Contemporary treatment of cystic fibrosis incorporates a combination therapy of elexacaftor, tezacaftor, and ivacaftor, which are CFTR modulators. Cystic fibrosis patients are characterized by the fact that the chloride channel (CFTR), which is expressed on the membrane of many cell types, does not function properly due to a mutation. As a result, dysfunction occurs in various organ systems, especially in the lungs. Recent studies have also revealed the presence of endothelial dysfunction, which is a key factor in the development of cardiovascular disease.
The aim of our work was therefore to investigate the clinical course of the disease cystic fibrosis and endothelial dysfunction in a patient with an advanced and unstable form of cystic fibrosis before and after starting treatment with the above-mentioned combination drug.
Over three years, encompassing the period before and after drug administration, we conducted a comprehensive analysis including clinical assessments, medical history reviews, laboratory blood tests, lung function evaluations, lung X-ray analyses, and extracted relevant data from the patient’s medical records. Importantly, we performed periodic measurements of endothelial function during the three-year treatment period.
Our findings indicate favorable long-term outcomes of the combination therapy on the clinical trajectory of advanced cystic fibrosis. Notable improvements were observed in the reduction of acute pulmonary exacerbation frequency and severity, nutritional status, lung function, and the structural condition of the lungs, as well as standard disease monitoring laboratory parameters. The therapy also significantly enhanced endothelial function over short and long terms in the absence of acute pulmonary incidents. However, the therapeutic benefits appeared to diminish during concurrent acute pulmonary exacerbations characterized by inflammatory onset.
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