@article{d099efd4e5054210a255384e6e422a99,
title = "Agreement between clinician-rated versus patient-reported outcomes in Huntington disease",
abstract = "Background: Clinician-rated measures of functioning are often used as primary endpoints in clinical trials and other behavioral research in Huntington disease. As study costs for clinician-rated assessments are not always feasible, there is a question of whether patient self-report of commonly used clinician-rated measures may serve as acceptable alternatives in low risk behavioral trials. Aim: The purpose of this paper was to determine the level of agreement between self-report and clinician-ratings of commonly used functional assessment measures in Huntington disease. Design: 486 participants with premanifest or manifest Huntington disease were examined. Total Functional Capacity, Functional Assessment, and Independence Scale assessments from the Unified Huntington Disease Rating scale were completed by clinicians; a self-report version was also completed by individuals with Huntington disease. Cronbach{\textquoteright}s α was used to examine internal consistency, one-way analysis of variance was used to examine group differences, and paired t tests, kappa agreement coefficients, and intra-class correlations were calculated to determine agreement between raters. Results: Internal consistency for self-reported ratings of functional capacity and ability were good. There were significant differences between those with premanifest, early-, and late-stage disease; those with later-stage disease reported less ability and independence than the other clinical groups. Although self-report ratings were not a perfect match with associated clinician-rated measures, differences were small. Cutoffs for achieving specified levels of agreement are provided. Conclusions: Depending on the acceptable margin of error in a study, self-reported administration of these functional assessments may be appropriate when clinician-related assessments are not feasible.",
keywords = "Clinician-ratings, Functioning, Huntington disease, Self-report ratings",
author = "Carlozzi, {Noelle E.} and Boileau, {Nicholas R.} and Perlmutter, {Joel S.} and Chou, {Kelvin L.} and Stout, {Julie C.} and Paulsen, {Jane S.} and McCormack, {Michael K.} and David Cella and Nance, {Martha A.} and Lai, {Jin Shei} and Praveen Dayalu",
note = "Funding Information: Conflicts of interest N. E. Carlozzi currently has research grants from the NIH; she is also supported by grant funding from the NIH and CHDI. She provides patient-reported outcome measurement selection and application consultation for Teva Pharmaceuticals. She declares no conflicts of interest. N. R. Boileau is supported by grant funding from the NIH; he declares no conflicts of interest. J. S. Perlmutter currently has research funding from the NIH, HDSA, CHDI, Michael J Fox Foundation, Barnes Jewish Hospital Foundation, the Lauren and Lee Fixel Family Foundation, and the APDA. He has received honoraria from the University of Rochester, American Academy of Neurology, Movement Disorders Society, Toronto Western Hospital, Alberta Innovates, Parkinson Disease Foundation, Columbia University, St. Louis University, Harvard University, University of Michigan, Huntington Study Group, Stanford University, University of Florida at Gainesville, and World Parkinson Congress. He declares no conflicts of interest. K. Chou currently has funding from the NIH and Cavion, receives royalties from UpToDate, Springer Publishing and Demos Health and serves as a consultant for Accordant and Sunovion Pharmaceuticals. He declares no conflicts of interest. J. C. Stout has received research funding in the past 3 years from the Australian National Health and Medical Research Council, University College London, the CHDI Foundation, Prana Biotechnology, and the University of California, Davis. She is a Director of Stout Neuropsych Pty Ltd, which has received funding from Omeros, Teva Pharmaceuticals, Vac-cinex, and Isis. She has been a consultant to Prana Biotechnology and Roche. She receives compensation as a member of the Board of the Huntington{\textquoteright}s Study Group. She declares no conflicts of interest. J. S. Paulsen currently has research grants from the NIH; she is also supported by grant funding from NIH, NINDS, and CHDI; she declares no conflicts of interest. M. K. McCormack currently has grants from the NJ Department of Health; he declares no conflicts of interest. D. Cella receives grant funding from the National Institutes of Health and reports that he has no conflicts of interest. M. A. Nance currently has funding from the NIH, HDSA, CHDI, and the Parkinson{\textquoteright}s Foundation. She has received research funding in the last 3 years from Teva Pharmaceuticals, Biotie, and Sunovion. She has received honoraria from WebMD, Worrell Inc, Optio Biopharma, and Augsburg College. She declares on conflicts of interest. J.-S. Lai currently has research grants from the NIH and Neurofibromatosis Therapeutic Acceleration Program at John Hopkins University; she declares no conflicts of interest. P. Dayalu currently has research grants from the NIH, Astra-Zeneca, and Vaccinex. He declares no conflicts of interest. Funding Information: Funding Work on this manuscript was supported by the National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (R01NS077946) and the National Center for Advancing Translational Sciences (UL1TR000433). In addition, a portion of this study sample was collected in conjunction with the Predict-HD study. The Predict-HD was supported by the NIH, National Institute of Neurological Disorders and Stroke (R01NS040068), the NIH, Center for Inherited Disease Research (provided supported for sample phenotyping), and the CHDI Foundation (award to the University of Iowa). Funding Information: Acknowledgements Work on this manuscript was supported by the National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (R01NS077946) and the National Center for Advancing Translational Sciences (UL1TR000433). In addition, a portion of this study sample was collected in conjunction with the Predict-HD study. The Predict-HD study was supported by the NIH, National Institute of Neurological Disorders and Stroke (R01NS040068), the NIH, Center for Inherited Disease Research (provided supported for sample phenotyping), and the CHDI Foundation (award to the University of Iowa). We thank the University of Iowa, the Investigators and Coordinators of this study, the study participants, the National Research Roster for Huntington Disease Patients and Families, the Huntington Study Group, and the Huntington{\textquoteright}s Disease Society of America. We acknowledge the assistance of Jeffrey D. Long, Hans J. Johnson, Jeremy H. Bockholt, Roland Zschiegner, and Jane S. Paulsen. We also acknowledge Roger Albin, Kelvin Chou, and Henry Paulsen Funding Information: Work on this manuscript was supported by the National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (R01NS077946) and the National Center for Advancing Translational Sciences (UL1TR000433). In addition, a portion of this study sample was collected in conjunction with the Predict-HD study. The Predict-HD study was supported by the NIH, National Institute of Neurological Disorders and Stroke (R01NS040068), the NIH, Center for Inherited Disease Research (provided supported for sample phenotyping), and the CHDI Foundation (award to the University of Iowa). We thank the University of Iowa, the Investigators and Coordinators of this study, the study participants, the National Research Roster for Huntington Disease Patients and Families, the Huntington Study Group, and the Huntington?s Disease Society of America. We acknowledge the assistance of Jeffrey D. Long, Hans J. Johnson, Jeremy H. Bockholt, Roland Zschiegner, and Jane S. Paulsen. We also acknowledge Roger Albin, Kelvin Chou, and Henry Paulsen for the assistance with participant recruitment. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH. HDQLIFE Site Investigators and Coordinators: Noelle Carlozzi, Praveen Dayalu, Stephen Schilling, Amy Austin, Matthew Canter, Siera Goodnight, Jennifer Miner, Nicholas Migliore (University of Michigan, Ann Arbor, MI); Jane Paulsen, Nancy Downing, Isabella DeSoriano, Courtney Shadrick, Amanda Miller (University of Iowa, Iowa City, IA); Kimberly Quaid, Melissa Wesson (Indiana University, Indianapolis, IN); Christopher Ross, Gregory Churchill, Mary Jane Ong (Johns Hopkins University, Baltimore, MD); Susan Perlman, Brian Clemente, Aaron Fisher, Gloria Obialisi, Michael Rosco (University of California Los Angeles, Los Angeles, CA); Michael McCormack, Humberto Marin, Allison Dicke (Rutgers University, Piscataway, NJ); Joel Perlmutter, Stacey Barton, Shineeka Smith (Washington University, St. Louis, MO); Martha Nance, Pat Ede (Struthers Parkinson?s Center); Stephen Rao, Anwar Ahmed, Michael Lengen, Lyla Mourany, Christine Reece, (Cleveland Clinic Foundation, Cleveland, OH); Michael Geschwind, Joseph Winer (University of California-San Francisco, San Francisco, CA), David Cella, Richard Gershon, Elizabeth Hahn, Jin-Shei Lai (Northwestern University, Chicago, IL). Publisher Copyright: {\textcopyright} 2018, Springer-Verlag GmbH Germany, part of Springer Nature.",
year = "2018",
month = jun,
day = "1",
doi = "10.1007/s00415-018-8852-5",
language = "English",
volume = "265",
pages = "1443--1453",
journal = "Journal of Neurology",
issn = "0340-5354",
number = "6",
}