@article{b954decb2ded4268b590419e81633dab,
title = "The development of a new computer adaptive test to evaluate chorea in Huntington disease: HDQLIFE Chorea",
abstract = "Purpose: Huntington{\textquoteright}s disease (HD) is an autosomal dominant neurodegenerative disease associated with motor, behavioral, and cognitive deficits. The hallmark symptom of HD, chorea, is often the focus of HD clinical trials. Unfortunately, there are no self-reported measures of chorea. To address this shortcoming, we developed a new measure of chorea for use in HD, HDQLIFE Chorea. Methods: Qualitative data and literature reviews were conducted to develop an initial item pool of 141 chorea items. An iterative process, including cognitive interviews, expert review, translatability review, and literacy review, was used to refine this item pool to 64 items. These 64 items were field tested in 507 individuals with prodromal and/or manifest HD. Exploratory and confirmatory factor analyses (EFA and CFA, respectively) were conducted to identify a unidimensional set of items. Then, an item response theory graded response model (GRM) and differential item functioning analyses were conducted to select the final items for inclusion in this measure. Results: EFA and CFA supported the retention of 34 chorea items. GRM and DIF supported the retention of all of these items in the final measure. GRM calibration data were used to inform the selection of a 6-item, static short form and to program the HDQLIFE Chorea computer adaptive test (CAT). CAT simulation analyses indicated a 0.99 correlation between the CAT scores and the full item bank. Conclusions: The new HDQLIFE Chorea CAT and corresponding 6-item short form were developed using established rigorous measurement development standards; this is the first self-reported measure developed to evaluate the impact of chorea on HRQOL in HD. This development work indicates that these measures have strong psychometric properties; future work is needed to establish test–retest reliability and responsiveness to change.",
keywords = "Chorea, HDQLIFE, Health-related quality of life, Huntington{\textquoteright}s disease, Motor function, Neuro-QoL, PROMIS, Patient-reported outcome (PRO)",
author = "Carlozzi, {N. E.} and Downing, {N. R.} and Schilling, {S. G.} and Lai, {J. S.} and Goodnight, {S. M.} and Miner, {J. A.} and {A. Frank}, S.",
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 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: Carlozzi, N.E. currently has research grants from the NIH; she is also supported by grant funding from the NIH, NIDILRR, and CHDI; she declares no conflicts of interest. Downing, N.R. declares no conflicts of interest. Schilling, S.G. has a research grant from NSF. He is also supported by grant funding from NIH. He declares no conflicts of interest. Lai J.-S. currently has research grants from the NIH; she declares no conflicts of interest. Goodnight, S.M. is supported by grant funding from the NIH and the Craig H. Neilsen Foundation; she declares no conflicts of interest. Miner, J.A. is supported by research grants from the NIH; she declares no conflict of interest. Frank, S. receives salary support from the Huntington Study Group for a study sponsored by Auspex Pharmaceuticals. There is no conflict of interest. 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 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 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. Publisher Copyright: {\textcopyright} 2016, Springer International Publishing Switzerland.",
year = "2016",
month = oct,
day = "1",
doi = "10.1007/s11136-016-1307-5",
language = "English (US)",
volume = "25",
pages = "2429--2439",
journal = "Quality of Life Research",
issn = "0962-9343",
publisher = "Springer Netherlands",
number = "10",
}
