R4Stars Monthly Contributors

Earlier Interventions in Cystic Fibrosis
by Morton N. Schwartzman MD, FAAP, FCCP
Joe DiMaggio Cystic Fibrosis and Pulmonary Center
Medical Director

There are approximately 35,000 cases of Cystic Fibrosis in North America with a birth incidence of 1:3000. The median age of survival is 32-33 years. There are several factors that determine survival rates in CF patients; the genetic mutation makeup, prevalence of the bacterial types, antibiotic usage (oral, intravenous, inhalation), and maybe an earlier intervention program.

CF lung disease is characterized by impairment in mucociliary transport and that encourages recurrent persistent bacterial infections. These infections are associated with an intense persistent inflammatory response. This inflammation is brought about by inflammatory agents released by destroyed white blood cells (WBC’s) (neutrophils) in the lung tissue with or without bacteria.

Bacterial and viral infections during infancy can lead to an increase in prolonged inflammatory reaction in the lungs. This is the vicious cycle between infection and inflammation in CF that leads to pneumonia, scarring, bronchiectasis, and lung destruction.

Bacterial infections in CF are mainly caused by staph aureus (prominent in younger children), pseudomonas aueriginosa and other occasional bacteria formed in CF sputum. Long-term treatment to counteract staphylococcus may not eradicate it and may lead to a quicker colonization of pseudomonas auerginosa. Early interventional treatment in the young patient with non-mucoid pseudomonas may be effective in eradication and may possibly postpone the development of chronic infection.

The inhalation of high dose tobramycin solution or colistin along with certain oral and intravenous antibiotics is very beneficial in the very young CF patient with mild disease. By using tobramycin solution for inhalation there is significant reduction in hospitalization and a trend towards improvement in PFT.

The use of azithromycin in early pseudomonas can decrease inflammation and allow the pseudomonas bacteria to become more amenable to treatments by other antibiotics. Antiinflammatory treatment is another approved modality to prevent deterioration of lung function. Inhaled steroids have been used and beneficial effects are variable in decreasing inflammation and stabilizing pulmonary function.

High doses of ibuprofen slowed lung function decline, but high blood levels must be maintained to decrease inflammation. There are side effects that must be watched for. Other researched anti-inflammatory agents are being tested for future use especially in the early intervention programs.

Pulmozyme usage prevents the increase in inflammation by decreasing mucous accumulation, better airway clearance, and therefore less pulmonary exacerbations. Pulmozyme may not be indicated for every child in the early stages of CF lung disease.

Chest physiotherapy has been well established with positive documentation in the early intervention program. An aggressive nutritional approach in treating CF to meet energy needs and the potential for physical growth, lung growth, and lung function must be addressed in the early intervention program.

To date there is still insufficient evidence and information on the optimal form of therapy that should be used to intervene in early disease; yet by addressing strategies for antiinfection and anti-inflammation along with chest physiotherapy and an aggressive nutritional program may delay the onset and hopefully prevent significant lung disease and lung damage.

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