Validation of Neuro-QoL and PROMIS Mental Health Patient Reported Outcome Measures in Persons with Huntington Disease

Article type: Research Article

Authors: Carlozzi, Noelle E.^{a; *} | Goodnight, Siera^a | Kratz, Anna L.^a | Stout, Julie C.^b | McCormack, Michael K.^{c; d} | Paulsen, Jane S.^e | Boileau, Nicholas R.^a | Cella, David^f | Ready, Rebecca E.^g

Affiliations: [a] Department of Physical Medicine and Rehabilitation, University of Michigan, Ann Arbor, MI, USA | [b] Institute of Cognitive and Clinical Neurosciences, School of Psychological Sciences, Monash University, Clayton, Victoria, Australia | [c] Department of Psychiatry, Rutgers-Robert Wood Johnson Medical School, Piscataway, NJ, USA | [d] Department of Pathology, Rowan-School of Medicine, Stratford, NJ, USA | [e] Departments of Psychiatry, Neurology, and Psychology, Carver College of Medicine, The University of Iowa, Iowa City, IA, USA | [f] Departments of Medical Social Sciences and Preventative Medicine, Northwestern University, Chicago, IL, USA | [g] Department of Psychological and Brain Sciences, University of Massachusetts, Amherst, MA, USA

Correspondence: [*] Correspondence to: Noelle E. Carlozzi, PhD, University of Michigan, Department of Physical Medicine & Rehabilitation, North Campus Research Complex, 2800 Plymouth Road, Building NCRC B14, Room G216, Ann Arbor, MI 48109-2800, USA. Tel.: +1 734 763 8917; Fax: +1 734 763 7186; E-mail: carlozzi@med.umich.edu.

Abstract: Background:Patient-reported outcomes (PROs) for mental health are important for persons with Huntington disease (HD) who commonly experience symptoms of depression, anxiety, irritability, anger, aggression, and apathy. Given this, there is a need for reliable and valid patient-reported outcomes measures of mental health for use as patient-centered outcomes in clinical trials. Objective:Thus, the purpose of this study was to establish the psychometric properties (i.e., reliability and validity) of six Neuro-QoL and PROMIS mental health measures to support their clinical utility in persons with HD. Methods:294 individuals with premanifest (n = 102) or manifest HD (n = 131 early HD; n = 61 late HD) completed Neuro-QoL/PROMIS measures of Emotional and Behavioral Dyscontrol, Positive Affect and Well-Being, Stigma, Anger, Anxiety, and Depression, legacy measures of self-reported mental health, and clinician-rated assessments of functioning. Results:Convergent validity and discriminant validity for the Neuro-QoL and PROMIS measures of Emotional and Behavioral Dyscontrol, Positive Affect and Well-Being, Stigma, Anger, Anxiety, and Depression, were supported in persons with HD. Neuro-QoL measures of Anxiety and Depression also demonstrated moderate sensitivity and specificity (i.e., they were able to distinguish between individuals with and without clinically significant anxiety and depression). Conclusions:Findings provide psychometric support for the clinical utility of the Neuro-QoL/PROMIS measures of mental health measures in persons with HD. As such, these measures should be considered for the standardized assessment of health-related quality of life in persons with HD.

Keywords: Neuro-QoL, PROMIS, emotion, mental health, validity, reliability, Huntington disease

DOI: 10.3233/JHD-190364

Journal: Journal of Huntington's Disease, vol. 8, no. 4, pp. 467-482, 2019