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BMC Pediatrics
Published in: BMC Pediatrics 1/2015

Open Access 01-12-2015 | Research article

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

Authors: Sara D Khangura, Maria D Karaceper, Yannis Trakadis, John J Mitchell, Pranesh Chakraborty, Kylie Tingley, Doug Coyle, Scott D Grosse, Jonathan B Kronick, Anne-Marie Laberge, Julian Little, Chitra Prasad, Lindsey Sikora, Komudi Siriwardena, Rebecca Sparkes, Kathy N Speechley, Sylvia Stockler, Brenda J Wilson, Kumanan Wilson, Reem Zayed, Beth K Potter, on behalf of the Canadian Inherited Metabolic Diseases Research Network (CIMDRN)

Published in: BMC Pediatrics | Issue 1/2015

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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.
Appendix
Available only for authorised users
Literature
1.
Clarke JTR. Is the current approach to reviewing new drugs condemning the victims of rare diseases to death? A call for a national orphan drug review policy. Can Med Assoc J. 2006;174(2):189–90.CrossRef
2.
Griggs RC, Batshaw M, Dunkle M, Gopal-Srivastava R, Kaye E, Krischer J, et al. Clinical research for rare disease: opportunities, challenges, and solutions. Mol Genet Metab. 2009;96(1):20–6. doi: 10.1016/j.ymgme.2008.10.003.CrossRefPubMed
3.
Schieppati A, Henter J-I, Daina E, Aperia A. Why rare diseases are an important medical and social issue. Lancet. 2008;371(9629):2039–41.CrossRefPubMed
4.
5.
Huang IC, Revicki D, Schwartz C. Measuring pediatric-patient-reported outcomes: good progress but a long way to go. Qual Life Res. 2014;23(3):747–50. doi:10.1007/s11136-013-0607-2.CrossRefPubMedPubMedCentral
6.
Arbuckle R, Abetz-Webb L. Not just little adults: qualitative methods to support the development of pediatric patient-reported outcomes. Patient. 2013;6(3):143–59. doi: 10.1007/s40271-013-0022-3.CrossRefPubMed
7.
Geist R, Grdisa V, Otley A. Psychosocial issues in the child with chronic conditions. Best Pract Res Clin Gastroenterol. 2003;17(2):141–52.CrossRefPubMed
8.
Kuo DZ, Houtrow AJ, Arango P, Kuhlthau KA, Simmons JM, Neff JM. Family-centered care: current applications and future directions in pediatric health care. Matern Child Health J. 2012;16(2):297–305. doi:10.1007/s10995-011-0751-7.CrossRefPubMed
9.
Reiss JG, Gibson RW, Walker LR. Health care transition: youth, family, and provider perspectives. Pediatrics. 2005;115(1):112–20. doi:10.1542/peds. 2004-1321.PubMed
10.
Liberati A. Need to realign patient-oriented and commercial and academic research. Lancet. 2011;378(9805):1777–8. doi:10.1016/S0140-6736(11)61772-8.CrossRefPubMed
11.
12.
Washington AE, Lipstein SH. The Patient-Centered Outcomes Research Institute — promoting better information, decisions, and health. N Engl J Med. 2011;365(15):e31. doi: 10.1056/NEJMp1109407.CrossRefPubMed
13.
Irwin DE, Gross HE, Stucky BD, Thissen D, DeWitt EM, Lai JS, et al. Development of six PROMIS pediatrics proxy-report item banks. Health Qual Life Outcomes. 2012;10:22. doi:10.1186/1477-7525-10-22.CrossRefPubMedPubMedCentral
14.
15.
16.
Gardiner R. The transition from ‘informed patient’ care to ‘patient informed’ care. Stud Health Technol Inform. 2008;137:241–56.PubMed
17.
Barry MJ, Edgman-Levitan S. Shared decision making — the pinnacle of patient-centered care. N Engl J Med. 2012;366(9):780–1. doi:10.1056/NEJMp1109283.CrossRefPubMed
18.
O’Connor AM, Llewellyn-Thomas, HA, Barry-Flood, A. Modifying unwarranted variations in health care: shared decision making using patient decision aids. Health Aff (Millwood) 2004; Suppl Variation:VAR63-72. doi:10.1377/hlthaff.var.63
19.
Hamburg MA, Collins FS. The path to personalized medicine. N Engl J Med. 2010;363(4):301–4. doi:10.1056/NEJMp1006304.CrossRefPubMed
20.
Teng K, Eng C, Hess CA, Holt MA, Moran RT, Sharp RR, et al. Building an innovative model for personalized healthcare. Cleve Clin J Med. 2012;79 Suppl 1:S1–9. doi:10.3949/ccjm.79.s1.01.CrossRefPubMed
21.
Sacristán JA. Patient-centered medicine and patient-oriented research: improving health outcomes for individual patients. BMC Med Inform Decis Mak. 2013;13(1):6. doi:10.1186/1472-6947-13-6.CrossRefPubMedPubMedCentral
22.
Sacristán JA. Evidence based medicine and patient centered medicine: some thoughts on thier integration. Rev Clin Esp. 2013;213(9):460–4. doi:10.1016/j.rceng.2013.09.002.CrossRefPubMed
23.
Porter ME. What is value in health care? N Engl J Med. 2010;363:2477–81. doi:10.1056/NEJMp1011024.CrossRefPubMed
24.
Potter BK, Chakraborty P, Kronick JB, Wilson K, Coyle D, Feigenbaum A, et al. Achieving the “triple aim” for inborn errors of metabolism: a review of challenges to outcomes research and presentation of a new practice-based evidence framework. Genet Med. 2013;15(6):415–22. doi:10.1038/gim.2012.153.CrossRefPubMed
25.
Khangura SD, Karaceper MD, Chakraborty P, Mitchell JJ, Trakadis Y, Grosse SD Laberge AM, et al. A scoping review method for synthesizing literature reporting patient- and family-oriented outcomes: case description. Manuscript under review. 2014
26.
Wastfelt M, Fadeel B, Henter JI. A journey of hope: lessons learned from studies on rare diseases and orphan drugs. J Intern Med. 2006;260(1):1–10. doi:10.1111/j.1365-2796.2006.01666.x.CrossRefPubMed
27.
Ebell MH, Siwek J, Weiss BD, Woolf SH, Susman J, Ewigman B, et al. Strength of recommendation taxonomy (SORT): a patient-centered approach to grading evidence in the medical literature. Am Fam Physician. 2004;69(3):548–56.PubMed
28.
Schardt C, Adams M, Owens T, Keitz S, Fontelo P. Utilization of the PICO framework to improve searching PubMed for clinical questions. BMC Med Inform Decis Mak. 2007;7:16. doi:10.1186/1472-6947-7-16.CrossRefPubMedPubMedCentral
29.
Khangura S, Konnyu K, Cushman R, Grimshaw J, Moher D. Evidence summaries: the evolution of a rapid review approach. Syst Rev. 2012;1(1):10. doi:10.1186/2046-4053-1-10.CrossRefPubMedPubMedCentral
30.
McDowell I. Measuring health: a guide to rating scales and questionnaires. 3rd ed. New York: Oxford University Press; 2006.CrossRef
31.
Liberati A, Altman DG, Tetzlaff J, Mulrow C, Gøtzsche PC, Ioannidis JPA, et al. The PRISMA statement for reporting systematic reviews and meta-analyses of studies that evaluate healthcare interventions: explanation and elaboration. BMJ. 2009;339:b2700. doi:10.1136/bmj.b2700.CrossRefPubMedPubMedCentral
32.
Institute of Medicine (IOM) Committee on Accelerating Rare Diseases Research and Orphan Product Development. Chapter 2: profile of rare diseases. In: Field MJ, Boat TF, editors. Rare diseases and orphan products: accelerating research and development. Washington, DC: National Academies Press; 2010. p. 41–72.
33.
Eiser C, Morse R. A review of measures of quality of life for children with chronic illness. Arch Dis Child. 2001;84(3):205–11. doi:10.1136/adc.84.3.205.CrossRefPubMedPubMedCentral
34.
Garvie PA, Lawford J, Banet MS, West RL. Quality of life measurement in paediatric and adolescent populations with HIV: a review of the literature. Child Care Health Dev. 2009;35(4):440–53. doi:10.1111/j.1365-2214.2009.00985.x.CrossRefPubMed
35.
Glasgow RE, Anderson BJ. Future directions for research on pediatric chronic disease management: lessons from diabetes. J Pediatr Psychol. 1995;20(4):389–402. doi:10.1093/jpepsy/20.4.389.CrossRefPubMed
36.
Ingerski LM, Modi AC, Hood KK, Pai AL, Zeller M, Piazza-Waggoner C, et al. Health-related quality of life across pediatric chronic conditions. J Pediatr. 2010;156(4):639–44. doi:10.1016/j.jpeds.2009.11.008.CrossRefPubMedPubMedCentral
37.
MacKean GL, Thurston WE, Scott CM. Bridging the divide between families and health professionals’ perspectives on family-centred care. Health Expect. 2005;8(1):74–85.CrossRefPubMed
38.
Butler A, Copnell B, Willetts G. Family-centred care in the paediatric intensive care unit: an integrative review of the literature. J Clin Nurs. 2013;23(15–16):2086–99. doi:10.1111/jocn.12498.PubMed
39.
Shields L, Zhou H, Pratt J, Taylor M, Hunter J, Pascoe E. Family-centred care for hospitalised children aged 0-12 years. Cochrane Database Syst Rev. 2012;10:CD004811. doi:10.1002/14651858.CD004811.pub3.PubMed
40.
41.
Epstein JF, Barker PR, Kroutil LA. Mode effects in self-reported mental health data. Public Opin Q. 2001;65(4):529–49. doi:10.1086/323577.CrossRef
42.
Tourangeau R, Yan T. Sensitive questions in surveys. Psychol Bull. 2007;133(5):859–83. doi:10.1037/0033-2909.133.5.859.CrossRefPubMed
43.
44.
45.
Jokovic A, Locker D, Guyatt G. How well do parents know their children? Implications for proxy reporting of child health-related quality of life. Qual Life Res. 2004;13(7):1297–307. doi:10.1023/B:QURE.0000037480.65972.eb.CrossRefPubMed
46.
Riley AW. Evidence that school-age children can self-report on their health. Ambul Pediatrics. 2004;4(4):371–6. doi:10.1367/A03-178R.1.CrossRef
47.
Von Elm E, Altman DG, Egger M, Von Elm E, Altman DG, Egger M, et al. The Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) statement: guidelines for reporting observational studies. PLoS Med. 2007;4(10):e296. doi:10.1371/journal.pmed.0040296.CrossRefPubMedPubMedCentral
48.
Kohlmann T. STROBE: strongly recommended by IJPH–but not enforced! Int J Public Health. 2008;53(1):1–2. doi:10.1007/s00038-007-0238-x.CrossRefPubMed
49.
Moher D, Schulz KF, Simera I, Altman DG. Guidance for developers of health research reporting guidelines. PLoS Med. 2010;7(2):e1000217. doi:10.1371/journal.pmed.1000217.CrossRefPubMedPubMedCentral
50.
Bennett C, Khangura S, Brehaut JC, Graham ID, Moher D, Potter BK, et al. Reporting guidelines for survey research: an analysis of published guidance and reporting practices. PLoS Med. 2011;8(8):e1001069. doi:10.1371/journal.pmed.1001069.CrossRefPubMedCentral
Metadata
Title
Scoping review of patient- and family-oriented outcomes and measures for chronic pediatric disease
Authors
Sara D Khangura
Maria D Karaceper
Yannis Trakadis
John J Mitchell
Pranesh Chakraborty
Kylie Tingley
Doug Coyle
Scott D Grosse
Jonathan B Kronick
Anne-Marie Laberge
Julian Little
Chitra Prasad
Lindsey Sikora
Komudi Siriwardena
Rebecca Sparkes
Kathy N Speechley
Sylvia Stockler
Brenda J Wilson
Kumanan Wilson
Reem Zayed
Beth K Potter
on behalf of the Canadian Inherited Metabolic Diseases Research Network (CIMDRN)
Publication date
01-12-2015
Publisher
BioMed Central
Published in
BMC Pediatrics / Issue 1/2015
Electronic ISSN: 1471-2431
DOI
https://doi.org/10.1186/s12887-015-0323-x

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