@article{92ffe4bd31074d08ba6f5adf071b4167,
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.",
keywords = "Assessment, Family-centered care, Inborn errors, Metabolism, Outcome measures, Outcomes research, Patient outcomes, Patient-centered care, Patient-centered outcomes research, Rare diseases",
author = "{On behalf of the Canadian Inherited Metabolic Diseases Research Network (CIMDRN)} and Khangura, {Sara D.} and Karaceper, {Maria D.} and Yannis Trakadis and Mitchell, {John J.} and Pranesh Chakraborty and Kylie Tingley and Doug Coyle and Grosse, {Scott D.} and Kronick, {Jonathan B.} and Laberge, {Anne Marie} and Julian Little and Chitra Prasad and Lindsey Sikora and Komudi Siriwardena and Rebecca Sparkes and Speechley, {Kathy N.} and Sylvia Stockler and Wilson, {Brenda J.} and Kumanan Wilson and Reem Zayed and Potter, {Beth K.} and Valerie Austin and Annette Feigenbaum and Laura Nagy and Marni Brownwell and Catherine Brunel and Robin Casey and Alicia Chan and Shailly Jain and Maggie Chapman and Sarah Dyack and Jane Gillis and Linda Dodds and Deshayne Fell and Michael Geraghty and Erica Langley and Alette Giezen and Keiko Ueda and Hilary Vallance and Cheryl Greenberg and Astrid Guttmann and Robin Hayeems and Fiona Miller and Aneal Khan and Jennifer MacKenzie and Bruno Maranda and Aizeddin Mhanni and Grant Mitchell and Meranda Nakhla and {van Karnebeek}, Clara",
note = "Funding Information: All authors declare that they have no non-financial competing interests. John J. Mitchell has received travel grants from BioMarin and consulting fees from BioMarin and Genzyme both of which are unrelated to this study; Komudi Siriwardena has funds from BioMarin Pharmaceuticals for 2 drug-studies (PKU-015 and PKU-016) and 1 investigator initiated study, both of which are unrelated to this study. All other authors declare that they have no financial competing interests. Funding Information: Thanks go to Ms. Joan Peterson for her assistance with data acquisition. All phases of this study were supported by a Canadian Institutes of Health Research (CIHR) grant, TR3-119195. The sponsor has had no role in the study design; the collection, analysis, and interpretation of data; the writing of the report; nor the decision to submit the paper for publication. Sara D. Khangura produced the first draft of the manuscript and is employed by the University of Ottawa under the auspices of this study{\textquoteright}s CIHR funding. The Canadian Inherited Metabolic Diseases Research Network (CIMDRN) is a pan-Canadian group of clinicians and scientists including the following investigators (in addition to those authors listed on this report): Valerie Austin (Hospital for Sick Children), Dr. Marni Brownwell (University of Manitoba), Dr. Catherine Brunel (Centre hospitalier universitaire Sainte-Justine), Dr. Robin Casey, Dr. Alicia Chan (University of Alberta Hospital), Maggie Chapman (IWK Health Centre), Dr. Linda Dodds (Dalhousie University), Dr. Sarah Dyack (IWK Health Centre), Dr. Annette Feigenbaum (Hospital for Sick Children), Dr. Deshayne Fell (Ottawa Hospital Research Institute), Dr. Michael Geraghty (Children{\textquoteright}s Hospital of Eastern Ontario), Alette Giezen (British Columbia Children{\textquoteright}s Hospital), Dr. Jane Gillis (IWK Health Centre), Dr. Cheryl Greenberg (Winnipeg Children{\textquoteright}s Hospital), Dr. Astrid Guttmann (Institute for Clinical Evaluative Sciences), Dr. Robin Hayeems (University of Toronto), Dr. Shailly Jain (University of Alberta Hospital), Dr. Aneal Khan (Alberta Children{\textquoteright}s Hospital), Erica Langley (Children{\textquoteright}s Hospital of Eastern Ontario), Dr. Jennifer MacKenzie (Kingston General Hospital), Dr. Bruno Maranda (Universit{\'e} de Sherbrooke), Dr. Aizeddin Mhanni (Health Sciences Centre Winnipeg), Dr. Fiona Miller (University of Toronto), Dr. Grant Mitchell (Le centre hospitalier universitaire m{\`e}re-enfant), Laura Nagy (Hospital for Sick Children), Dr. Meranda Nakhla (McGill University), Amy Pender (Hamilton Health Sciences), Dr. Murray Potter (Hamilton Health Sciences), Dr. Lesley Turner (Memorial University of Newfoundland), Keiko Ueda (British Columbia Children{\textquoteright}s Hospital), Dr. Clara VanKarnebeek (University of British Columbia) and Dr. Hilary Vallance (British Columbia Children{\textquoteright}s Hospital). Publisher Copyright: {\textcopyright} 2015 Khangura et al.; licensee BioMed Central.",
year = "2015",
month = feb,
day = "13",
doi = "https://doi.org/10.1186/s12887-015-0323-x",
language = "English",
volume = "15",
journal = "BMC Pediatrics",
issn = "1471-2431",
publisher = "BioMed Central",
number = "1",
}