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Cystic fibrosis - Treatment
Medical expert of the article
Last reviewed: 06.07.2025
Cystic fibrosis is a common monogenic disease caused by a mutation in the cystic fibrosis transmembrane regulator (CFTR) gene, characterized by damage to the exocrine glands of vital organs and systems and usually having a severe course and prognosis.
The disease occurs with a frequency of 7-8:100,000 population. In 1989, the cystic fibrosis gene was isolated, and then its structure was deciphered: it contains 27 exons, covers 250,000 nucleotide pairs, located in the middle of the long arm of autosome 7. Due to the mutation of the cystic fibrosis gene, the structure and function of the CFTR protein, which acts as a chloride channel and regulates water-electrolyte metabolism in the epithelial cells of the respiratory tract, pancreas, intestines, liver, sweat glands of the reproductive system, is disrupted. Due to the disruption of the CFTR protein function, chloride ions are inside the apical part of the cell membrane. As a result, the electrical potential in the lumen of the excretory ducts changes, which contributes to an increased escape of sodium ions and water from the lumen into the cell.
As a result of these disorders, there is a thickening of the secretions of the above-mentioned glands of external secretion, difficulty in their secretion and secondary changes in these organs, most pronounced in the bronchopulmonary system.
Damage to the bronchopulmonary system in cystic fibrosis is expressed in the following clinical variants.
- bronchitis (acute, recurrent, chronic);
- pneumonia (repeated, recurrent).
As the disease progresses, it becomes complicated by atelectasis, lung abscesses, pyopneumothorax, development of bronchiectasis, and pulmonary heart disease.
Treatment of cystic fibrosis includes the following measures:
- Improving the drainage function of the bronchi, freeing the bronchial tree from viscous sputum:
- use of mucolytic expectorants;
- treatment with bronchodilators;
- kinesitherapy (positional drainage, bedbug massage and vibration massage of the chest, special coughing exercises, active breathing cycles and forced exhalation, positive exhalation pressure using a flutter or a special mask.
- Fight against bronchopulmonary system infection.
Chronic and recurrent respiratory tract infection is a major cause of morbidity and mortality in patients with cystic fibrosis.
The main causative agent of the infection is Pseudomonas aeruginosa, which is found in 70-90% of patients. According to Ficlc (1989), Pseudomonas aeruginosa almost constantly persists in the sputum of patients. Staphylococcus aureus and Haemophilus influenzae are often detected simultaneously.
Pseudomonas aeruginosa produces various factors that damage lung tissue (exotoxins A and S, alkaline protease, elastase, leukocidin, pigments), and also synthesizes a mucoid membrane consisting of a polymer of alginic acid. This membrane combines with viscous bronchial secretions, increases obstruction and complicates the effect of antibiotics on the pathogen. Pseudomonas aeruginosa is extremely resistant to beta-lactam antibiotics.
Antipseudomonas penicillins, aminoglycosides, fluoroquinolones, monobactams, carbapenems, antipseudomonas cephalosporins of the third (cefoperazone, ceftazidime) and fourth (cefpirome, cefsulodin and cefepime) generations are used to treat pseudomonas aeruginosa infection. Cefsulodin is a specific antipseudomonas antibiotic; it has a weak effect on other microorganisms. Cefoperazone is inferior to other antipseudomonas antibiotics. Ceftazidime is the most effective against pseudomonas infection. Cefoperazone and ceftazidime affect not only pseudomonas aeruginosa, but also most gram-negative bacteria. Cefpirome and cefepime are active not only against Pseudomonas aeruginosa, but also against non-positive flora, as well as Enterobacter, Citrobacter, Klebsiella and Escherichia coli.
- Correction of exocrine pancreatic insufficiency is performed by using drugs containing pancreatic enzymes. The most effective are microspherical drugs coated with an acid-resistant shell (creon, lancitrate, prolipase, pancrease).
In recent years, the possibility of treating cystic fibrosis with omiloride and sodium adenosine triphosphate, which open alternative chloride channels, has been discussed; the possibilities of treatment with anticytokines and antiinterleukins (anti-IL-2, anti-IL-8) are being studied; genetic engineering approaches to correcting the genetic defect in cystic fibrosis are being developed.
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