Weight changes after initiating Elexacaftor/Tezacaftor/Ivacaftor in
Patients with Cystic Fibrosis
Abstract
Background: Cystic Fibrosis Transmembrane Conductance Regulator
(CFTR) modulators have varying levels of benefit regarding weight gain
and growth, ultimately improving lung function and decreasing mortality
rates in Cystic Fibrosis (CF) patients. The newly approved triple
combination therapy (TCT) has shown weight gain benefits in clinical
trials, but its long-term effects have yet to be studied in a site-wide
setting. Methods: This retrospective study of 106 adult and
pediatric CF patients on triple combination therapy for a year. We
measured Body Mass Index (BMI) and BMI percentile changes and compared
changes before and one year after initiation of TCT Results:
TCT use showed weight gain over one year by increasing BMI in adult
patients by 1.48 kg/m 2 (p-value < 0.0001).
Pediatric patients saw significant benefit in BMI percentile with an
average gain of 8.34 percentile (p-value= 0.0047). Discussion:
The results of this study suggest that the new triple combination
therapy improves BMI and BMI percentile in CF patients. This finding
will help future CFF guidelines navigate the era of new modulators and
the changes in baseline health that come with it.