Stephanie D Davis1, Felix Ratjen2, Lyndia C Brumback3, Robin C Johnson4, Amy G Filbrun5, Gwendolyn S Kerby6, Howard B Panitch7, Scott H Donaldson8, Margaret Rosenfeld9. 1. Section of Pediatric Pulmonology, Allergy and Sleep Medicine, Department of Pediatrics, Riley Hospital for Children, Indiana University School of Medicine, Indianapolis, IN, USA. 2. Division of Respiratory Medicine, Department of Pediatrics, the Hospital for Sick Children, University of Toronto, Toronto, ON, Canada. 3. Department of Biostatistics, University of Washington, Seattle, WA, USA. 4. Division of Pediatric Pulmonology, Department of Pediatrics, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA. 5. Pediatric Pulmonary Division, Department of Pediatrics, CS Mott Children's Hospital, University of Michigan School of Medicine, Ann Arbor, MI, USA. 6. Department of Pediatrics, The Breathing Institute, University of Colorado and Children's Hospital, Aurora, CO, USA. 7. Division of Pulmonary Medicine, Department of Pediatrics, The Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. 8. Division of Pulmonary and Critical Care, Department of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA. 9. Division of Pulmonary Medicine, Seattle Children's Hospital and Department of Pediatrics, University of Washington School of Medicine, Seattle, WA, USA.
Abstract
BACKGROUND: The Infant Study of Inhaled Saline (ISIS) in CF was the first multicenter clinical trial to utilize infant pulmonary function tests (iPFTs) as an endpoint. METHODS: Secondary analysis of ISIS data was conducted in order to assess feasibility of iPFT measures and their associations with respiratory symptoms. Standard deviations were calculated to aid in power calculations for future clinical trials. RESULTS:Seventy-three participants enrolled, 70 returned for the final visit; 62 (89%) and 45 (64%) had acceptable paired functional residual capacity (FRC) and raised volume measurements, respectively. Mean baselineFEV0.5, FEF75 and FRC z-scores were 0.3 (SD: 1.2), -0.2 (SD: 2.0), and 1.8 (SD: 2.0). CONCLUSIONS: iPFTs are not appropriate primary endpoints for multicenter clinical trials due to challenges of obtaining acceptable data and near-normal average raised volume measurements. Raised volume measures have potential to serve as secondary endpoints in future clinical CF trials.
RCT Entities:
BACKGROUND: The Infant Study of Inhaled Saline (ISIS) in CF was the first multicenter clinical trial to utilize infant pulmonary function tests (iPFTs) as an endpoint. METHODS: Secondary analysis of ISIS data was conducted in order to assess feasibility of iPFT measures and their associations with respiratory symptoms. Standard deviations were calculated to aid in power calculations for future clinical trials. RESULTS: Seventy-three participants enrolled, 70 returned for the final visit; 62 (89%) and 45 (64%) had acceptable paired functional residual capacity (FRC) and raised volume measurements, respectively. Mean baseline FEV0.5, FEF75 and FRC z-scores were 0.3 (SD: 1.2), -0.2 (SD: 2.0), and 1.8 (SD: 2.0). CONCLUSIONS:iPFTs are not appropriate primary endpoints for multicenter clinical trials due to challenges of obtaining acceptable data and near-normal average raised volume measurements. Raised volume measures have potential to serve as secondary endpoints in future clinical CF trials.
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