Table 2

Summary of RCTs in CF measuring QoL: mucolytic therapies, exercise and pancreatic enzymes

Authors
Brief description
Sample
QoL scale used
Authors' main conclusion
Authors' conclusion about QoL outcome

MUCOLYTIC THERAPIES





Ranasinha et al. [27]
DNase vs placebo; two parallel groups
n = 71 (age = 16–55 years) mean FEV1 ≈ 47%
Ad hoc 9-item scale
Significant improvement in FEV1 but not in FVC
DNase did not improve overall well-being but improvements in feeling, cough frequency and chest congestion
Ramsey et al. [28]
3 doses of DNase vs placebo; 4 parallel groups
n = 181 (age 8–65 years) mean FEV1 between 58.6% and 84.6% for the 4 groups
Ad hoc 9-item scale
FEV1 and FVC improved across all doses compared with placebo
DNase associated with decreased dyspnoea and improved well-being
Fuchs et al. [29]
2 doses of DNase vs placebo; 3 parallel groups
n= 968 (age 5–54 years) mean FEV1 ≈ 60%
Ad hoc 9-item scale
Improved lung function on DNase
Increase in general well-being
Wilmott et al. [30]
2.5 mg DNase or placebo twice daily
n = 80 children and adults (age >5 years; mean ≈ 20) mean FEV1 ≈ 40%
Ad hoc scale
No effect of drug on change in FEV1 or FVC
No differences on well-being scales
Suri et al. [31-33]
Open crossover study of DNase once daily 2.5 mg vs alternate day 2.5 mg and saline
n = 48 (age 7–17 years) (n = 40 completed study)
QWB-SA
No evidence of differences between active treatments; daily treatment better than saline for FEV1
No effects
Eng et al. [34]
10 ml of either normal or hypertonic saline; parallel groups
n = 58 (age 7–26 years) mean FEV1≈ 52%
Ad hoc VAS of perceived change
Significant differential improvement from baseline in FEV1 for hypertonic saline
An improvement, but group difference did not reach statistical significance
EXERCISE





Selvadurai et al. [36]
Comparison of aerobic/ resistance training and standard care; 3 parallel groups
n = 66 (age 8–16 years) mean FEV1 ≈ 57%
QWB
Aerobic training better for peak aerobic capacity. Resistance training better for weight gain, lung function and leg strength
Aerobic training associated with better QoL
Klijn et al. [37]
Anaerobic training vs normal daily activity; 2 parallel groups
n = 20 (age 9–18 years; mean 14 years) mean FEV1 = 75.2% (exercise group); 82.1% (control group)
Dutch CFQ
Anaerobic and aerobic performance improved in training group, but not control group
QoL improved in training group but not in control group
Orenstein et al. [38]
Aerobic versus upper-body strength training
n = 62 (age 8–18 years) Analysis on 53 cases of complete data
QWB
Strength and aerobic training may increase upper-body strength, and physical work capacity
No significant effects
PANCREATIC ENZYMES





Gan et al. [39]
4 versus 1 capsule daily crossover design
n = 13 (age 19–46 years; mean 28 years) mean BMI = 21
Symptoms and general well-being on 10-point scale
No difference between treatments
No significant changes in scores for well-being

FEV1 = forced expiratory volume in one second, expressed as percent predicted; FVC = forced vital capacity; QWB = Quality of Well-being Scale; CFQ = Cystic Fibrosis Questionnaire; BMI= body mass index, ≈ = approximately. QoL is secondary outcome measure unless otherwise indicated. All authors refer to QoL in the title, abstract or paper except [34] [39] who refer to well-being.

Abbott and Hart Health and Quality of Life Outcomes 2005 3:19   doi:10.1186/1477-7525-3-19