PMID- 21478216
OWN - NLM
STAT- MEDLINE
DCOM- 20120620
LR  - 20131121
IS  - 1399-3003 (Electronic)
IS  - 0903-1936 (Linking)
VI  - 38
IP  - 5
DP  - 2011 Nov
TI  - Inhaled dry powder mannitol in cystic fibrosis: an efficacy and safety study.
PG  - 1071-80
LID - 10.1183/09031936.00187510 [doi]
AB  - This international phase III study of inhaled dry powder mannitol was a 
      randomised, double-blind, 26-week study, followed by a further 26-week, 
      open-label (OL) extension. 324 cystic fibrosis (CF) patients were randomised, in 
      a 3:2 ratio, to mannitol (400 mg b.i.d.) and control groups. The primary efficacy 
      end-point was to determine the change in forced expiratory volume in 1 s (FEV(1)) 
      over the double-blind phase. Secondary end-points included changes in forced 
      vital capacity and pulmonary exacerbations. A significant improvement in FEV(1) was 
      seen over 26 weeks (p<0.001) and was apparent by 6 weeks, irrespective of 
      concomitant recombinant human deoxyribonuclease (rhDNase) use. At 26 weeks, there 
      was a significant improvement in FEV(1) of 92.9 mL for subjects receiving mannitol 
      compared with controls (change from baseline 118.9 mL (6.5%) versus 26.0 mL 
      (2.4%); p<0.001). Improvements in FEV(1) were maintained up to 52 weeks in the OL 
      part of the study. There was a 35.4% reduction in the incidence of having an 
      exacerbation on mannitol (p=0.045). The incidence of adverse events (AEs) was 
      similar in both groups, although treatment-related AEs were higher in the 
      mannitol compared with the control group. The most common mannitol-related AEs 
      were cough, haemoptysis and pharyngolaryngeal pain. Mannitol showed sustained, 
      clinically meaningful benefit in airway function in CF, irrespective of 
      concomitant rhDNase use. Mannitol appears to have an acceptable safety profile 
      for patients with CF.
FAU - Bilton, D
AU  - Bilton D
AD  - Royal Brompton Hospital, London, SW3 6NP, UK. d.bilton@rbht.nhs.uk
FAU - Robinson, P
AU  - Robinson P
FAU - Cooper, P
AU  - Cooper P
FAU - Gallagher, C G
AU  - Gallagher CG
FAU - Kolbe, J
AU  - Kolbe J
FAU - Fox, H
AU  - Fox H
FAU - Jaques, A
AU  - Jaques A
FAU - Charlton, B
AU  - Charlton B
CN  - CF301 Study Investigators
LA  - eng
SI  - ClinicalTrials.gov/NCT00446680
PT  - Clinical Trial, Phase III
PT  - Journal Article
PT  - Multicenter Study
PT  - Randomized Controlled Trial
PT  - Research Support, Non-U.S. Gov't
DEP - 20110408
PL  - England
TA  - Eur Respir J
JT  - The European respiratory journal
JID - 8803460
RN  - 0 (Recombinant Proteins)
RN  - 3OWL53L36A (Mannitol)
RN  - EC 3.1.- (Deoxyribonucleases)
SB  - IM
MH  - Administration, Inhalation
MH  - Adolescent
MH  - Child
MH  - Cystic Fibrosis/*drug therapy/physiopathology
MH  - Deoxyribonucleases/therapeutic use
MH  - Double-Blind Method
MH  - *Dry Powder Inhalers
MH  - Female
MH  - Forced Expiratory Volume
MH  - Humans
MH  - Male
MH  - Mannitol/*administration & dosage/adverse effects
MH  - Recombinant Proteins/therapeutic use
MH  - Vital Capacity
EDAT- 2011/04/12 06:00
MHDA- 2012/06/21 06:00
CRDT- 2011/04/12 06:00
PHST- 2011/04/12 06:00 [entrez]
PHST- 2011/04/12 06:00 [pubmed]
PHST- 2012/06/21 06:00 [medline]
AID - 09031936.00187510 [pii]
AID - 10.1183/09031936.00187510 [doi]
PST - ppublish
SO  - Eur Respir J. 2011 Nov;38(5):1071-80. doi: 10.1183/09031936.00187510. Epub 2011 
      Apr 8.