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Edwards TC, Emerson J, Genatossio A, et al. Initial development and pilot testing of observer-reported outcomes (ObsROs) for children with cystic fibrosis ages 0-11years. J Cyst Fibros. 2018;17(5):680-686doi: 10.1016/j.jcf.2017.12.008.
Edwards, T. C., Emerson, J., Genatossio, A., McNamara, S., Goss, C., Patrick, D. L., Onchiri, F., & Rosenfeld, M. (2018). Initial development and pilot testing of observer-reported outcomes (ObsROs) for children with cystic fibrosis ages 0-11years. Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 17(5), 680-686. https://doi.org/10.1016/j.jcf.2017.12.008
Edwards, T C, et al. "Initial development and pilot testing of observer-reported outcomes (ObsROs) for children with cystic fibrosis ages 0-11years." Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society vol. 17,5 (2018): 680-686. doi: https://doi.org/10.1016/j.jcf.2017.12.008
Edwards TC, Emerson J, Genatossio A, McNamara S, Goss C, Patrick DL, Onchiri F, Rosenfeld M. Initial development and pilot testing of observer-reported outcomes (ObsROs) for children with cystic fibrosis ages 0-11years. J Cyst Fibros. 2018 Sep;17(5):680-686. doi: 10.1016/j.jcf.2017.12.008. Epub 2018 Feb 01. PMID: 29358075; PMCID: PMC6051933.
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J Cyst Fibros. 2018 Sep;17(5):680-686. doi: 10.1016/j.jcf.2017.12.008. Epub 2018 Feb 01.

Initial development and pilot testing of observer-reported outcomes (ObsROs) for children with cystic fibrosis ages 0-11years.

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society

T C Edwards, J Emerson, A Genatossio, S McNamara, C Goss, D L Patrick, F Onchiri, M Rosenfeld

Affiliations

  1. Department of Health Services, University of Washington, 1208 NE 43rd St. - Campus Box 359455, Seattle, WA 98195-9455, United States. Electronic address: [email protected].
  2. Division of Pulmonary Medicine, Seattle Children's Hospital, United States. Electronic address: [email protected].
  3. Division of Pulmonary Medicine, Seattle Children's Hospital, United States. Electronic address: [email protected].
  4. Division of Pulmonary Medicine, Seattle Children's Hospital, United States. Electronic address: [email protected].
  5. Department of Medicine and Pediatrics, University of Washington, Division of Pulmonary and Critical Care Medicine, United States. Electronic address: [email protected].
  6. Department of Health Services, University of Washington, 1208 NE 43rd St. - Campus Box 359455, Seattle, WA 98195-9455, United States. Electronic address: [email protected].
  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: [email protected].
  8. Department of Pediatrics, University of Washington, Division of Pulmonary Medicine, Seattle Children's Hospital, United States. Electronic address: [email protected].

PMID: 29358075 PMCID: PMC6051933 DOI: 10.1016/j.jcf.2017.12.008

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.

Copyright © 2018 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.

Keywords: Children; Cystic fibrosis; Diary; Observation; Outcome; Signs

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