T C Edwards1, J Emerson2, A Genatossio3, S McNamara4, C Goss5, D L Patrick6, F Onchiri7, M Rosenfeld8. 1. Department of Health Services, University of Washington, 1208 NE 43rd St. - Campus Box 359455, Seattle, WA 98195-9455, United States. Electronic address: toddce@uw.edu. 2. Division of Pulmonary Medicine, Seattle Children's Hospital, United States. Electronic address: julia.emerson@seattlechildrens.org. 3. Division of Pulmonary Medicine, Seattle Children's Hospital, United States. Electronic address: alan.genatossio@seattlechildrens.org. 4. Division of Pulmonary Medicine, Seattle Children's Hospital, United States. Electronic address: sharon.mcnamara@seattlechildrens.org. 5. Department of Medicine and Pediatrics, University of Washington, Division of Pulmonary and Critical Care Medicine, United States. Electronic address: goss@uw.edu. 6. Department of Health Services, University of Washington, 1208 NE 43rd St. - Campus Box 359455, Seattle, WA 98195-9455, United States. Electronic address: donald@uw.edu. 7. Center for Biomedical Statistics, Seattle Children's Research Institute, Seattle, WA, United States; Center for Clinical and Translational Research, Seattle Children's Research Institute, Seattle, WA, United States. Electronic address: Frankline.onchiri@seattlechildrens.org. 8. Department of Pediatrics, University of Washington, Division of Pulmonary Medicine, Seattle Children's Hospital, United States. Electronic address: mrosen@uw.edu.
Abstract
PURPOSE: Patient-reported outcomes are important clinical trial endpoints. Young children may not be able to reliably report on how they feel or function, so observer-reported outcomes (ObsROs) may be more appropriate for them. The purpose of this study was to develop and pilot field test electronic parent-reported observational instruments for children with cystic fibrosis (CF) 0-6 and 7-11years of age. METHODS: We performed concept elicitation interviews with parents of children with CF ≤11years of age to elicit the respiratory signs they could observe at baseline and during an acute respiratory illness. The resulting instruments were refined based on interviews with parents and clinicians. We conducted a pilot field test to evaluate test-retest reliability and the ability of items to distinguish well and sick periods. RESULTS: The instruments consist of 17 items assessing respiratory signs and observable CF-related impacts. Test-retest reliability was acceptable for both age groups but discrimination was low for ages 7-11, likely reflecting less direct observation of older children by their parents. CONCLUSIONS: An ObsRO for children with CF ages 0-6 appears promising, while self-report may be more appropriate for children >6years of age. Next steps for the 0-6year old instrument will be utilizing it as an exploratory endpoint in clinical trials to enable item reduction, scale development, and further reliability and validity testing. Ultimately, this ObsRO could be a promising endpoint for early intervention trials in young children with CF.
PURPOSE:Patient-reported outcomes are important clinical trial endpoints. Young children may not be able to reliably report on how they feel or function, so observer-reported outcomes (ObsROs) may be more appropriate for them. The purpose of this study was to develop and pilot field test electronic parent-reported observational instruments for children with cystic fibrosis (CF) 0-6 and 7-11years of age. METHODS: We performed concept elicitation interviews with parents of children with CF ≤11years of age to elicit the respiratory signs they could observe at baseline and during an acute respiratory illness. The resulting instruments were refined based on interviews with parents and clinicians. We conducted a pilot field test to evaluate test-retest reliability and the ability of items to distinguish well and sick periods. RESULTS: The instruments consist of 17 items assessing respiratory signs and observable CF-related impacts. Test-retest reliability was acceptable for both age groups but discrimination was low for ages 7-11, likely reflecting less direct observation of older children by their parents. CONCLUSIONS: An ObsRO for children with CF ages 0-6 appears promising, while self-report may be more appropriate for children >6years of age. Next steps for the 0-6year old instrument will be utilizing it as an exploratory endpoint in clinical trials to enable item reduction, scale development, and further reliability and validity testing. Ultimately, this ObsRO could be a promising endpoint for early intervention trials in young children with CF.
Authors: Antonia V Bennett; Donald L Patrick; James F Lymp; Todd C Edwards; Christopher H Goss Journal: J Cyst Fibros Date: 2010-12 Impact factor: 5.482
Authors: Margaret Rosenfeld; Felix Ratjen; Lyndia Brumback; Stephen Daniel; Ron Rowbotham; Sharon McNamara; Robin Johnson; Richard Kronmal; Stephanie D Davis Journal: JAMA Date: 2012-06-06 Impact factor: 56.272
Authors: Bradley S Quon; Donald L Patrick; Todd C Edwards; Moira L Aitken; Ronald L Gibson; Alan Genatossio; Sharon McNamara; Christopher H Goss Journal: J Cyst Fibros Date: 2012-01-04 Impact factor: 5.482
Authors: Louis S Matza; Anne M Rentz; Kristina Secnik; Andrine R Swensen; Dennis A Revicki; David Michelson; Thomas Spencer; Jeffrey H Newcorn; Christopher J Kratochvil Journal: J Dev Behav Pediatr Date: 2004-06 Impact factor: 2.225
Authors: Todd C Edwards; Carrie L Heike; Kathleen A Kapp-Simon; Salene M Jones; Brian G Leroux; Laura P Stueckle; Claudia Crilly Bellucci; Janine M Rosenberg; Meredith Albert; Cassandra L Aspinall; Donald L Patrick Journal: Cleft Palate Craniofac J Date: 2021-09-13
Authors: Salene M W Jones; Todd C Edwards; Brian G Leroux; Kathleen A Kapp-Simon; Donald L Patrick; Laura P Stueckle; Janine M Rosenberg; Meredith Albert; Claudia Crilly Bellucci; Cassandra L Aspinall; Katherine Vick; Carrie L Heike Journal: Cleft Palate Craniofac J Date: 2022-03-28