Heterogeneity in Reported Side Effects Following Initiation of
Elexacaftor-Tezacaftor-Ivacaftor: Trikafta Experiences at a Quaternary
CF Care Center
Abstract
Background The benefits of Elexecaftor-Tezacaftor-Ivacaftor
(ETI) therapy on the health and wellbeing of people with CF (pwCF) are
well documented. Since approval, however, a growing number of potential
side effects have emerged in reports from clinical practice. With
current understanding of ETI tolerability limited to data from clinical
trials, the prevalence of side effects and their impact on care decision
making remains poorly categorized. Methods A ten-question
survey was developed and distributed to patients 18 years or older who
were treated at the Massachusetts General Hospital CF centers. Reports
of side effects were measured across twelve distinct categories, and
dose adjustments and discontinuation due to side effects were collected.
If a patient reported no side effects, they did not have to complete the
entire survey. Results Among ninety-two respondents initiated
on ETI, 51 respondents (55.4%) reported potential side effects and 41
(44.5%) respondents reported no adverse events. The most commonly
reported side effects were mental health, changes in appearance, and
gastrointestinal complaints, which were reported by 22.8%, 30.4%, and
21.7% of patients, respectively. Eighteen (19.6%) respondents modified
their dosing in response to side effects, and six discontinued treatment
permanently (6.52%) due to persistent side effects.
Conclusions Responses demonstrated marked heterogeneity, with
most respondents reporting at least one side effect following
initiation. Dose modification was commonly utilized to mitigate adverse
effects, however few respondents had to discontinue treatment. These
findings demonstrate the importance of monitoring for potential
drug-related side effects of ETI in clinical settings.