Hamblett has no conflicts of interest to declare. Ms. Kelsie Pearson has no conflicts of interest to declare. Dr. David P. Nichols has received grant funding from the Cystic Fibrosis Foundation.
(Copyright © 2019. Published by Elsevier B.V.)