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The size of the treatment effect: do patients and proxies agree?

van der Linden, FAH; Kragt, JJ; Hobart, JC; Klein, M; Thompson, AJ; van der Ploeg, HM; Polman, CH; (2009) The size of the treatment effect: do patients and proxies agree? BMC Neurology , 9 , Article 12. 10.1186/1471-2377-9-12. Green open access

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Abstract

Background: This study examined whether MS patients and proxy respondents agreed on change in disease impact, which was induced by treatment. This may be of interest in situations when patients suffer from limitations that interfere with reliable self-assessment, such as cognitive impairment.Methods: MS patients and proxies completed the Multiple Sclerosis Impact Scale (MSIS-29) before and after intravenous steroid treatment. Analyses focused on patient-proxy agreement between MSIS-29 change scores. Transition ratings were used to measure the patient's judgement of change and whether this change was reflected in the MSIS-29 change of patients and proxies. Receiver operating characteristic (ROC) analyses were also performed to examine the diagnostic properties of the MSIS-29 when completed by patients and proxies.Results: 42 patients and proxy respondents completed the MSIS-29 at baseline and follow-up. Patient-proxy differences between change scores on the physical and psychological MSIS-29 subscale were quite small, although large variability was found. The direction of mean change was in concordance with the transition ratings of the patients. Results of the ROC analyses of the MSIS-29 were similar when completed by patients (physical scale: AUC = 0.79, 95% CI: 0.65 - 0.93 and 0.66, 95% CI: 0.48 - 0.84 for the psychological scale) and proxies (physical scale: 0.80, 95% CI: 0.72 - 0.96 and 0.71, 95% CI: 0.56 - 0.87 for the psychological scale)Conclusion: Although the results need to be further explored in larger samples, these results do point towards possible use of proxy respondents to assess patient perceived treatment change at the group level.

Type: Article
Title: The size of the treatment effect: do patients and proxies agree?
Open access status: An open access version is available from UCL Discovery
DOI: 10.1186/1471-2377-9-12
Publisher version: http://dx.doi.org/10.1186/1471-2377-9-12
Language: English
Additional information: © 2009 van der Linden et al; licensee BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
Keywords: IMPACT-SCALE MSIS-29, QUALITY-OF-LIFE, MULTIPLE-SCLEROSIS, SELF-REPORT, RESPONSIVENESS
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences
URI: https://discovery.ucl.ac.uk/id/eprint/147321
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