Treatment of cystic fibrosis: drugs, inhalation and supportive therapy

Cystic fibrosis is a disease that affects many organs of the human body. It is a multi-organ disease. Therefore, it is important to address the therapy comprehensively and carry out treatment aimed at each organ system separately.

Treatment of lung damage

• In CF, the most important therapy is to thin the mucus in the airways. Reducing the density of mucus will improve the cleansing function of the mucosa. Various inhaled products that liquefy mucus are used in therapy.

For example, acetylcysteine or carbocysteine are used. When taken orally, they also reduce the viscosity of mucus in the gastrointestinal tract.

• Inhaled beta-mimetics are also used, for example, in asthma. They relieve obstruction of the lower airways - the bronchi.

Inhaling solutions such as a 6% NaCl solution or an amiloride solution effectively hydrates the sputum. This greatly facilitates its expectoration.

• DNA molecules are released from the nuclei of dead bacteria, mucous cells and leukocytes, which also thicken the sputum. The enzyme that dissolves the DNA fibres is called DNAase or alpha dornase. This is an enzyme created by scientists using genetic engineering methods.

• Antimicrobial therapy is an essential part of treatment. CF patients have airways colonized with resistant clones of bacteria such as Staphylococcus, Haemophilus, Pseudomonas, Burkholderia cepacia or Stenotrophomonas.

Sustained prophylactic administration of oral antibiotics is only possible for certain strains. Antibiotics are absorbed differently in CF patients than in healthy patients. Therefore, a longer duration of use is necessary.

Intravenous administration is most effective, especially for pathogens such as Pseudomonas, Burkholderia cepacia and Stenotrophomonas. If chronic colonisation with these strains is present, prophylactic intravenous re-treatment every 3 months for 14 days is recommended.

When colonization of the respiratory tract by fungi and yeasts, antifungal agents are included in the treatment.

Analgesics, non-steroidal anti-inflammatory drugs and corticosteroids, e.g. prednisone, are supportive treatment for respiratory infections.

• Modern treatments include two types of drugs. One contains a lumafactor and an ivafactor and the other contains three active ingredients, an ivafactor, a terafactor and an elexafactor.
  • These drugs improve lung function by increasing the number of chloride channels.
  • The combination of active ingredients also improves the function of pre-existing channels. These so-called CFTR protein modulators reduce the number of hospital admissions and the need for antibiotic treatment in CF patients.
  • There are even studies that they improve the function of other organs, such as the liver.

Treatment of digestive problems and malnutrition

• Taking pancreatic enzymes in gastro-resistant tablets (tablets that resist stomach acid) improves digestion and absorption of important nutrients, especially fats and proteins.
• Damage to the pancreas causes diabetes called diabetes mellitus related to CF. When diabetes is diagnosed, the diabetes specialist will start treatment with insulin.
• Hepatoprotective agents are drugs that protect liver tissue and make it easier for the liver to excrete bile acids. For example, ursodeoxycholic acid is used.
• Mucolytics, such as acetylcysteine, are mucus thinners. When given orally, they can improve the absorption of important nutrients and reduce the risk of intestinal obstruction.
• Patients with CF require a high-calorie diet and vitamin replacement. Vitamin therapy focuses primarily on dietary supplements containing fat-soluble vitamins (vitamins A, D, E, K).

A high caloric intake is ensured by a diet containing high quality proteins and fats with limitation of rapidly absorbed sugars. Nutridrins are suitable supplements that increase caloric value and are enriched with valuable trace elements.

When conservative pharmacotherapy fails, organ transplantation is necessary in patients with CF. In some individuals, transplantation is planned early, e.g. in adolescence.