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)