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Randomised controlled trials in cystic fibrosis: what, when and how?

2011; European Respiratory Society; Volume: 37; Issue: 5 Linguagem: Inglês

10.1183/09031936.00166810

1399-3003

Peter D. Sly, Robert S. Ware, Nicholas de Klerk, Stephen M. Stick,

Tracheal and airway disorders

The major morbidity and mortality from cystic fibrosis (CF) comes from progressive lung disease with bronchiectasis leading to respiratory failure. There is no doubt that survival of CF patients has increased progressively through the latter part of the 20th and the early part of the 21st centuries, and that the quality of life for patients with CF has increased substantially. However, CF still remains a serious life-shortening disease. Indeed, the most recent data from national CF registries show that the slope of survival curves 1 and the rate of loss of lung function have not changed at all, at least beyond childhood. Potential reasons for this are that CF lung disease begins in early life and that while current treatments may increase the health of children, they do little, if anything, to prevent the onset or progression of destructive lung disease, nor the inevitable onset of chronic respiratory failure. Data from a number of sources have demonstrated that CF lung disease begins very early in life 2–6. Arguably, the best data come from the Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST CF), where essentially all children diagnosed with CF in a geographically defined region of Victoria and in Western Australia following detection by newborn screening are assessed soon after diagnosis and annually until 6–7 yrs of age 7. These data show that a substantial proportion of infants have evidence of pulmonary inflammation and infection at 3 months of age, even in the absence of clinically apparent lung disease 8. Structural lung disease, including bronchiectasis, is prevalent in infants and young children with CF 8–10; the major risk factors being neutrophilic inflammation, the presence of free neutrophil elastase activity in the lungs and pulmonary infection, especially with Pseudomonas aeruginosa 8, …