How to recognize and follow patients with cystic fibrosis in the era of new drugs

Issue 2/2023

Baicheva1, M., Petrova2, G.
1 Clinic of Gastroenterology, SBALDB “Prof. Ivan Mitov”, Department of Pediatrics, Sofia University
2 Clinic of Pediatrics UMBAL “Alexandrovska” EAD, Department of Pediatrics, Medical University – Sofia

Cystic fibrosis (CF) is a complex autosomal recessive disease in which the functions of the respiratory system, digestive tract and all exocrine glands are affected. The disease is caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, whose main function is to regulate the volume of fluids on the epithelial surface by secreting chlorides and suppressing sodium absorption. The newest medications for the disease are CFTR modulators and known in the literature as gene modifiers. Improvements in care and these new therapeutic options have led to increased life expectancy for these patients. The article outlines the main symptoms that point to CF and how these patients should be followed up.

Address for correspondence:
Baicheva, M. 
Clinic of Pediatrics, SBALDB „Prof. Ivan Mitov“ – Sofia
11, Аcad. Ivan Geshov, Blvd. 
1612, Sofia