Budesonide is a widely used corticosteroid to help prevent the symptoms of asthma. Although it has not been approved by the U.S. Food and Drug Administration specifically for the treatment of cystic fibrosis (CF), it can be prescribed to alleviate disease symptoms in this group of patients.

How budesonide works

Corticosteroids such as budesonide are anti-inflammatory agents or medicines that act by reducing inflammation.

CF is characterized by the production of thick, sticky mucus that accumulates in various organs, including the lungs. This happens because the transport of water is compromised across the cystic fibrosis transmembrane regulator (CFTR) protein — a channel present on the surface of mucus-producing cells. This alters the watery consistency of the mucus, leading to the formation of thick,  sticky mucus.

Because this mucus is rich in polysaccharides and proteins, it acts as an ideal medium for the growth of airborne bacteria that have easy access to the lungs, causing chronic lung infections. As the body tries to clear the infection, it triggers an inflammatory response. Prolonged inflammation can cause damage to the lungs. Anti-inflammatory agents such as budesonide may prevent lung damage by reducing inflammation in patients with CF.

Budesonide is an inhaled corticosteroid, administered through a breathing device. This offers the additional advantage of the budesonide being directly delivered to the lungs and acting only within the inflammed tissue, increasing its efficacy and reducing undesirable side effects.

Research about budesonide in CF

An early study led by investigators at University Medical Center Utrecht, in the Netherlands, evaluated the role of budesonide in the context of lung function and airway hyperresponsiveness in adult patients with CF. Around 40-60 percent of adult CF patients exhibit airway hyperresponsiveness (the increased tendency of the airways to rapidly change in diameter and stretching in response to external stimuli, causing unanticipated and rapid variability in airflow). In this study, 12 patients were treated with either 1,600 micrograms per day of budesonide or placebo in two intervals over a six-week period. The results revealed small but significant improvements in symptoms such as cough and shortness of breath.

Another early study assessed the efficacy of budesonide in 55 patients with CF and chronic Pseudomonas aeruginosa infection, a common problem in CF. The patients were treated with 800 micrograms of budesonide twice daily for six months. The patients were also given antibiotics against Pseudomonas aeruginosa. The results showed that budesonide improved lung function and provided short-term benefits.

A recent Turkish clinical study evaluated the effect of eight weeks of treatment with 2 mg per day of nebulized budesonide on 29 CF patients.  Although there was a significant decrease in the percentage of neutrophils (key mediators of the inflammation) following treatment, the treatment did not reduce the severity of the symptoms of CF.


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