Scoping review of patient- and family-oriented outcomes and measures for chronic pediatric disease Academic Article uri icon

  • Overview
  • Research
  • Identity
  • Additional Document Info
  • View All


  • BACKGROUND: Improvements in health care for children with chronic diseases must be informed by research that emphasizes outcomes of importance to patients and families. To support a program of research in the field of rare inborn errors of metabolism (IEM), we conducted a broad scoping review of primary studies that: (i) focused on chronic pediatric diseases similar to IEM in etiology or manifestations and in complexity of management; (ii) reported patient- and/or family-oriented outcomes; and (iii) measured these outcomes using self-administered tools. METHODS: We developed a comprehensive review protocol and implemented an electronic search strategy to identify relevant citations in Medline, EMBASE, DARE and Cochrane. Two reviewers applied pre-specified criteria to titles/abstracts using a liberal accelerated approach. Articles eligible for full-text review were screened by two independent reviewers with discrepancies resolved by consensus. One researcher abstracted data on study characteristics, patient- and family-oriented outcomes, and self-administered measures. Data were validated by a second researcher. RESULTS: 4,118 citations were screened with 304 articles included. Across all included reports, the most-represented diseases were diabetes (35%), cerebral palsy (23%) and epilepsy (18%). We identified 43 unique patient- and family-oriented outcomes from among five emergent domains, with mental health outcomes appearing most frequently. The studies reported the use of 405 independent self-administered measures of these outcomes. CONCLUSIONS: Patient- and family-oriented research investigating chronic pediatric diseases emphasizes mental health and appears to be relatively well-developed in the diabetes literature. Future research can build on this foundation while identifying additional outcomes that are priorities for patients and families.


  • MacKenzie, Jennifer
  • Potter, Murray Alexander
  • Khangura, Sara D
  • Karaceper, Maria D
  • Trakadis, Yannis
  • Mitchell, John J
  • Chakraborty, Pranesh
  • Tingley, Kylie
  • Coyle, Doug
  • Grosse, Scott D
  • Kronick, Jonathan B
  • Laberge, Anne-Marie
  • Little, Julian
  • Prasad, Chitra
  • Sikora, Lindsey
  • Siriwardena, Komudi
  • Sparkes, Rebecca
  • Speechley, Kathy N
  • Stockler, Sylvia
  • Wilson, Brenda J
  • Wilson, Kumanan
  • Zayed, Reem
  • Potter, Beth K

publication date

  • December 2015