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. Author manuscript; available in PMC: 2010 Sep 16.
Published in final edited form as: Paediatr Drugs. 2009;11(2):101–113. doi: 10.2165/00148581-200911020-00002

Table I.

Clinical trials of inhaled corticosteroids (ICS) in cystic fibrosis (CF)

Study (year) Design No. of patients Mean or median age in years (range) Drug/total daily dose Treatment duration Efficacy for primary outcome Lung function (when reported) Adverse effects
Schiotz et al.[49] (1983) R, DB, PC, matched pairs 26 14 (4–29) BEC 400 μg 16 wk No effect on sputum inflammatory markers FVC (mean, start of study → after treatment): PL 98% → 78%, BEC 89% → 91%, overall 90% → 85% No statistical tests reported None reported
Van Haren et al.[50] (1995) DB, PC, CO 12 26 (16–45) BUD 1600 μg 6 wk Decreased airway reactivity in 58% (≥1 doubling dose increase in PC20 histamine). Small improvement in cough and dyspnea scores No effect FEV1 (after treatment): PL 2.2 L, BUD 2.3 L, FVC: PL 3.3 L, BUD 3.6 L None reported
Nikolaizik and Schoni[51] (1996) R 49 19.8, SD 6.8 BEC 1500 μg 30 d Positive effect on thoracic gas volume (p = 0.012). ICS had no effect on other measures No effect FVC (mean, start of study → after treatment): no ICS 58% → 65%, ICS 55% → 65%, FEV1 (mean, start of study → after treatment): no ICS 40% → 48%, ICS 37% → 45% No mention of AE reporting in paper
Balfour-Lynn et al.[52] (1997) R, DB, PC, CO 23 10.3 (7–17) FLU 400 μg 6 wk No improvement in sputum inflammatory markers No effect FVC: baseline 75%, PL 78%, FLU 78%, FEV1: baseline 64 %, PL 67%, FLU 65% Reported AEs: PL 15 (65%), FLU 18 (78%). Most URI/CF pulmonary exacerbations. Two possibly drug related: transient cough, rash
Bisgaard et al.[53] (1997) R, DB, parallel 55 20 (not given) BUD 1600 μg 24 wk Trend toward reduced decline in FEV1 Intention to treat ΔFEV1: PL −0.187 L, BUD −0.032 L(p = 0.08) PL 13, BUD 17 (overall incidences comparable)
Dauletbaev et al.[54] (1999) R, open label, parallel 26 26 (16–38) FLU 1000 μg 3 wk No improvement in clinical symptom score, lung function or sputum inflammatory markers FEV1 (mean, start of study → after treatment): PL 57 → 56% p > 0.2, FLU 56 → 53% p > 0.1 No clinical AEs reported. Sputum superoxide anion release was higher in FLU group (34 vs 25 nmol/h/106 cells p = 0.02)
Wojtczak et al.[55] (2001) Uncontrolled, prospective 12 6 (1.5–13) BEC 420 μg 8 wk Decreased BALF neutrophils (to 1/3 pretreatment levels, p = 0.03) Not done as outcome measure None reported. No increase in airway infection. No new Pseudomonas infections
Balfour-Lynn et al.[56] (2006) R, DB, PC, MC, withdrawal trial 171 14.6 (6–48) [FLU] 15.8 (6–53) [PL] FLU varied doses 6 mo No difference in time to first exacerbation, HR 1.07 (0.68–1.7) FVC (mean, start of study → after treatment): PL 90% → 90%, FLU 90% → 90%, FEV1 (mean, start of study → after treatment): PL 74% → 73%, FLU 76% → 76% 24 withdrawals from each group. 136 AE in FLU group (3 SAE: head injury, gallstones, intra-abdominal sepsis). 161 AE in PL group (0 SAE)
De Boeck et al.[57] (2007) R, DB, PC, MC 27 8.2 (FLU) 9 (PL) FLU 1000 μg 12 mo No difference in FEV1 ΔFEV1 (prebronchodilator): PL −3.6%, FLU +3.9%, ΔFEV1 (post-bronchodilator): PL −3.9%, FLU +3.3% Growth suppression: height gain PL 5.49 cm, height gain FLU 3.96 cm (p = 0.005). No increase in Pseudomonas infection or resp infections
Ren et al.[58] (2008) Observational 2565 6–17 Varied 2 y Decreased rate of decline of FEV1 in patients regularly using ICS (p = 0.04) Rate of change of FEV1 (% pred/y) after ICS: patients using ICS −0.44%/y, comparison group not using ICS −1.44%/y Decreased WFA and HFA z-scores, increased use of insulin or oral hypoglycemics, and increased rates of Stenotrophomonas maltophilia, Burkholderia cepacia, and Aspergillus spp. in ICS users

AE= adverse event; BALF = bronchoalveolar lavage fluid; BEC= beclomethasone; BUD= budesonide; CO= crossover; DB= double-blind; FEV1 = forced expiratory volume in 1 second; FLU = fluticasone propionate; FVC= forced vital capacity; HFA= height for age; HR= hazard ratio; MC= multicenter; NS= not significant; PC= placebo-controlled; PC20 = concentration provoking a 20% decline in FEV1; PL = placebo; pred = prednisone; R= randomized; resp = respiratory; SAE= serious AE; URI = upper respiratory tract infection; WFA=weight for age; Δ indicates change; → indicates after intervention/treatment.