Table 2 Summary of RCTs in CF measuring QoL: mucolytic therapies, exercise and pancreatic enzymes

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