title: Early intervention studies in infants and preschool children with cystic fibrosis: are we ready?
abstract: Cystic fibrosis (CF) lung disease starts early in life and progresses even in the absence of clinical symptoms. Therefore, sensitive outcome measures to quantify and track these early abnormalities in infants and young children are needed; both for clinical care and interventional trials. Currently, the efficacy of most therapeutic interventions in CF has not been tested in children under the age of 6 years and drug development programmes have focused on assessing safety rather than efficacy in this age group. This article summarises the current status for outcome measures that can be utilised in clinical trials in infants and children with CF. Two methodologies are specifically highlighted in this review; chest computed tomography to assess structural damage of the lung and multiple breath washout as a technique to quantify ventilation inhomogeneity. While not all questions regarding the utility of these outcome measures in infants and young children have been resolved, significant advances have been made and it now appears feasible to design and conduct adequately powered efficacy studies in this age group. This could be a crucial step to further improve outcomes in CF patients as initiating effective treatment early is considered essential to prevent permanent lung damage.
authors.affiliation: Princess Margaret Hospital for Children and University of Western Australia, Perth, Western Australia 6005, Australia. Stephen.Stick@health.wa.gov
authors.forename: Stephen
authors.initials: S
authors.lastname: Stick
authors.forename: Harm
authors.initials: H
authors.lastname: Tiddens
authors.forename: Paul
authors.initials: P
authors.lastname: Aurora
authors.forename: Per
authors.initials: P
authors.lastname: Gustafsson
authors.forename: Sarath
authors.initials: S
authors.lastname: Ranganathan
authors.forename: Paul
authors.initials: P
authors.lastname: Robinson
authors.forename: Margaret
authors.initials: M
authors.lastname: Rosenfeld
authors.forename: Peter
authors.initials: P
authors.lastname: Sly
authors.forename: Felix
authors.initials: F
authors.lastname: Ratjen
mesh_terms: D018893:Bronchoalveolar Lavage; D002675:Child, Preschool; D003550:Cystic Fibrosis / Q000628:therapy*; D006801:Humans; D007223:Infant; D007249:Inflammation; D008168:Lung / Q000502:physiology; D008171:Lung Diseases / Q000628:therapy; D012119:Respiration; D012129:Respiratory Function Tests; D013147:Spirometry; D014057:Tomography, X-Ray Computed; D016896:Treatment Outcome