Weight changes after initiating
Elexacaftor/Tezacaftor/Ivacaftor in Patients with Cystic Fibrosis
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/m2 (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.