| Title: | Real -World Safety and tolerability of Elexacaftor/Tezacaftor/Ivacaftor in the treatment of Cystic Fibrosis: A single centre experience. |
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| Author(s): | Michelle Casey Claudie Gabillard-Lefort Orla Kerr Elaine Marron Anne-Marie Lyons Ciara Reddy Cedric Gunaratnam Noel G. McElvaney |
| Institution: | 1. The Irish Centre of Genetic Lung Disease, Department of Medicine, Royal College of Surgeons in Ireland, Dublin, Ireland. 2. Department of Respiratory Medicine, Beaumont Hospital, Ireland. |
| Poster: | Click to view poster |
| Category: | CF and Pulmonary Infections |
| Abstract: | Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies, including the most recent combination Elexacaftor/Tezacaftor/Ivacaftor(Elex/Tez/Iva) are generally well tolerated; however, real-world studies of previous combinations indicate the frequency of adverse events(AEs) and discontinuation may be greater then that observed in clinical trials. We prospectively assessed the safety, tolerability, and clinical efficacy of Elex/Tez/Iva in patients with Cystic Fibrosis(PWCF) at 3monthly periods post initiation of treatment. A total of 55 patients have been initiated on therapy to date, with 6-month follow-up data available for 30 patients across a spectrum of disease severity. The most common AEs reported are listed in Table1, with no patient discontinuing treatment and treatment interruption required for 3(10%) patients. In our experience most AEs have been mild and of short duration. Interestingly, we describe adverse events not reported in the original studies, such as the neurocognitive side effects and gender specific side effects of testicular swelling/discomfort in men and increased vaginal discharge in women. As the number of PWCF commencing CFTR modulator therapy increases, it is essential CF teams are aware of all potential AEs and that systematic approaches for prevention and/or management of AEs are developed. Table 1: Adverse events reported 3 months post-Kaftrio in PWCF. Total number of PWCF included: 30, N= number of patients (%). Adverse events PWCF N (%) Gastrointestinal cramps 15 (50%) Sleep disturbance 12 (40%) Anxiety 9 (30%) Testicular pain/swelling 7 (23.3%) Vaginal discharge 6 (20%) Deranged liver function tests 4 (13%) Mucus plugging 3 (10%) Headache 3 (10%) Worsening Acne 2 (7%) Rash 2(7%) |
