TY - JOUR
T1 - Rescuing suboptimal patient-reported outcome instrument data in clinical trials
T2 - A new strategy
AU - Yang, Chengwu
AU - Vrana, Kent E.
N1 - Funding Information:
Acknowledgments: This study was sponsored, in part, by the NIH (National Institute of Neurological Disorders and Stroke), U01NS043127, U01NS043128, and U10NS44415 through 44555, and by the National Institute on Aging (NIA) Resource Centers for Minority Aging Research (RCMAR) Grant 3P30 AG021677-02S1. We thank the NINDS NET-PD Investigators for the high-quality data collected in NET-PD FS1 and FS-TOO. The authors wish to acknowledge Barbara C. Tilley and Jay S. Schneider for their contributions to a poster presentation made at the 35th Annual Meeting of the Society for Clinical Trials (SCT), Philadelphia, Pennsylvania, 18–21 May 2014. Part of the materials in this study are contained within that poster.
Funding Information:
This study was sponsored, in part, by the NIH (National Institute of Neurological Disorders and Stroke), U01NS043127, U01NS043128, and U10NS44415 through 44555, and by the National Institute on Aging (NIA) Resource Centers for Minority Aging Research (RCMAR) Grant 3P30 AG021677-02S1. We thank the NINDS NET-PD Investigators for the high-quality data collected in NET-PD FS1 and FS-TOO. The authors wish to acknowledge Barbara C. Tilley and Jay S. Schneider for their contributions to a poster presentation made at the 35th Annual Meeting of the Society for Clinical Trials (SCT), Philadelphia, Pennsylvania, 18–21 May 2014. Part of the materials in this study are contained within that poster.
Publisher Copyright:
© 2018 by the authors. Licensee MDPI, Basel, Switzerland.
PY - 2018/3
Y1 - 2018/3
N2 - Background: Psychometric instruments such as the Repeated Battery for the Assessment of Neuropsychological Status (RBANS) are commonly used under conditions for which they were not developed or validated. They may then generate troublesome data that could conceal potential findings. Methods: Based on a previously published refinement of the RBANS, we reanalyzed the data on 303 patients from two National Institutes of Health (NIH) trails in Parkinson’s disease and contrasted the results using the original versus refined scores. Results: Findings from the original RBANS scores were inconsistent; however, use of the refined scores produced potential findings that were in agreement with independent reports. Conclusion: This study demonstrates that, for negative trials using instrument scores as primary outcomes, it is possible to rescue potential findings. The key to this new strategy is to validate and refine the instrument for the specific disease and conditions under study and then to reanalyze the data. This study offers a demonstration of this new strategy for general approaches.
AB - Background: Psychometric instruments such as the Repeated Battery for the Assessment of Neuropsychological Status (RBANS) are commonly used under conditions for which they were not developed or validated. They may then generate troublesome data that could conceal potential findings. Methods: Based on a previously published refinement of the RBANS, we reanalyzed the data on 303 patients from two National Institutes of Health (NIH) trails in Parkinson’s disease and contrasted the results using the original versus refined scores. Results: Findings from the original RBANS scores were inconsistent; however, use of the refined scores produced potential findings that were in agreement with independent reports. Conclusion: This study demonstrates that, for negative trials using instrument scores as primary outcomes, it is possible to rescue potential findings. The key to this new strategy is to validate and refine the instrument for the specific disease and conditions under study and then to reanalyze the data. This study offers a demonstration of this new strategy for general approaches.
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U2 - 10.3390/healthcare6010027
DO - 10.3390/healthcare6010027
M3 - Article
AN - SCOPUS:85068314314
SN - 2227-9032
VL - 6
JO - Healthcare (Switzerland)
JF - Healthcare (Switzerland)
IS - 1
M1 - 27
ER -
