A Modern Era in Approaching Cystic Fibrosis

Tel: 562933-8748 Cystic fibrosis(CF) is an autosomal recessive genetic disorder characterized by multi-system manifestations and limited life expectancy. Although it is a multi-system disease its main manifestation is expressed as a progressive chronic lung disease which at the present time accounts for the vast majority of morbidity and mortality. The CF lung disease is chronic and progressive represented by bronchiectasis, recurrent pulmonary infections with tenacious secretions, mucus plugging and gradual decline in lung function. The essence of Cystic Fibrosisis a dysfunctional Cystic FibrosisTrans membrane Conductance Regulator (CFTR) protein which is deficient or defective. Median life expectancybefore 1950 was less than five years, but with the introduction of pancreatic enzymes median life expectancyrose to 10 years by 1960. As anti-staphylococcal antimicrobials were introduced into the care of CF median life expectancyby 1970 was approximately 15 years. In the 1970s anti-pseudomonas regimens were deployed and by 1980 median life expectancyapproximated 20 years. In the 1980’s and in subsequent years additional aggressive therapeutic regimens targeting primarily the lungs were introduced and they included among others better anti-pseudomonas treatments in oral and intravenous forms but more importantly in inhaled or aerosolized forms. The main prototype of antimicrobial delivered by inhalation was “TOBI" ( tobramycinby inhalation). Other important regimens included more effective airway clearance devises, inhaled Pulmozyme (DNase), hypertonic saline, anti-inflammatory agents and the ultimate intervention with lung transplantation for end-stage lung disease. The success in the period between 1980 and 2006 was manifested by improvement in the median life expectancyto approximately 37.5 years. Presently, in 2015, median life expectancyis about 40 years.