Background and Importance Cystic fibrosis (CF) is a life-limiting recessive genetic disorder caused by pathogenic variants in the CFTR (cystic fibrosis transmembrane conductance regulator) gene, resulting in increased viscosity and difficult mucus clearance. Introduction of elexacaftor (ELX)/tezacaftor(TEZ)/ivacaftor(IVA) to clinical practice has brought a change in the clinical approach since they modulate CFTR.
Aim and Objectives To assess effectiveness and security of ELX/TEZ/IVA in patients on a tertiary hospital.
Material and Methods Observational, retrospective study carried out between March 2020 and September 2022, including all adult patients treated with ELX/TEZ/IVA+IVA in our hospital.
Variables included: age, sex, age of diagnose, pulmonary function: measured with % pFEV1 (median percent predicted forced expiratory volume in 1 second) and pulmonary exacerbations; treatment adjustment, adverse events and treatment suspension.
Data were collected from electronic medical records and pharmacy dispensing programs.
Thirty-one patients were included: male 45% (n=14), median of 31 years old (rank 17-45), median age of diagnosis of 4 months (0-38). Before taking ELE/TEZ/IVA+IVA, 45% (n=14) patients received TEZ/IVA+IVA as CFTR modulator; 55% (n=17) did not receive any CFTR modulator. Median length of ELX/TEZ/IVA+IVA treatment at the moment of the analysis was 9.43 months (4.5-31.4).
% pFEV1 during treatment augmented in 83% patients (n=26), slightly decreased in 13% (n=3) and did not vary in 1 patient. Two patients (6.5%) presented pulmonary exacerbations that required antibiotic treatment but not hospital admission.
Two patients (6.5%) required ELX/TEZ/IVA+IVA adjustment: one due to interactions with potent CYP3A4 inhibitors and other because of hepatic insufficiency (Child–Pugh B). Nine (29%) patients presented an increase of transaminase and/or bilirubin in clinical analysis: one patient temporarily discontinued therapy and one suspended treatment definitely.
Conclusion and Relevance The introduction of ELEX/TEZ/IVA to CF treatment has been a hopeful advance that has shown in our population to have a good safety profile -which can be managed with regular check-ups- and with a good efficacy profile, achieving an increase of % pFVE1in a short time.
References and/or Acknowledgements No conflict of interest.
Conflict of Interest No conflict of interest
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