Pooled analysis of two large randomised phase III inhaled mannitol studies in
cystic fibrosis.
Author(s): Bilton D(1), Bellon G, Charlton B, Cooper P, De Boeck K, Flume PA, Fox HG,
Gallagher CG, Geller DE, Haarman EG, Hebestreit HU, Kolbe J, Lapey A, Robinson P,
Wu J, Zuckerman JB, Aitken ML; CF301 and CF302 Investigators.
Affiliation(s): Author information:
(1)Royal Brompton Hospital, London, United Kingdom. d.bilton@rbht.nhs.uk
Publication date & source: 2013, J Cyst Fibros. , 12(4):367-76
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.
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