Pooled analysis of two large randomised phase III inhaled mannitol studies in cystic fibrosis

J Cyst Fibros. 2013 Jul;12(4):367-76. doi: 10.1016/j.jcf.2012.11.002. Epub 2012 Dec 9.


Background: To evaluate safety and efficacy of inhaled mannitol treatment in subgroups of a large global CF population.

Methods: Data were pooled from two multicentre, double-blind, randomised, controlled, parallel group phase III studies in which 600 patients inhaled either mannitol (400 mg) or control (mannitol 50 mg) twice a day for 26 weeks.

Results: Both the mean absolute change in FEV(1) (mL) and relative change in FEV(1) by % predicted from baseline for mannitol (400 mg) versus control were statistically significant (73.42 mL, 3.56%, both p<0.001). Increases in FEV(1) were observed irrespective of rhDNase use. Significant improvements in FEV1 occurred in adults but not children (6-11) or adolescents (aged 12-17). Pulmonary exacerbation incidence was reduced by 29% (p=0.039) in the mannitol (400 mg) group.

Conclusions: Sustained six-month improvements in lung function and decreased pulmonary exacerbation incidence indicate that inhaled mannitol is an important additional drug in the treatment of CF.

Publication types

  • Clinical Trial, Phase III
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Inhalation
  • Adolescent
  • Adult
  • Child
  • Cystic Fibrosis / drug therapy*
  • Female
  • Humans
  • Male
  • Mannitol / administration & dosage*
  • Mannitol / adverse effects
  • Middle Aged
  • Young Adult


  • Mannitol