loading page

Heterogeneity in Reported Side Effects Following Initiation of Elexacaftor-Tezacaftor-Ivacaftor: Trikafta Experiences at a Quaternary CF Care Center
  • +8
  • Lena Papadakis,
  • Tayler Stander,
  • Jacqueline Mombourquette,
  • Christopher Richards,
  • Lael Yonker,
  • Brenden Lawton,
  • Margot Hardcastle,
  • Julia Zweifach,
  • Leonard Sicilian,
  • Lindsay Bringhurst,
  • Isabel Neuringer
Lena Papadakis
Massachusetts General Hospital
Author Profile
Tayler Stander
Massachusetts General Hospital
Author Profile
Jacqueline Mombourquette
Massachusetts General Hospital
Author Profile
Christopher Richards
Massachusetts General Hospital
Author Profile
Lael Yonker
Massachusetts General Hospital
Author Profile
Brenden Lawton
Massachusetts General Hospital
Author Profile
Margot Hardcastle
Massachusetts General Hospital
Author Profile
Julia Zweifach
Massachusetts General Hospital
Author Profile
Leonard Sicilian
Massachusetts General Hospital
Author Profile
Lindsay Bringhurst
Massachusetts General Hospital
Author Profile
Isabel Neuringer
Massachusetts General Hospital

Corresponding Author:[email protected]

Author Profile

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.
Submitted to Pediatric Pulmonology
Submission Checks Completed
Assigned to Editor
Reviewer(s) Assigned
29 Jul 2024Reviewer(s) Assigned
23 Aug 2024Review(s) Completed, Editorial Evaluation Pending
13 Sep 2024Editorial Decision: Revise Minor
29 Sep 20241st Revision Received
03 Oct 2024Submission Checks Completed
03 Oct 2024Assigned to Editor
03 Oct 2024Review(s) Completed, Editorial Evaluation Pending
03 Oct 2024Reviewer(s) Assigned
22 Oct 2024Editorial Decision: Accept