Nebuliser therapy for cystic fibrosis

Inhalation therapy can fulfil a range of treatment goals. One of these is to loosen the thick mucus and liquify it. This means it can be effectively coughed up and removed from the airways. It can also help to reduce or eliminate pathogens in the airways if an infection has already taken hold. In this case, antibiotics are administered by inhalation.

When using nebuliser therapy for cystic fibrosis, it is important to take the steps in a specific order. The following sequence is recommended ¹:

• Fast-acting bronchodilators that aim to dilate the bronchi if they are hypersensitive, or to prevent the risk of the airways constricting.
• After a suitable period of about 10 minutes, this is followed by e.g. nebulising hypertonic saline solution to liquify the thick mucus. Physiotherapy breathing exercises are also an ideal additional therapy.
• If necessary, medications such as antibiotics can be inhaled subsequently to fight an existing infection with bacteria.

To liquify the thick mucus in patients with cystic fibrosis, DNase (deoxyribonuclease), mannitol or hypertonic saline solutions are inhaled.

DNase is an enzyme that acts on the mucus by breaking down the free DNA strands that are generally released by dead neutrophils, which enter the airways through the inflammatory event. This DNA chain is a main component of the viscous mucus it. Inhalation therapy with DNase breaks down these strands and can liquify the mucus.

Mannitol is a natural sugar alcohol with an osmotic effect. Inhaling the active ingredient makes the mucus less thick and makes it easier to cough up.

Using a nebuliser with hypertonic saline solution is a long-established therapy with proven efficacy. Inhalation therapy with salt is also natural and has no known undesirable side effects. Hypertonic saline solutions use osmotic processes to act on the thick mucus and so can liquify it. They are also beneficial for infants and young children for whom there is currently no data about other therapies to treat the symptoms.²

If the airways are infected with pathogens, nebuliser therapy with antibiotics is generally prescribed. The advantage of inhalation over taking tablets: The antibiotics directly reach the site they are needed: the lungs. This minimises any side effects.

The choice of antibiotics, the pharmaceutical form and the treatment term must be defined by the medical experts and must be strictly observed. There are state-of-the-art inhalation devices that are optimally adapted for nebuliser therapy with the specific antibiotic, such as the eFlow^®rapid inhalation system and serveral specific variants of it.

Over the course of cystic fibrosis, the lungs will generally be infected with various pathogens. In the early stages, Staphylococcus aureus is the most common. As the patient gets older, the dominant infection is often chronic infection with Pseudomonas aeruginosa (PSA). This is associated with a rapid loss of lung function and frequent exacerbations (sudden flare-ups).²

There are various inhaled antibiotics available for the suppressive treatment of PSA infections. Their use depends on the patient’s individual tolerance and other patient-related considerations.²

Accumulations of secretions in the nose and paranasal sinuses provide a favourable environment for the colonisation and growth of viruses and bacteria. In some patients, the initial infection with Staphylococcus aureus and Pseudomonas aeruginosa actually starts in the upper airways. If the pathogen is only treated in the lungs, it may be that it persists in the sinuses. It is then often only a matter of time for the infection to move from the upper airways back down into the lungs. 

It is a major challenge for the active agent to reach the sinuses. The PARI SINUS2 is a nebuliser system that is designed to transport active agents into the sinuses with pinpoint accuracy, thanks to the pulsation technology and special application. Inhalation of antibiotics by pulsation can be considered a promising approach to the treatment of infection with Pseudomonas aeruginosa in the sinuses.²

Nebuliser therapy is the most important column of symptomatic therapy for cystic fibrosis.It follows a treatment plan tailored to the patient and their current situation. The goals of therapy include dilating the bronchi, loosening the mucus (secretolysis) and, if necessary, fighting pathogen infection. These treatment steps follow a specific sequence in cystic fibrosis.

Other links on the subject:

https://www.pari.com/de/blog/inhalation-triple-cftr-modulator-interview/   
https://www.pari.com/de/blog/inhalieren-trotz-triple-cftr-modulatoren/  

References

_{^[1]  Ballmann M, et al, CF-Manual, 3. Auflage 2022, Thieme Verlag
^[2]  Grasemann H, Ratjen F, N Engl J Med 2023;389:1693-707
^[3]  Mainz JG, et al, Drug Des Devel Ther. 2014 Feb 10;8:209-17 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3930477/pdf/dddt-8-209.pdf}