Randomized controlled trials in cystic fibrosis (1966-1997) categorized bytime, design, and intervention

Citation
K. Cheng et al., Randomized controlled trials in cystic fibrosis (1966-1997) categorized bytime, design, and intervention, PEDIAT PULM, 29(1), 2000, pp. 1-7
Citations number
24
Categorie Soggetti
Pediatrics
Journal title
PEDIATRIC PULMONOLOGY
ISSN journal
87556863 → ACNP
Volume
29
Issue
1
Year of publication
2000
Pages
1 - 7
Database
ISI
SICI code
8755-6863(200001)29:1<1:RCTICF>2.0.ZU;2-3
Abstract
The improved prognosis of cystic fibrosis (CF) over the last three decades has been associated with an increased use of a range of treatments, but it is important that the use of an individual treatment is based on evidence. Well-designed randomized controlled trials (RCTs) are a robust method for e valuating the effectiveness of such treatments. We have developed a registe r of RCTs in CF and have studied when they were performed, their design, an d what interventions were investigated. We identified 506 RCTs; 37.5% were identified solely as abstract reports in conference proceedings. There has been about a 30-fold increase in the num ber of RCTs in CF since 1966. A high proportion of the RCTs (72.7%) had a s ample size of 30 or less, and only 8.7% were multicenter trials. Reporting of study design was poor: in 51.4% the report did not state whether there w as any blinding in the trial design; 53.6% of studies were of crossover des ign. The most common interventions studied were antibiotic treatments and p hysiotherapy, but a number of commonly used therapies had been evaluated on ly in a small number of patients. Although the number of RCTs of interventi ons in CF patients has increased over the last 25 years, the sample sizes o f these trials are generally too small to indicate whether the intervention was effective, and very few were multicenter. Future RCTs in CF are more l ikely to provide clinically useful answers if higher numbers of patients ar e recruited into large, well-designed multicenter trials. This should be a priority of the organization of future research in CF. (C) 2000 Wiley-Liss, Inc.