Core Outcome Measures in Effectiveness Trials

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

General Information

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.

Authors:
Khangura, S. D. Karaceper, M. D. Trakadis, Y. Mitchell, J. J. Chakraborty, P. Tingley, K. Coyle, D. Grosse, S. D. Kronick, J. B. Laberge, A. M. Little, J. Prasad, C. Sikora, L. Siriwardena, K. Sparkes, R. Speechley, K. N. Stockler, S. Wilson, B. J. Wilson, K. Zayed, R. Potter, B. K. Canadian Inherited Metabolic Diseases Research, Network

Publication

Journal:
BMC Pediatr
Volume:
15
Issue:
Pages:
7 -
Year:
2015
DOI:
Further Study Information

Date:
Funding source(s):

Health Area

Disease Category
Child health

Disease Name
Chronic pediatric disease

Target Population

Age Range
-

Sex
Unknown


Nature / type of Intervention
Unknown

Method(s)

Unknown

Stakeholders Involved

Unknown

Study Type

Systematic review of outcomes measured in trials

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