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A systematic review of studies that aim to determine which outcomes to measure in clinical trials in children

Sinha, Ian; Jones, Leanne; Smyth, Rosalind L. and Williamson, Paula R. (2008) A systematic review of studies that aim to determine which outcomes to measure in clinical trials in children. PLoS Medicine, 5 (4). Article Number: e96. 0569-0578. ISSN 1549-1277

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Abstract

Background In clinical trials the selection of appropriate outcomes is crucial to the assessment of whether one intervention is better than another. Selection of inappropriate outcomes can compromise the utility of a trial. However, the process of selecting the most suitable outcomes to include can be complex. Our aim was to systematically review studies that address the process of selecting outcomes or outcome domains to measure in clinical trials in children. Methods and Findings We searched Cochrane databases (no date restrictions) in December 2006; and MEDLINE (1950 to 2006), CINAHL (1982 to 2006), and SCOPUS (1966 to 2006) in January 2007 for studies of the selection of outcomes for use in clinical trials in children. We also asked a group of experts in paediatric clinical research to refer us to any other relevant studies. From these articles we extracted data on the clinical condition of interest, description of the method used to select outcomes, the people involved in the selection process, the outcomes selected, and limitations of the method as defined by the authors. The literature search identified 8,889 potentially relevant abstracts. Of these, 70 were retrieved, and 25 were included in the review. These studies described the work of 13 collaborations representing various paediatric specialties including critical care, gastroenterology, haematology, psychiatry, neurology, respiratory paediatrics, rheumatology, neonatal medicine, and dentistry. Two groups utilised the Delphi technique, one used the nominal group technique, and one used both methods to reach a consensus about which outcomes should be measured in clinical trials. Other groups used semistructured discussion, and one group used a questionnaire-based survey. The collaborations involved clinical experts, research experts, and industry representatives. Three groups involved parents of children affected by the particular condition. Conclusions Very few studies address the appropriate choice of outcomes for clinical research with children, and in most paediatric specialties no research has been undertaken. Among the studies we did assess, very few involved parents or children in selecting outcomes that should be measured, and none directly involved children. Research should be undertaken to identify the best way to involve parents and children in assessing which outcomes should be measured in clinical trials.

Item Type:Article
Additional Information:Published: April 29, 2008. Citation: Sinha I, Jones L, Smyth RL, Williamson PR (2008) A Systematic Review of Studies That Aim to Determine Which Outcomes to Measure in Clinical Trials in Children . PLoS Med 5(4): e96 doi:10.1371/journal.pmed.0050096
Uncontrolled Keywords:SURROGATE END-POINTS; LUPUS-ERYTHEMATOSUS; CORE SET; INTERNATIONAL CONSENSUS; PROSPECTIVE VALIDATION; DISEASE-ACTIVITY; WEST-SYNDROME; DEFINITIONS; IMPROVEMENT; CRITERIA
Subjects:R Medicine > R Medicine (General)
Departments, Research Centres and Related Units:Academic Faculties, Institutes and Research Centres > Faculty of Medicine > School of Reproductive & Developmental Medicine
Academic Faculties, Institutes and Research Centres > Research Centres > Medical Statistics and Health Evaluation, Centre for
DOI:10.1371/journal.pmed.0050096
Publisher's Statement:Copyright: © 2008 Sinha et al. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
Related URLs:
Refereed:Yes
Status:Published
ID Code:834
Deposited On:24 Jul 2008 14:56
Last Modified:23 Mar 2012 10:51

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