. 2015 Feb 13:15:7.

doi: 10.1186/s12887-015-0323-x.

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

Sara D Khangura¹, Maria D Karaceper², Yannis Trakadis³, John J Mitchell⁴, Pranesh Chakraborty^{5

6}, Kylie Tingley⁷, Doug Coyle⁸, Scott D Grosse⁹, Jonathan B Kronick^{10

11}, Anne-Marie Laberge¹², Julian Little¹³, Chitra Prasad¹⁴, Lindsey Sikora¹⁵, Komudi Siriwardena¹⁶, Rebecca Sparkes¹⁷, Kathy N Speechley¹⁸, Sylvia Stockler¹⁹, Brenda J Wilson²⁰, Kumanan Wilson²¹, Reem Zayed²², Beth K Potter²³; Canadian Inherited Metabolic Diseases Research Network

Collaborators, Affiliations

Collaborators

Canadian Inherited Metabolic Diseases Research Network:
Valerie Austin, Marni Brownwell, Catherine Brunel, Robin Casey, Alicia Chan, Maggie Chapman, Linda Dodds, Sarah Dyack, Annette Feigenbaum, Michael Geraghty, Alette Giezen, Jane Gillis, Cheryl Greenberg, Astrid Guttmann, Robin Hayeems, Shailly Jain, Aneal Khan, Erica Langley, Jennifer MacKenzie, Bruno Maranda, Aizeddin Mhanni, Fiona Miller, Grant Mitchell, Laura Nagy, Meranda Nakhla, Amy Pender, Murray Potter, Lesley Turner, Keiko Ueda, Clara VanKarnebeek, Hilary Vallance

Affiliations

¹ University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. khangura@uottawa.ca.
² University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. maria.karaceper@gmail.com.
³ Montreal Children's Hospital, McGill University Health Centre, 2300 Tupper Street, Montreal, QC, Canada. yannis.trakadis@mcgill.ca.
⁴ Montreal Children's Hospital, McGill University Health Centre, 2300 Tupper Street, Montreal, QC, Canada. john.mitchell@muhc.mcgill.ca.
⁵ University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. pchakraborty@cheo.on.ca.
⁶ Newborn Screening Ontario, Children's Hospital of Eastern Ontario, 415 Smyth Road, Ottawa, ON, Canada. pchakraborty@cheo.on.ca.
⁷ University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. kylietingley@gmail.com.
⁸ University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. doug.coyle@uottawa.ca.
⁹ National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, 1600 Clifton Road, Atlanta, GA, USA. sgg4@cdc.gov.
¹⁰ University of Toronto, 27 King's College Circle, Toronto, ON, Canada. jonathan.kronick@sickkids.ca.
¹¹ Hospital for Sick Children, 555 University Avenue, Toronto, ON, Canada. jonathan.kronick@sickkids.ca.
¹² Centre Hospitalier Universitaire Sainte-Justine, 3175 Chemin de la Côte-Sainte-Catherine, Montreal, QC, Canada. anne-marie.laberge.hsj@ssss.gouv.qc.ca.
¹³ University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. jlittle@uottawa.ca.
¹⁴ Western University, 1151 Richmond Street, London, ON, Canada. chitra.prasad@lhsc.on.ca.
¹⁵ University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. lindsey.sikora@uottawa.ca.
¹⁶ Hospital for Sick Children, 555 University Avenue, Toronto, ON, Canada. komudi.siriwardena@sickkids.ca.
¹⁷ Alberta Children's Hospital, 2888 Shaganappi Trail NW, Calgary, AB, Canada. rebecca.sparkes@albertahealthservices.ca.
¹⁸ Western University, 1151 Richmond Street, London, ON, Canada. kathy.speechley@lhsc.on.ca.
¹⁹ British Columbia Children's Hospital, 4480 Oak Street, Vancouver, BC, Canada. sstockler@cw.bc.ca.
²⁰ University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. bwilson@uottawa.ca.
²¹ Ottawa Hospital Research Institute, 725 Parkdale Avenue, Ottawa, ON, Canada. kwilson@ohri.ca.
²² University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. reemzayed@yahoo.com.
²³ University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. beth.potter@uottawa.ca.

PMID: 25886474
PMCID: PMC4334411
DOI: 10.1186/s12887-015-0323-x

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

Sara D Khangura et al. BMC Pediatr. 2015.

. 2015 Feb 13:15:7.

doi: 10.1186/s12887-015-0323-x.

Authors

Collaborators

Canadian Inherited Metabolic Diseases Research Network:
Valerie Austin, Marni Brownwell, Catherine Brunel, Robin Casey, Alicia Chan, Maggie Chapman, Linda Dodds, Sarah Dyack, Annette Feigenbaum, Michael Geraghty, Alette Giezen, Jane Gillis, Cheryl Greenberg, Astrid Guttmann, Robin Hayeems, Shailly Jain, Aneal Khan, Erica Langley, Jennifer MacKenzie, Bruno Maranda, Aizeddin Mhanni, Fiona Miller, Grant Mitchell, Laura Nagy, Meranda Nakhla, Amy Pender, Murray Potter, Lesley Turner, Keiko Ueda, Clara VanKarnebeek, Hilary Vallance

Affiliations

¹ University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. khangura@uottawa.ca.
² University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. maria.karaceper@gmail.com.
³ Montreal Children's Hospital, McGill University Health Centre, 2300 Tupper Street, Montreal, QC, Canada. yannis.trakadis@mcgill.ca.
⁴ Montreal Children's Hospital, McGill University Health Centre, 2300 Tupper Street, Montreal, QC, Canada. john.mitchell@muhc.mcgill.ca.
⁵ University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. pchakraborty@cheo.on.ca.
⁶ Newborn Screening Ontario, Children's Hospital of Eastern Ontario, 415 Smyth Road, Ottawa, ON, Canada. pchakraborty@cheo.on.ca.
⁷ University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. kylietingley@gmail.com.
⁸ University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. doug.coyle@uottawa.ca.
⁹ National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, 1600 Clifton Road, Atlanta, GA, USA. sgg4@cdc.gov.
¹⁰ University of Toronto, 27 King's College Circle, Toronto, ON, Canada. jonathan.kronick@sickkids.ca.
¹¹ Hospital for Sick Children, 555 University Avenue, Toronto, ON, Canada. jonathan.kronick@sickkids.ca.
¹² Centre Hospitalier Universitaire Sainte-Justine, 3175 Chemin de la Côte-Sainte-Catherine, Montreal, QC, Canada. anne-marie.laberge.hsj@ssss.gouv.qc.ca.
¹³ University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. jlittle@uottawa.ca.
¹⁴ Western University, 1151 Richmond Street, London, ON, Canada. chitra.prasad@lhsc.on.ca.
¹⁵ University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. lindsey.sikora@uottawa.ca.
¹⁶ Hospital for Sick Children, 555 University Avenue, Toronto, ON, Canada. komudi.siriwardena@sickkids.ca.
¹⁷ Alberta Children's Hospital, 2888 Shaganappi Trail NW, Calgary, AB, Canada. rebecca.sparkes@albertahealthservices.ca.
¹⁸ Western University, 1151 Richmond Street, London, ON, Canada. kathy.speechley@lhsc.on.ca.
¹⁹ British Columbia Children's Hospital, 4480 Oak Street, Vancouver, BC, Canada. sstockler@cw.bc.ca.
²⁰ University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. bwilson@uottawa.ca.
²¹ Ottawa Hospital Research Institute, 725 Parkdale Avenue, Ottawa, ON, Canada. kwilson@ohri.ca.
²² University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. reemzayed@yahoo.com.
²³ University of Ottawa, 451 Smyth Road, Ottawa, ON, Canada. beth.potter@uottawa.ca.

PMID: 25886474
PMCID: PMC4334411
DOI: 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.

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Figures

**Figure 1**
**PRISMA diagram for scoping review of patient- & family-oriented outcomes, measures for chronic, pediatric disease.**

See this image and copyright information in PMC

References

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

Collaborators

Affiliations

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

Authors

Collaborators

Affiliations

Abstract

Figures

References

Publication types

MeSH terms

Grants and funding

LinkOut - more resources

Full Text Sources

Other Literature Sources

Medical