Abstract
Abstract There has been remarkable progress in the treatment of cystic fibrosis (CF) patients over the past 20 years. However, limitations of standard therapies have highlighted the need for a convenient alternative treatment to effectively target the pathophysiologic basis of CF-related disease by improving mucociliary clearance of airway secretions and consequently improve lung function and reduce respiratory exacerbations. Mannitol is an osmotic agent available as a dry powder, dispensed in a convenient disposable inhaler device for the treatment of adult patients with CF. Inhalation of mannitol as a dry powder is thought to change the viscoelastic properties of airway secretions, increase the hydration of the airway surface liquid and contribute to increased mucociliary and cough clearance of retained secretions. In two large phase 3 studies [1, 2], long-term use of inhaled mannitol resulted in a significant and clinically meaningful improvement in lung function relative to control in adult CF subjects and had an acceptable safety profile. Clinical experience with inhaled mannitol confirms that it is safe and effective. A minority of patients are unable to tolerate the medication. However, through training in proper inhaler technique and setting clear expectations regarding therapeutic effects, both the tolerance and adherence necessary for long term efficacy can be positively influenced.
Educational aims
To discuss the importance of airway clearance treatments in the management of cystic fibrosis.
To describe the clinical data that supports the use of mannitol in adult patients with cystic fibrosis.
To highlight the role of mannitol tolerance testing in screening for hyperresponsiveness.
To provide practical considerations for patient education in use of mannitol inhaler.
Key points
Inhaled mannitol is a safe and effective option in adult patients with cystic fibrosis.
Mannitol tolerance testing effectively screens for hyperresponsiveness prior to initiation of therapy.
Physiotherapists and respiratory therapists play an integral role in the introduction and maintenance of dry powder inhalation therapy.
Patient training and follow-up is important for optimising longer term adherence.
Footnotes
As a result of an erratum, the online version of this article has been revised
Conflict of interest Pharmaxis Limited sponsored the study. The study sponsor participated in the study design, data collection, data analysis, data interpretation and writing of the reports. Following completion of the trials, the data were held and analysed by the sponsor. The corresponding author had full access to all of the data, and had final responsibility for publication.
Diana Bilton has received fees for chairing an advisory board for Pharmaxis. Diana Bilton, John Kolbe, Moira Aitken, Patrick Flume, Jonathan Zucherman and Helge Hebestreit were all investigators during the studies and their institutions received standard clinical trial support from Pharmaxis. Investigators received travel support for the investigator meetings. No Investigator received any personal funding to participate in the study. Brett Charlton is the Medical Director of and holds stock options in Pharmaxis Ltd. Howard Fox is Chief Medical Officer of and holds stock options in Pharmaxis Ltd.
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