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.