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Optimising inhaled mannitol for cystic fibrosis in an adult population.

Flume PA, Aitken ML, Bilton D, Agent P, Charlton B, Forster E, Fox HG, Hebestreit H, Kolbe J, Zuckerman JB, Button BM - Breathe (Sheff) (2015)

Bottom Line: Clinical experience with inhaled mannitol confirms that it is safe and effective.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.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.

View Article: PubMed Central - PubMed

Affiliation: Flume: Pulmonary and Critical Care Medicine, Medical University of South Carolina, Charleston, SC, USA.

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.

No MeSH data available.


Related in: MedlinePlus

Change in FEV1 from baseline sustained during double-blind phase of studies (intention-to-treat).
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Figure 3: Change in FEV1 from baseline sustained during double-blind phase of studies (intention-to-treat).

Mentions: For the pooled analysis, 317 adult subjects (188 mannitol and 129 control) had FEV1 data at baseline and one post-baseline visit. Over 26 weeks, mannitol improved lung function as demonstrated by a mean absolute change in FEV1 from baseline compared with control of +99.5 mL (95% CI 49.1–149.9; p=0.001) and a relative change in % predicted for normal compared with control of +4.72% (95% CI 1.86–7.59; p=0.001). Statistically significant improvements in FEV1 were sustained over the duration of the double-blind phase (fig. 3). Improvements in lung function were maintained for an additional 26 weeks in mannitol-treated subjects who consented to enter the open-label phase, indicating that the beneficial effects of mannitol on lung function can be sustained with prolonged treatment (fig. 4).Figure 3


Optimising inhaled mannitol for cystic fibrosis in an adult population.

Flume PA, Aitken ML, Bilton D, Agent P, Charlton B, Forster E, Fox HG, Hebestreit H, Kolbe J, Zuckerman JB, Button BM - Breathe (Sheff) (2015)

Change in FEV1 from baseline sustained during double-blind phase of studies (intention-to-treat).
© Copyright Policy - open-access
Related In: Results  -  Collection

License
Show All Figures
getmorefigures.php?uid=PMC4487380&req=5

Figure 3: Change in FEV1 from baseline sustained during double-blind phase of studies (intention-to-treat).
Mentions: For the pooled analysis, 317 adult subjects (188 mannitol and 129 control) had FEV1 data at baseline and one post-baseline visit. Over 26 weeks, mannitol improved lung function as demonstrated by a mean absolute change in FEV1 from baseline compared with control of +99.5 mL (95% CI 49.1–149.9; p=0.001) and a relative change in % predicted for normal compared with control of +4.72% (95% CI 1.86–7.59; p=0.001). Statistically significant improvements in FEV1 were sustained over the duration of the double-blind phase (fig. 3). Improvements in lung function were maintained for an additional 26 weeks in mannitol-treated subjects who consented to enter the open-label phase, indicating that the beneficial effects of mannitol on lung function can be sustained with prolonged treatment (fig. 4).Figure 3

Bottom Line: Clinical experience with inhaled mannitol confirms that it is safe and effective.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.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.

View Article: PubMed Central - PubMed

Affiliation: Flume: Pulmonary and Critical Care Medicine, Medical University of South Carolina, Charleston, SC, USA.

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.

No MeSH data available.


Related in: MedlinePlus