© 2002 Thorax
EDITORIAL
Cystic fibrosis
How do we choose a therapeutic regimen in cystic fibrosis?
Correspondence to:
Correspondence to:
A/Prof CF Robertson, Department of Respiratory Medicine, Royal Children’s Hospital, Parkville 3052, Victoria, Australia;
cfrob@cryptic.rch.unimelb.edu.au
As the range of treatments for cystic fibrosis increases and the costs rise, the clinician is faced with an increasingly complex dilemma to develop a treatment schedule that provides optimal benefit, given financial restraints and the impact of an increasingly complex therapeutic regimen on patient adherence.
Keywords: cystic fibrosis; rhDNase; hypertonic saline; cost
| The first 150 words of the full text of this article appear below. |
Over the past 20 years there has been an exponential increase in research to develop new treatments for the management of cystic fibrosis—particularly following the identification of the CFTR protein and its impact on the composition and function of the surface epithelial cell airway surface liquid. While early progress was achieved in gene therapy, it is the pharmacological modulation of function of the CFTR protein and the airway surface liquid that is more likely to be translated into treatment.
One of the major consequences of the defect in CFTR production is alteration of the airway surface liquid to impair mucociliary clearance and promote infection. There have been several attempts to alter the properties of the airway surface liquid and to reduce the viscosity of the mucus to improve mucociliary clearance. Agents investigated include recombinant human rhDNase,1 hypertonic saline,2 dry powder mannitol,3 amiloride,2 and 5'-uridine triphosphate.4 Each has shown
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