The New Landscape of Cystic Fibrosis in the Era of Highly Effective Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy.

BACKGROUND

For many years, the management of cystic fibrosis has had a personalised character. We have now entered an era of therapy based on a patient's specific genotype. Clinical trials have confirmed the efficacy and safety of cystic fibrosis transmembrane conductance regulator modulator therapies, including ivacaftor as monotherapy and triple-drug combinations, such as elexacaftor/tezacaftor/ivacaftor and vanzacaftor/tezacaftor/deutivacaftor. These therapies result in substantial and sustained improvements in lung function, a reduction in the frequency of bronchopulmonary exacerbations, enhanced nutritional status, and an improved quality of life. These clinical outcomes have been correlated with a marked reduction of sweat chloride values.

MATERIAL AND METHODS

Early initiation of therapy is crucial to halt disease progression or even reverse disease-related complications, which have even been observed in children aged six to twelve years. Initiating treatment in even younger children before the onset of clinical symptoms offers the possibility of a much later disease manifestation or even absence of symptoms. Nevertheless, careful evaluation of these therapies' long-term effects on the respiratory system and other organs remains necessary.

RESULTS

According to current knowledge, patients continue to require regular follow-up by a multidisciplinary care team and ongoing adjustment of both modulator and symptomatic therapies to meet individual needs.

CONCLUSION

As patient life expectancy continues to increase, comorbidities such as arterial hypertension, hypercholesterolaemia, cardiovascular disease, and malignancies will assume greater clinical importance. Selecting optimal therapeutic strategies remains a significant clinical challenge due to the numerous potential interactions between modulators and other medications.

© 2026 Anna Wołkowicz et al., published by Institute of Mother and Child.