Long-term inhaled dry powder mannitol in cystic fibrosis: An international randomized study

Moira L. Aitken*, Gabriel Bellon, Kris De Boeck, Patrick A. Flume, Howard G. Fox, David E. Geller, Eric G. Haarman, Helge U. Hebestreit, Allen Lapey, I. Manjula Schou, Jonathan B. Zuckerman, Brett Charlton

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

113 Citations (Scopus)


Rationale: New treatment strategies are needed to improve airway clearance and reduce the morbidity and the time burden associated with cystic fibrosis (CF). Objectives: Todeterminewhether long-term treatment with inhaled mannitol, an osmotic agent, improves lung function and morbidity. Methods: Double-blind, randomized, controlled trial of inhaled mannitol, 400 mg twice a day (n = 192, "treated" group) or 50 mg twice a day (n = 126, "control" group) for 26 weeks, followed by 26 weeks of open-label treatment. Measurements and Main Results: The primary endpoint was absolute change in FEV1 from baseline in treated versus control groups, averagedover the study period. Secondary endpoints included other spirometric measurements, pulmonary exacerbations, and hospitalization. Clinical, microbiologic, and laboratory safety were assessed. The treated group had a mean improvement in FEV1 of 105 ml (8.2% above baseline). The treated group had a relative improvement in FEV 1 of 3.75% (P = 0.029) versus the control group. Adverse events and sputum microbiology were similar in both treatment groups. Exacerbation rates were low, but there were fewer in the treated group (hazard ratio, 0.74; 95% confidence interval, 0.42-1.32; P = 0.31), although this was not statistically significant. In the 26-week open-label extension study, FEV 1 was maintained in the original treated group, and improved in the original control group to the same degree.

Original languageEnglish
Pages (from-to)645-652
Number of pages8
JournalAmerican Journal of Respiratory and Critical Care Medicine
Issue number6
Publication statusPublished - 15 Mar 2012
Externally publishedYes


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