title: Scoping review of patient- and family-oriented outcomes and measures for chronic pediatric disease.
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.affiliation: University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. khangura@uottawa.ca.
authors.forename: Sara D
authors.initials: SD
authors.lastname: Khangura
authors.affiliation: University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. maria.karaceper@gmail.com.
authors.forename: Maria D
authors.initials: MD
authors.lastname: Karaceper
authors.affiliation: Montreal Children's Hospital, McGill University Health Centre, 2300 Tupper Street, Montreal, QC, Canada. yannis.trakadis@mcgill.ca.
authors.forename: Yannis
authors.initials: Y
authors.lastname: Trakadis
authors.affiliation: Montreal Children's Hospital, McGill University Health Centre, 2300 Tupper Street, Montreal, QC, Canada. john.mitchell@muhc.mcgill.ca.
authors.forename: John J
authors.initials: JJ
authors.lastname: Mitchell
authors.affiliation: University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. pchakraborty@cheo.on.ca.|Newborn Screening Ontario, Children's Hospital of Eastern Ontario, 415 Smyth Road, Ottawa, ON, Canada. pchakraborty@cheo.on.ca.
authors.forename: Pranesh
authors.initials: P
authors.lastname: Chakraborty
authors.affiliation: University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. kylietingley@gmail.com.
authors.forename: Kylie
authors.initials: K
authors.lastname: Tingley
authors.affiliation: University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. doug.coyle@uottawa.ca.
authors.forename: Doug
authors.initials: D
authors.lastname: Coyle
authors.affiliation: National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, 1600 Clifton Road, Atlanta, GA, USA. sgg4@cdc.gov.
authors.forename: Scott D
authors.initials: SD
authors.lastname: Grosse
authors.affiliation: University of Toronto, 27 King's College Circle, Toronto, ON, Canada. jonathan.kronick@sickkids.ca.|Hospital for Sick Children, 555 University Avenue, Toronto, ON, Canada. jonathan.kronick@sickkids.ca.
authors.forename: Jonathan B
authors.initials: JB
authors.lastname: Kronick
authors.affiliation: Centre Hospitalier Universitaire Sainte-Justine, 3175 Chemin de la Côte-Sainte-Catherine, Montreal, QC, Canada. anne-marie.laberge.hsj@ssss.gouv.qc.ca.
authors.forename: Anne-Marie
authors.initials: AM
authors.lastname: Laberge
authors.affiliation: University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. jlittle@uottawa.ca.
authors.forename: Julian
authors.initials: J
authors.lastname: Little
authors.affiliation: Western University, 1151 Richmond Street, London, ON, Canada. chitra.prasad@lhsc.on.ca.
authors.forename: Chitra
authors.initials: C
authors.lastname: Prasad
authors.affiliation: University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. lindsey.sikora@uottawa.ca.
authors.forename: Lindsey
authors.initials: L
authors.lastname: Sikora
authors.affiliation: Hospital for Sick Children, 555 University Avenue, Toronto, ON, Canada. komudi.siriwardena@sickkids.ca.
authors.forename: Komudi
authors.initials: K
authors.lastname: Siriwardena
authors.affiliation: Alberta Children's Hospital, 2888 Shaganappi Trail NW, Calgary, AB, Canada. rebecca.sparkes@albertahealthservices.ca.
authors.forename: Rebecca
authors.initials: R
authors.lastname: Sparkes
authors.affiliation: Western University, 1151 Richmond Street, London, ON, Canada. kathy.speechley@lhsc.on.ca.
authors.forename: Kathy N
authors.initials: KN
authors.lastname: Speechley
authors.affiliation: British Columbia Children's Hospital, 4480 Oak Street, Vancouver, BC, Canada. sstockler@cw.bc.ca.
authors.forename: Sylvia
authors.initials: S
authors.lastname: Stockler
authors.affiliation: University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. bwilson@uottawa.ca.
authors.forename: Brenda J
authors.initials: BJ
authors.lastname: Wilson
authors.affiliation: Ottawa Hospital Research Institute, 725 Parkdale Avenue, Ottawa, ON, Canada. kwilson@ohri.ca.
authors.forename: Kumanan
authors.initials: K
authors.lastname: Wilson
authors.affiliation: University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. reemzayed@yahoo.com.
authors.forename: Reem
authors.initials: R
authors.lastname: Zayed
authors.affiliation: University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. beth.potter@uottawa.ca.
authors.forename: Beth K
authors.initials: BK
authors.lastname: Potter
mesh_terms: D002648:Child; D002662:Child Health Services / Q000592:standards*; D002908:Chronic Disease / Q000628:therapy*; D005190:Family; D006801:Humans; D017063:Outcome Assessment, Health Care; D018802:Patient-Centered Care / Q000592:standards*