@article{371a0279a664441f98af64dff5e12a2e,
title = "HDQLIFE and neuro-QoL physical function measures: Responsiveness in persons with huntington's disease",
abstract = "Background: Huntington's disease (HD) is a neurological disorder that causes severe motor symptoms that adversely impact health-related quality of life. Patient-reported physical function outcome measures in HD have shown cross-sectional evidence of validity, but responsiveness has not yet been assessed. Objectives: This study evaluates the responsiveness of the Huntington Disease Health-Related Quality of Life (HDQLIFE) and the Quality of Life in Neurological Disorders (Neuro-QoL) physical function measures in persons with HD. Methods: A total of 347 participants completed baseline and at least 1 follow-up (12-month and 24-month) measure (HDQLIFE Chorea, HDQLIFE Swallowing Difficulties, HDQLIFE Speech Difficulties, Neuro-QoL Upper Extremity Function, and/or Neuro-QoL Lower Extremity Function). Of the participants that completed the baseline assessment, 338 (90.9%) completed the 12-month assessment, and 293 (78.8%) completed the 24-month assessment. Standardized response means and general linear models evaluated whether the physical function measures were responsive to self-reported and clinician-rated change over time. Results: Small to moderate effect sizes for the standardized response means supported 12-month and 24-month responsiveness of the HDQLIFE and Neuro-QoL measures for those with either self-reported or clinician-rated declines in function. General linear models supported 12-month and 24-month responsiveness for all HRQOL measures relative to self-reported declines in health, but generally only 24-month responsiveness was supported relative to clinician-rated declines in function. Conclusions: Longitudinal analyses indicate that the HDQLIFE and the Neuro-QoL physical function measures are sensitive to change over time in individuals with HD. Thus, these scales exhibit evidence of responsiveness and may be useful outcome measures in future clinical trials.",
keywords = "Huntington's disease, health-related quality of life, patient-reported outcome (PRO), psychometric, validity",
author = "Carlozzi, {Noelle E.} and Boileau, {Nicholas R.} and Chou, {Kelvin L.} and Ready, {Rebecca E.} and David Cella and McCormack, {Michael K.} and Miner, {Jennifer A.} and Praveen Dayalu",
note = "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), th e 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. 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. Funding Information: N.E.C. is supported by research grants from the National Institutes of Health (NIH), the Neilsen Foundation, and CHDI as well as contracts from Goldfinch, LLC, and Health and Human Services–Centers for Medicare & Medicaid Services; she receives honoraria for her role on the CHDI scientific advisory board; she declares no conflicts of interest. N.R.B. has been supported by research grants from the NIH, the Neilsen Foundation, and CHDI. K.L.C. has received research support from the NIH (NS091856‐01, NS10061102, NS107158), Parkinson Study Group (Efficacy of Isradipine in early Parkinson Disease (STEADY‐PD III), Study of Urate Elevation in Parkinson's Disease, Phase 3 (SURE‐PD3), safety and tolerability study of nilotinib in Parkinson's disease (NILO‐PD)), and Eli Lilly. He has been a consultant for Accordant, Boston Scientific, and Sunovion Pharmaceuticals. He also receives royalties from UpToDate and Springer Publishing. R.E.R. declares that she has no conflicts of interest. D.C. receives grant funding from the NIH and reports that he has no conflicts of interest. M.M. currently has grants from the New Jersey Department of Health; he declares no conflicts of interest. J.A.M. is supported by funding from the NIH, patient‐centered outcomes research institute (PCORI), the Craig H. Neilsen Foundation, and Goldfinch Bio; she declares no conflicts of interest. P.D. is supported by grants from Vaccinex and CHDI. He declares no conflicts of interest. Publisher Copyright: {\textcopyright} 2019 International Parkinson and Movement Disorder Society",
year = "2020",
month = feb,
day = "1",
doi = "10.1002/mds.27908",
language = "English (US)",
volume = "35",
pages = "326--336",
journal = "Movement Disorders",
issn = "0885-3185",
publisher = "John Wiley and Sons Inc.",
number = "2",
}