2016-11-23
Small Airways Disease in Cystic Fibrosis: Improving efficacy of treatment
Publication
Publication
Afwijkingen van de kleine luchtwegen in cystic fibrosis: Verbetering van de effectiviteit van behandeling
Cystic fibrosis (CF) is a severe hereditary and life-threatening disease in the Caucasian
population, affecting 70,000 patients worldwide. In the 1950s, a child with CF would
rarely live long enough to attend elementary school. Luckily, life expectancy has dramatically
improved due to the development of new treatments and treatment approaches.
The current median predicted survival is close to 40.
The main cause of mortality and also morbidity is progressive lung disease.3 Patients
with CF have an increased susceptibility to airway infections due to the defects in the CF
transmembrane conductance regulator (CFTR). Due to a dysfunction in the CFTR channel,
ion transfer over the membrane of epithelial cells is abnormal what leads to dehydration
of the airway surface liquid in the lungs. This results in thick, viscous mucus that impairs
mucociliary clearance and obstructs the airways. Because of these alterations, it is difficult
for CF patients to eliminate inhaled bacteria from the lungs. A persistent infection
arises, which damages the airways and eventually leads to respiratory failure.
The most common mutation causing CFTR dysfunction is the dF508 mutation, but over
2000 mutations have been identified. The disease is complex and the rate of progression
of CF lung disease varies widely from person to person, even among patients carrying
identical CFTR mutations. Despite the significant improvements in the treatment of the
disease, there is still no cure and lung infections remain a serious problem for patients
living with CF.
Additional Metadata | |
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H.A.W.M. Tiddens (Harm) , H.M. Janssens (Hettie) | |
Erasmus University Rotterdam | |
The studies performed in this thesis were supported by Chiesi Farmaceutici S.p.A. and Roche Pharmaceuticals. Printing of this thesis was kindly supported by Cresco Pharma, AbbVie B.V., Longfonds, Mediq Romedic, Dr. Weigert Nederland B.V., Teva Netherlands B.V., the department of Radiology and Erasmus Medical Center. | |
hdl.handle.net/1765/94117 | |
Organisation | Department of Pediatrics |
Bos, A. (2016, November 23). Small Airways Disease in Cystic Fibrosis: Improving efficacy of treatment. Retrieved from http://hdl.handle.net/1765/94117 |
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161123_Bos-Aukje-Catharina.pdf Final Version , 20mb | |
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