Understanding patient-reported outcome measures in Huntington disease: at what point is cognitive impairment related to poor measurement reliability?

PURPOSE: Symptom progression in Huntington disease (HD) is associated with cognitive decline which may interfere with the self-report of symptoms. Unfortunately, data to support or refute the psychometric reliability of patient-reported outcomes (PROs) as HD progresses are limited. This is problematic given that PROs are increasingly recognized as important measures of efficacy for new treatments. METHODS: We examined PRO data from the HDQLIFE Measurement System (Speech Difficulties; Swallowing Difficulties; Chorea) in 509 individuals with premanifest, early-stage, or late-stage HD. Clinician-administered assessments of motor functioning (items from the UHDRS) and standardized objective assessments of cognition (Stroop, Symbol Digit Modalities) were also collected. We examined item bias using differential item functioning (DIF) across HD stage (premanifest, early-, late-) and relative to cognitive performance. We also examined the correlations between self-report and clinician ratings. Regression models that considered total cognitive ability were utilized to determine psychometric reliability of the PROs. RESULTS: Most PRO items were free from DIF for both staging and cognition. There were modest correlations between PROs and clinician report (ranged from - 0.40 to - 0.60). Modeling analyses indicated that psychometric reliability breaks down with poorer cognition and more progressed disease stage; split-half reliability was compromised (i.e., split-half reliability < 0.80) when scores were < 136 for Chorea, < 109 for Speech Difficulties, and < 179 for Swallowing Difficulties. CONCLUSIONS: Results indicate that the psychometric reliability of PROs can be compromised as HD symptoms progress and cognition declines. Clinicians should consider PROs in conjunction with other types of assessments when total cognition scores exceed critical thresholds.

Are intensive data collection methods in pain research feasible in those with physical disability? A study in persons with chronic pain and spinal cord injury.

PURPOSE: Intensive repeated measures data collection procedures, such as ecological momentary assessment (EMA) and end-of-day (EOD) diaries, are becoming more prominent in pain research. Existing data on the feasibility of such methods is encouraging; however, almost nothing is known about feasibility in clinical populations with significant physical disabilities. Research methodology feasibility is crucial to the inclusion of individuals with physical disability in pain research given the high prevalence and impact of pain in these populations. The aim of this study was to examine study compliance, protocol acceptability, and reactivity of intensive data collection methods in adults with chronic pain and spinal cord injury (SCI). METHODS: Secondary analysis of data from a 7-day EMA and EOD diary study in a sample of 131 community dwelling adults with SCI. RESULTS: Results showed rates of missing data ranged from 18.4 to 22.8% across measures. Participant compliance was related to time of day/presence of audible prompts, mobility aid use, race, and baseline levels of pain and pain interference, with more missing data at wake and bedtimes/no prompts, and for those who used hand-held mobility devices, identified as black/African American, and/or reported higher baseline pain and pain interference. Participants rated the study methodology as generally highly acceptable and expressed willingness to participate in similar studies of much longer duration. There was no evidence of reactivity, defined as temporal shifts in pain or pain interference ratings. CONCLUSIONS: Overall, intensive pain data collection is feasible in persons with SCI with no evidence that the methodology impacts pain intensity or pain interference ratings.

HDQLIFE: development and assessment of health-related quality of life in Huntington disease (HD).

PURPOSE: Huntington disease (HD) is a chronic, debilitating genetic disease that affects physical, emotional, cognitive, and social health. Existing patient-reported outcomes (PROs) of health-related quality of life (HRQOL) used in HD are neither comprehensive, nor do they adequately account for clinically meaningful changes in function. While new PROs examining HRQOL (i.e., Neuro-QoL-Quality of Life in Neurological Disorders and PROMIS-Patient-Reported Outcomes Measurement Information System) offer solutions to many of these shortcomings, they do not include HD-specific content, nor have they been validated in HD. HDQLIFE addresses this by validating 12 PROMIS/Neuro-QoL domains in individuals with HD and by using established PROMIS methodology to develop new, HD-specific content. METHODS: New item pools were developed using cognitive debriefing with individuals with HD, and expert, literacy, and translatability reviews. Existing item banks and new item pools were field tested in 536 individuals with prodromal, early-, or late-stage HD. RESULTS: Moderate to strong relationships between Neuro-QoL/PROMIS measures and generic self-report measures of HRQOL, and moderate relationships between Neuro-QoL/PROMIS and clinician-rated measures of similar constructs supported the validity of Neuro-QoL/PROMIS in individuals with HD. Exploratory and confirmatory factor analysis, item response theory, and differential item functioning analyses were utilized to develop new item banks for Chorea, Speech Difficulties, Swallowing Difficulties, and Concern with Death and Dying, with corresponding six-item short forms. A four-item short form was developed for Meaning and Purpose. CONCLUSIONS: HDQLIFE encompasses both validated Neuro-QoL/PROMIS measures, as well as five new scales in order to provide a comprehensive assessment of HRQOL in HD.

How well do physical activity questions perform? A European cognitive testing study.

BACKGROUND: Only few studies have focused on the cognitive processes of the respondents that are involved when answering physical activity questionnaires (PAQs). This study aimed at examining whether two PAQs work as intended with different segments of the survey population in different cultural settings in Europe. METHODS: The International Physical Activity Questionnaire - Short Form (IPAQ-SF) and the US National Health Interview Survey - Adult Core Physical Activity Questionnaire (NHIS-PAQ) were tested in Belgium, Estonia, Germany and the UK using a standardized cognitive interviewing procedure. IPAQ-SF measures total vigorous physical activity (PA), moderate PA, walking and sitting. NHIS-PAQ measures leisure-time vigorous PA, light and moderate PA and muscle-strengthening PA. In total 62 persons completed cognitive interviews, at least 15 interviews were conducted in each country. RESULTS: Both PAQs performed as intended with young and high-skilled persons and those having a regular exercise schedule. For the others, however, the testing revealed that problems occurred with both PAQs relating to understanding the concepts of '(light and) moderate' and 'vigorous' PA, classifying activities into the provided answer options of different PA intensities, recalling instances of 'normal' activities such as walking and sitting, and calculating the total duration of more than one activity or instance of an activity. The revealed problems with the questionnaires were quite similar in different countries; profound cultural differences were not observed. CONCLUSIONS: Both questionnaires were difficult to answer for many respondents and rather user-unfriendly. They are designed to measure an exactness of PA quantity (frequency and duration) and intensity which would be desirable to obtain from a scientific point of view; however, respondents can hardly provide this information for cognitive reasons. Studies investigating the respondents' perspective are useful for improving physical activity information based on self-reports.

Patterns of pain across the acute SCI rehabilitation stay: evidence from hourly ratings.

STUDY DESIGN: Cohort. OBJECTIVES: To examine patterns of pain intensity and variability during acute spinal cord injury (SCI) rehabilitation. SETTING: Large medical university in the Midwestern United States. METHODS: Data were collected from the medical records of consecutively admitted patients with new (< or =2 months after onset), traumatic (that is, injury resulting from external forces) or non-traumatic (that is, injury resulting from disease processes) SCI. A total of 11,001 hourly pain ratings on 1709 inpatient days were collected from 56 inpatients. Multi-leveling modeling was used to test models of pain intensity, pain variability, diurnal variability and pain medication administration. RESULTS: Pain intensity and variability decreased during the inpatient stay. Compared with those with non-traumatic injuries, those with traumatic injuries had significantly higher pain; those with American Spinal Injury Association Impairment Score (AIS) A scores had a slower decline of pain, while those with AIS D scores had a sharper decline. Pain increased from morning to evening during the latter days of the inpatient stay whereas pain was relatively stable during the early days in the inpatient stay. Those not using a ventilator at admission were significantly less likely to receive a pain medication than those who were, despite no significant differences in pain levels. CONCLUSION: Pain changes during acute rehabilitation, however, the moderating effect of time suggests that change is not consistent across all injury characteristics. Findings suggest that not only should pain management be individualized but it should also reflect a greater understanding of change over time.