Scoping review of patient- and family-oriented outcomes and measures for chronic pediatric disease

Authors

Sara D. Khangura, University of Ottawa
Maria D. Karaceper, University of Ottawa
Yannis Trakadis, Centre Universitaire de Santé McGill, Hôpital de Montreal Pour Enfants
John J. Mitchell, Centre Universitaire de Santé McGill, Hôpital de Montreal Pour Enfants
Pranesh Chakraborty, University of Ottawa
Kylie Tingley, University of Ottawa
Doug Coyle, University of Ottawa
Scott D. Grosse, Coordinating Center for Health Promotion
Jonathan B. Kronick, University of Toronto
Anne Marie Laberge, CHU Sainte-Justine - Le Centre Hospitalier Universitaire Mère-Enfant
Julian Little, University of Ottawa
Chitra Prasad, Western UniversityFollow
Lindsey Sikora, University of Ottawa
Komudi Siriwardena, Hospital for Sick Children University of Toronto
Rebecca Sparkes, Alberta Children's Hospital
Kathy N. Speechley, Western UniversityFollow
Sylvia Stockler, BC​ Children​'​​s Hospital
Brenda J. Wilson, University of Ottawa
Kumanan Wilson, Ottawa Hospital Research Institute
Reem Zayed, University of Ottawa
Beth K. Potter, University of Ottawa
Valerie Austin, Hospital for Sick Children University of Toronto
Annette Feigenbaum, Hospital for Sick Children University of Toronto
Laura Nagy, Hospital for Sick Children University of Toronto
Marni Brownwell, University of Manitoba
Catherine Brunel, CHU Sainte-Justine - Le Centre Hospitalier Universitaire Mère-Enfant

Document Type

Article

Publication Date

2-13-2015

Journal

BMC Pediatrics

Volume

15

Issue

1

URL with Digital Object Identifier

10.1186/s12887-015-0323-x

Abstract

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.