Study Participants
Between October 2019 and May 2020, 124 patients initiated TCT. Nine
patients were excluded from the analysis for the following reasons: two
patients due to pregnancy during their baseline visits; five patients
aged up to 20 years old during their first year on TCT and their BMI
percentile could not be calculated after their 20th birthday; and two
patients underwent bilateral lung transplantation. Finally, nine
patients were excluded for being lost to follow-up after a year of TCT.
Ultimately, 106 patients were included in the study and analyzed for
weight gain while on TCT.
Of these patients, 60.4% were male, and 39.6% were female. Sixty-eight
participants were 20 years or older, and 38 were under 20 years old.
Sixty-two participants had the homozygous F508del genotype, while the
other 44 had a heterozygous F508del genotype. Twelve participants were
previously treated with ivacaftor, 18 were previously treated with
tezacaftor/ivacaftor, 48 were previously treated with
lumacaftor/ivacaftor, and the remaining 28 participants had not been
previously treated with a modulator. The participants’ mean (SD) FEV1
percent predicted was 74.92 (26.31)%. Of all subjects, 86.8% were
pancreatic insufficient based on their pancrelipase usage. Fifteen
participants were identified as having CFRD; 60 had GERD. Twenty-six
patients were on calorie-boosting supplementation; 24 used oral
supplementation and two used tube feed. (Table 1)