Cystic Fibrosis Clinical Medicine and Assessment

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This in turn leads to cytoplasmic water retention and the buildup of viscous mucus in the lungs and other areas of the body. This is particularly problematic for the lungs because the thick mucus impairs clearance of invasive particles and infectious agents to maintain a sterile environment.

Microbial contamination of airway surfaces triggers an inflammatory response, including a massive invasion by neutrophils (Rodrigues et al., 2008). As the neutrophils react to bacterial contamination of the lung tissue they generate considerable amounts of cellular debris that increases the viscosity of the mucous, primarily by the deposition of genomic DNA and elastase proteins. The result of this process is chronic microbial lung infection, chronic immune-mediated inflammation, and progressive tissue damage. Because cystic fibrosis lung disease is so severe the life expectancy used to be late teens or early adulthood, but the development of more effective methods for disease management has almost doubled life expectancy in recent years (Cystic Fibrosis Foundation, n.d.).

Haematology

There is increasing evidence that CFTR is expressed in polymorphonuclear leukocytes and may be required for proper phagosome acidification and bacterial killing (Conese et al., 2011). Impaired phagolysosomal function would explain why microbial lung infections are resistant to an immune response.

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In addition, platelets play an important role in haemostasis and may mediate some aspects of an inflammatory response. In individuals with cystic fibrosis, platelets display a number of defects and secrete proteins that signal the end of an inflammatory response. These findings have caused some researchers to debate whether stem cell therapy might be an effective treatment for cystic fibrosis, thus providing a fully functional immune system that may be able to better control microbial infections in the lungs and thus reduce how fast lung disease progresses, or even the generation of fully functional epithelial cells.

Although animal studies have revealed promising results, the efficiency of reconstituting cells expressing intact CFTR at the apical surface using bone marrow transplants, or lung engraftment of genetically-modified stem cells, has been extremely small (Conese et al., 2011). Currently, the most promising approach would be to use bone marrow mesenchymal cells to reconstitute 'wild type' function to the lung epithelium. The benefits would be reducing mucus viscosity and inflammation, and promoting tissue repair and an effective immune response......

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