In addition to HDQLIFE™ , selected measures from three additional measurement systems have been tested and validated for use in HD.
PROMIS® (Patient-Reported Outcomes Measurement Information System) is a set of person-centered measures that evaluates and monitors physical, mental, and social health in adults and children. It can be used with the general population and with individuals living with chronic conditions.
Neuro-QoL (Quality of Life in Neurological Disorders) is a measurement system that evaluates and monitors the physical, mental, and social effects experienced by adults and children living with neurological conditions.
HD-PRO-TRIAD is a Huntington's Disease (HD)-specific, patient-reported outcome (PRO) instrument of the HD symptom triad (cognitive decline, emotional/behavioral dyscontrol, and motor dysfunction) designed for clinical research and practice.
Validation of PROMIS/Neuro-QoL for Use in HD
The PROMIS/Neuro-QoL measures were examined in over 500 individuals with HD. Pearson correlations between the PROMIS/Neuro-QoL measures and comparator measures were examined to establish construct validity. Comparator measures included two commonly used self-report measures of HRQOL (Rand 12 and WHODAS 2.0), as well as clinician-rated assessments that are commonly administered in HD. The clinician-rated assessments included several components of the United Huntington Disease Rating Scales (Total Motor Score, Independence Scale, Symbol Digit Modalities Test, and Stroop) and the Problem Behaviors Assessment (PBA-s; Apathy, Irritability, Aggression, Anxiety, and Depression). Evidence for convergent validity can be established when correlations between scores from measures of the same trait are strong whereas evidence for discriminant validity can be established when correlations between scores of different traits are poor. Construct validity would be supported by stronger correlations among the PROMIS/Neuro-QoL measures and the comparator self-report measures, and less robust correlations with clinician-rated assessments. As hypothesized, Neuro-QoL/PROMIS measures had stronger relationships with comparator self-report measures than with the clinician-rated measures. Thus, preliminary support for the validity of Neuro-QoL/PROMIS for use in HD was obtained.
PROMIS/Neuro-QoL Measures used in validation:
Neuro-QoL Upper Extremity Function - Fine Motor, ADL
Neuro-QoL Lower Extremity Function - Mobility
Neuro-QoL Ability to Participate in Social Roles and Activities
Neuro-QoL Satisfaction with Social Roles and Activities
Neuro-QoL Emotional and Behavioral Dyscontrol
Neuro-QoL Positive Affect and Well-Being
Neuro-QoL Applied Cognition - Executive Function
Neuro-QoL Applied Cognition - General Concerns
Carlozzi, N. E., Hahn, E. A., Goodnight, S. M., Kratz, A. L., Paulsen, J. S., Stout, J. C., . . . Ready, R. E. (2018). Patient-reported outcome measures in Huntington disease: Quality of life in neurological disorders (Neuro-QoL) social functioning measures. Psychol Assess, 30(4), 450-458. doi:10.1037/pas0000479
Lai, J. S., Goodnight, S., Downing, N. R., Ready, R. E., Paulsen, J. S., Kratz, A. L., . . . Carlozzi, N. E. (2018). Evaluating cognition in individuals with Huntington disease: Neuro-QoL cognitive functioning measures. Qual Life Res, 27(3), 811-822. doi:10.1007/s11136-017-1755-6
Carlozzi, N. E., Ready, R. E., Frank, S. , Cella, D. , Hahn, E. A., Goodnight, S. M., Schilling, S. G., Boileau, N. R. and Dayalu, P. (2017), Patient‐reported outcomes in Huntington's disease: Quality of life in neurological disorders (Neuro‐QoL) and Huntington's disease health‐related quality of life (HDQLIFE) physical function measures. Mov Disord., 32: 1096-1102. doi:10.1002/mds.27046
Validation of HD-PRO-TRIAD for Use in HD
To validate the use of HD-PRO-TRIAD as a clinical measure in individuals with HD, 482 individuals with HD completed the HD-PRO-TRIAD questionnaire. Clinician-rated assessments from the Unified Huntington Disease Rating Scales (UHDRS), the Problem Behaviors Assessment (PBA-s), and three generic measures of HRQOL (WHODAS 2.0, RAND-12, and EQ-5D) were also examined. It was discovered that internal reliability for all domains was excellent. Convergent and discriminant validity were supported by significant associations between the HD-PRO-TRIAD domains and other patient reported outcome measures as well as clinician-rated measures. Known groups validity was also supported as the HD-PRO-TRIAD differentiated between stages of the disease. Thus, the findings supported excellent internal reliability, convergent and discriminant validity, known groups validity, and overall clinical efficacy of the HD-PRO-TRIAD.
HD-PRO-TRIAD Measures used in validation:
Cognition (9 of 14 questions used)
Emotional and Behavioral Dyscontrol (13 of 14 questions used)
Motor Functioning (13 of 19 questions used)
Carlozzi, N. E., Victorson, D., Sung, V., Beaumont, J. L., Cheng, W., Gorin, B., Duh, M. S., Samuelson, D., Tulsky, D., Gutierrez, S., Nowinski, C. J., Mueller, A., Shen, V., … Frank, S. (2014). HD-PRO-TRIAD™ Validation: A Patient-reported Instrument for the Symptom Triad of Huntington's Disease. Tremor and other hyperkinetic movements (New York, N.Y.), 4, 223. doi:10.7916/D8PN93NZ
Boileau, N. R., Stout, J. C., Paulsen, J. S., Cella, D., McCormack, M. K., Nance, M. A., . . . Carlozzi, N. E. (2017). Reliability and Validity of the HD-PRO-TriadTM, a Health-Related Quality of Life Measure Designed to Assess the Symptom Triad of Huntington's Disease. J Huntingtons Dis, 6(3), 201-215. doi:10.3233/JHD-170238