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

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• 作者：Sara D Khangura (1)
  Maria D Karaceper (1)
  Yannis Trakadis (2)
  John J Mitchell (2)
  Pranesh Chakraborty (1) (3)
  Kylie Tingley (1)
  Doug Coyle (1)
  Scott D Grosse (4)
  Jonathan B Kronick (5) (6)
  Anne-Marie Laberge (7)
  Julian Little (1)
  Chitra Prasad (8)
  Lindsey Sikora (1)
  Komudi Siriwardena (6)
  Rebecca Sparkes (9)
  Kathy N Speechley (8)
  Sylvia Stockler (10)
  Brenda J Wilson (1)
  Kumanan Wilson (11)
  Reem Zayed (1)
  Beth K Potter (1)
  on behalf of the Canadian Inherited Metabolic Diseases Research Network (CIMDRN)
  
  1. University of Ottawa ; 451 Smyth Road ; Ottawa ; ON ; Canada
  2. Montreal Children鈥檚 Hospital ; McGill University Health Centre ; 2300 Tupper Street ; Montreal ; QC ; Canada
  3. Newborn Screening Ontario ; Children鈥檚 Hospital of Eastern Ontario ; 415 Smyth Road ; Ottawa ; ON ; Canada
  4. National Center on Birth Defects and Developmental Disabilities ; Centers for Disease Control and Prevention ; 1600 Clifton Road ; Atlanta ; GA ; USA
  5. University of Toronto ; 27 King鈥檚 College Circle ; Toronto ; ON ; Canada
  6. Hospital for Sick Children ; 555 University Avenue ; Toronto ; ON ; Canada
  7. Centre Hospitalier Universitaire Sainte-Justine ; 3175 Chemin de la C么te-Sainte-Catherine ; Montreal ; QC ; Canada
  8. Western University ; 1151 Richmond Street ; London ; ON ; Canada
  9. Alberta Children鈥檚 Hospital ; 2888 Shaganappi Trail NW ; Calgary ; AB ; Canada
  10. British Columbia Children鈥檚 Hospital ; 4480 Oak Street ; Vancouver ; BC ; Canada
  11. Ottawa Hospital Research Institute ; 725 Parkdale Avenue ; Ottawa ; ON ; Canada
  
• 关键词：Patient ; centered outcomes research ; Patient ; centered care ; Outcomes research ; Outcome measures ; Metabolism ; Inborn errors ; Assessment ; Patient outcomes ; Rare diseases ; Family ; centered care
• 刊名：BMC Pediatrics
• 出版年：2015
• 出版时间：December 2015
• 年：2015
• 卷：15
• 期：1
• 全文大小：805 KB
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• 刊物主题：Pediatrics; Internal Medicine;
• 出版者：BioMed Central
• ISSN：1471-2431

文摘

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.