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

T C Edwards1, J Emerson2, A Genatossio2

  • 1Department of Health Services, University of Washington, 1208 NE 43rd St. - Campus Box 359455, Seattle, WA 98195-9455, United States.

Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
|January 24, 2018
PubMed

Insights

Observer-reported outcomes (ObsROs) are promising for young children with cystic fibrosis (CF). Parent-reported instruments showed good reliability for ages 0-6, suggesting potential for early intervention trials in pediatric CF patients.

Area of Science:

  • Pediatric clinical trial methodology
  • Respiratory medicine
  • Patient-reported outcomes research

Background:

  • Patient-reported outcomes (PROs) are crucial clinical trial endpoints.
  • Young children often cannot reliably self-report, necessitating observer-reported outcomes (ObsROs).
  • Cystic Fibrosis (CF) requires reliable outcome measures in pediatric populations.

Purpose of the Study:

  • To develop and pilot electronic parent-reported observational instruments for children with CF aged 0-6 and 7-11 years.
  • To assess the feasibility and preliminary psychometric properties of these instruments.
  • To inform the development of appropriate endpoints for early intervention trials in young children with CF.

Main Methods:

  • Concept elicitation interviews with parents of children with CF (≤11 years) to identify observable signs.
  • Instrument refinement based on parent and clinician feedback.
  • Pilot field testing to evaluate test-retest reliability and discriminative ability between well and sick states.

Main Results:

  • Developed a 17-item observational instrument assessing respiratory signs and CF impacts.
  • Achieved acceptable test-retest reliability for both age groups (0-6 and 7-11 years).
  • Observed low discriminative ability for the 7-11 year age group, potentially due to decreased parental observation.

Conclusions:

  • Parent-reported ObsROs show promise for children with CF aged 0-6 years.
  • Self-report may be more suitable for children with CF older than 6 years.
  • The 0-6 year instrument is a potential endpoint for early intervention clinical trials in pediatric CF, requiring further validation.
Abstract

Keywords:
ChildrenCystic fibrosisDiaryObservationOutcomeSigns

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