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Pulmonary exacerbations as indicators of progression of lung disease in young children with CF
  1. Don B Sanders1,
  2. Christopher Hooper Goss2,3
  1. 1 Department of Pediatrics, University of Wisconsin, Madison, Wisconsin, USA
  2. 2 Division of Pulmonary and Critical Care Medicine, Department of Medicine, University of Washington, Seattle, Washington, USA
  3. 3 Division of Pulmonary Medicine, Department of Pediatrics, Seattle Children's Hospital, Seattle, Washington, USA
  1. Correspondence to Dr Christopher Hooper Goss, Division of Pulmonary and Critical Care Medicine, Department of Medicine, University of Washington Medical Center, Campus Box 356522, 1959 NE Pacific, Seattle, WA 98195, USA; goss{at}

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The approval of ivacaftor by the European Medicines Agency (EMA) and the US Food and Drug Administration (US FDA) and the ongoing development of other drugs that target the underlying defect that causes cystic fibrosis (CF) have generated a great deal of excitement and hope for patients with CF.1 ,2 To truly maximise the potential benefits of these drugs, they will need to be administered before irreversible lung disease (eg, bronchiectasis) develops. Most patients with CF who have taken part in therapeutic drug trials have been at least 6 years of age, when most patients begin to be able to perform spirometry, the most commonly used endpoint in CF therapeutic trials. However, an observational study in Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST CF) demonstrated that structural lung disease, including bronchiectasis, may be present even in infancy.3 Thus, to minimise the progression of lung disease, we must initiate treatment as early as safely possible for those patients who are at risk for developing lung disease. The challenge in obtaining EMA or FDA approval for new therapeutic interventions to be administered in early life, where the progression of lung disease occurs in a ‘black box’, is demonstrating safety and efficacy in children too young to perform traditional spirometry. Given that pulmonary exacerbations occur frequently even in young children, they offer an inviting clinical endpoint for future studies in this age group. The FDA defines clinical endpoints as direct measures of how a patient feels, functions or survives.4 Pulmonary exacerbations are …

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  • Contributors Both authors were involved in drafting and reviewing the manuscript for important intellectual content.

  • Competing interests CHG receives funding from the US Cystic Fibrosis Foundation, the NIH (R01HL103965, R01 AI101307, P30 DK089507) and the FDA (R01 FD003704). CHG has participated on advisory board and received an unrestricted grant from Transave Inc; participated on an advisory board for KaloBios Pharm; and received an unrestricted grant from Vertex Pharmaceuticals to perform secondary data analyses. DBS has received funding from the US Cystic Fibrosis Foundation and the NIH.

  • Provenance and peer review Commissioned; internally peer reviewed.

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