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

• Published in: BMC pediatrics 2015-02, Vol.15 (1), p.7-7, Article 7
• Main Authors: 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
• Format: Article
• Language: English
• Subjects: Case studies; Child; Child Health Services - standards; Chronic Disease - therapy; Database searching; Family; Humans; Internet/Web search services; Medical research; Medicine, Experimental; Online searching; Outcome Assessment (Health Care); Patient-Centered Care - standards; Physiological aspects
• ISSN: 1471-2431; 1471-2431
• DOI: 10.1186/s12887-015-0323-x

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. 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. 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. 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.