Effets indésirables de la thérapie par elexacaftor, tezacaftor et ivacaftor (Kaftrio®) et impacts chez les patients suivis aux Cliniques universitaires Saint-Luc. Adaptation(s) et autoadaptation(s) des traitements médicamenteux.

Details

Supervisors

Henri, Laurence ; Berardis, Silvia

Faculty

Faculté de pharmacie et des sciences biomédicales

Degree label

Master de spécialisation en pharmacie hospitalière

Abstract

Background: Cystic fibrosis (CF) is a prevalent autosomal recessive disease among Caucasians, affecting approximately 1 in 2850 newborns. The mutation of the CFTR gene, located on chromosome 7, leads to dysfunctional CFTR protein, affecting chloride ion transport and resulting in thick mucus affecting organs such as the lungs, pancreas, and liver. Kaftrio® (a combination of elexacaftor, tezacaftor, and ivacaftor) has been shown to improve respiratory function and is reimbursed in Belgium for adults with at least one F508del mutation since September 2022. Objective: This study aims to evaluate adverse effects and treatment adaptations following the introduction of Kaftrio® in patients with CF at Cliniques universitaires Saint-Luc. Methods: A retrospective and prospective study was conducted at the Cliniques universitaires Saint-Luc, including 112 patient records and 55 completed questionnaires. Data on adverse effects, treatment modifications, and patient demographics were collected and analyzed. Results: Over half of the patients (n = 66, 58.9%) experienced at least one adverse effect, with the most common being pulmonary exacerbations (32.1%), abdominal pains (6.3%), headaches (6.3%), and skin rashes (4.5%). Six patients (5.4%) discontinued Kaftrio® permanently due to severe adverse effects. Treatment modifications concerning Kaftrio® included temporary discontinuation (n = 1, 0.9%), dose adjustments (n = 2, 1.8%), and changes in the timing of medication administration (n = 2, 1.8%). Furthermore, changes regarding other treatments besides Kaftrio® have been considered, primarily involving discontinuations or dose reductions. Conclusion: The introduction of Kaftrio® in CF patients led to notable improvements in patient outcomes but also presented significant adverse effects in a substantial proportion of patients. These findings underscore the need for personalized monitoring and potential reporting to regulatory bodies to ensure patient safety and optimize therapeutic efficacy.