Siera Goodnight

New measures to capture end of life concerns in Huntington disease: Meaning and Purpose and Concern with Death and Dying from HDQLIFE (a patient-reported outcomes measurement system).

PurposeHuntington disease (HD) is an incurable terminal disease. Thus, end of life (EOL) concerns are common in these individuals. A quantitative measure of EOL concerns in HD would enable a better understanding of how these concerns impact health-related quality of life. Therefore, we developed new measures of EOL for use in HD.MethodsAn EOL item pool of 45 items was field tested in 507 individuals with prodromal or manifest HD. Exploratory and confirmatory factor analyses (EFA and CFA, respectively) were conducted to establish unidimensional item pools. Item response theory (IRT) and differential item functioning analyses were applied to the identified unidimensional item pools to select the final items.ResultsEFA and CFA supported two separate unidimensional sets of items: Concern with Death and Dying (16 items), and Meaning and Purpose (14 items). IRT and DIF supported the retention of 12 Concern with Death and Dying items and 4 Meaning and Purpose items. IRT data supported the development of both a computer adaptive test (CAT) and a 6-item, static short form for Concern with Death and Dying.ConclusionThe HDQLIFE Concern with Death and Dying CAT and corresponding 6-item short form, and the 4-item calibrated HDQLIFE Meaning and Purpose scale demonstrate excellent psychometric properties. These new measures have the potential to provide clinically meaningful information about end-of-life preferences and concerns to clinicians and researchers working with individuals with HD. In addition, these measures may also be relevant and useful for other terminal conditions.

The development of a new computer adaptive test to evaluate chorea in Huntington disease: HDQLIFE Chorea

Purpose Huntington’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.MethodsQualitative 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.ResultsEFA 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.ConclusionsThe 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.

HDQLIFE: the development of two new computer adaptive tests for use in Huntington disease, Speech Difficulties, and Swallowing Difficulties

PurposeHuntington disease (HD) is an autosomal dominant neurodegenerative disease which results in several progressive symptoms, including bulbar dysfunction (i.e., speech and swallowing difficulties). Although difficulties in speech and swallowing in HD have a negative impact on health-related quality of life, no patient-reported outcome measure exists to capture these difficulties that are specific to HD. Thus, we developed a new patient-reported outcome measure for use in the Huntington Disease Health-Related Quality of Life (HDQLIFE) Measurement System that focused on the impact that difficulties with speech and swallowing have on HRQOL in HD.MethodsFive hundred and seven individuals with prodromal and/or manifest HD completed 47 newly developed items examining speech and swallowing difficulties. Unidimensional item pools were identified using exploratory factor analysis and confirmatory factor analysis (EFA and CFA, respectively). Item response theory (IRT) was used to calibrate the final measures.ResultsEFA and CFA identified two separate unidimensional sets of items: Speech Difficulties (27 items) and Swallowing Difficulties (16 items). Items were calibrated separately for these two measures and resulted in item banks that can be administered as computer adaptive tests (CATs) and/or 6-item, static short forms. Reliability of both of these measures was supported through high correlations between the simulated CAT scores and the full item bank.ConclusionsCATs and 6-item calibrated short forms were developed for HDQLIFE Speech Difficulties and HDQLIFE Swallowing Difficulties. These measures both demonstrate excellent psychometric properties and may have clinical utility in other populations where speech and swallowing difficulties are prevalent.

Body mass index, but not blood pressure is related to the level of pain in persons with chronic pain

Objective: To determine whether obesity or blood pressure relate to the level of pain experienced by persons with chronic back pain. Design: Retrospective Study. Setting: University spine program. Patients (or Participants): Adults seeking care for back pain of more than 3 months duration. Methods or Interventions(or Assessment of Risk Factors ): Measurement of weight, blood pressure, and pain on a numeric scale. Main Outcome Measurements : Relationships between body mass index (BMI) and pain, blood pressure and pain. Results: 198 subjects had a mean BMI of 29.50 ± 7.12, with 33.3% obese. The relationship between pain as a continuous variable and BMI approached significance ( r = 0.125, n = 175, p = 0.098). The BMI of persons with lower pain scores of 0-3 was significantly different from those with high pain scores of 7-10. No significant relationship was found between pain a nd systolic (r = 0.126, p = 0.078) or diastolic (r = 0.099, p = 0.165) blood pressure. Conclusions: Contrary to assumptions about acute pain, increased chronic pain does not appear to relate to increased blood pressure. The relationship of obesity with increased pain l evel among persons who have chronic pain raises the possibility that psychological or physiological mechanisms may be important above and beyond the biomechanical impact of obesity.

Construct validity of the NIH toolbox cognition battery in individuals with stroke

Objective: The National Institutes of Health (NIH) Toolbox (NIHTB) for the Assessment of Behavior and Neurological Function Cognition Battery (NIHTB-CB) provides a brief assessment (approximately 30 min) of key components of cognition. This article examines construct validity to support the clinical utility of the NIHTB-CB in individuals with stroke. Research Method: A total of 131 individuals with stroke (n = 71 mild stroke; n = 60 moderate/severe stroke) completed the NIHTB-CB. Univariate analyses were conducted to examine the cognitive profiles of the two different stroke groups (mild vs. moderate/severe stroke) on NIHTB-CB measures and composite scores. Pearson correlations were conducted between NIHTB-CB and established measures to examine convergent and discriminant validity. Effect sizes and clinical impairment rates for the different NIHTB-CB measures and composite scores were also examined. Results: Participants experiencing moderate to severe stroke had poorer performance than did individuals with mild stroke on several of the NIHTB cognition measures. Evidence of convergent validity was provided by moderate to strong correlations between the NIHTB measures and the corresponding standard neuropsychological test (Pearson rs ranged from 0.31 to 0.88; median = .60). Evidence of discriminant validity was provided by smaller correlations between different cognitive domains than correlations of measures within the same domain. Effect sizes for composite and subtest scores regarding stroke severity were generally moderate-to-large. In addition, 42% of the sample were exhibiting mild cognitive impairment (i.e., ≥2 low scores on fluid tests). Conclusions: Findings provide support for the construct validity of the NIHTB-CB in individuals with stroke.

Validation of the NIH Toolbox in Individuals with Neurologic Disorders

Objective Individuals with spinal cord injury (SCI), traumatic brain injury (TBI), and stroke experience a variety of neurologically related deficits across multiple domains of function. The NIH Toolbox for the Assessment of Neurological and Behavioral Function (NIHTB) examines motor, sensation, cognition, and emotional functioning. The purpose of this paper is to establish the validity of the NIHTB in individuals with neurologic conditions. Methods Community-dwelling individuals with SCI (n = 209), TBI (n = 184), or stroke (n = 211) completed the NIHTB. Relative risks for impaired performance were examined relative to a matched control groups. Results The largest group differences were observed on the Motor domain and for the Fluid Cognition measures. All groups were at increased risk for motor impairment relative to normative standards and matched controls. Fluid cognitive abilities varied across groups such that individuals with stroke and TBI performed more poorly than individuals with SCI; increased relative risks for impaired fluid cognition were seen for individuals in the stroke and TBI groups, but not for those in the SCI group. All three neurologic groups performed normally on most measures in the Sensation Battery, although TBI participants evidenced increased risk for impaired odor identification and the stroke group showed more vision difficulties. On the Emotion Battery, participants in all three groups showed comparably poor psychological well-being, social satisfaction, and self-efficacy, whereas the TBI group also evidenced slightly increased negative affect. Conclusions Data provide support for the validity of the NIHTB in individuals with neurologic conditions.

The nature and prevalence of disability in a Ghanaian community as measured by the Language Independent Functional Evaluation.

Introduction The current study uses the Language Independent Functional Evaluation (L.I.F.E.) to evaluate disability in a smaller Ghanaian coastal town to characterize the extent and nature of disability. The L.I.F.E. is a video animated, language free equivalent of the standard 10-item verbal/written Barthel Index functional assessment. Methods Over a four-month period, the L.I.F.E. survey was given to members of the village of Anomabo in a preliminary survey which consisted of recruitment in an un-controlled manner, followed by a systematic, comprehensive survey of three neighborhood clusters. Basic demographics were also collected, along with the observers assessment of disability. Results 541 inhabitants (264 in the preliminary survey and 277 in systematic survey) completed the L.I.F.E. Participants ranged from 7-100 years old (mean age 32.88, s.d. 20.64) and were 55.9% female. In the systematic study, 16.6% of participants had a less than perfect score on the L.I.F.E., indicating some degree of impairment. Significant differences were found between age groups, but not between sexes, the preliminary and systematic survey, and study location (a=.05). Conclusion The L.I.F.E. and this study methodology can be used to measure the prevalence of disability in African communities. Disability in this community was higher than the frequently cited estimate of 10%. African policymakers can use the L.I.F.E. to measure disability and thus more rationally allocate resources for medical rehabilitation.

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

Background: There is a need for patient‐reported outcome measures that capture the impact that motor impairments have on health‐related quality of life in individuals with Huntingtons disease.

Understanding the need for assistance with survey completion in people with Huntington disease

PurposeIn Huntington disease (HD), motor, cognitive, and psychiatric changes can have a detrimental impact on health-related quality of life (HRQOL). The purpose of this paper is to describe the extent and type of assistance needed to complete online HRQOL surveys, and the impact of assistance on HRQOL scores.MethodsA patient-reported outcome measurement system was developed for HD-specific HRQOL. Individuals across the prodromal and diagnosed disease severity spectrum (n = 532) completed surveys by computer, and reported the amount and type of assistance they received.ResultsSome participants (n = 56; 10.5%) did not complete all surveys; this group had larger proportions with late stage disease, racial/ethnic minority status, low education and single marital status, and poorer motor, independence and cognitive function compared to those who completed all surveys (n = 476). Overall, 72% of individuals did not receive assistance, 11% received computer assistance only, and 17% received assistance answering the survey questions. The majority of late stage individuals (78%) received some assistance compared to early stage (29%) and prodromal individuals (< 1%). Those who received assistance had higher proportions with late stage disease, were older, had less education, and had poorer functional and cognitive skills. Before and after adjustment for sociodemographic and clinical characteristics, those who received assistance had poorer scores on some HRQOL outcomes than those who did not receive assistance.ConclusionsComputer-based assessments are feasible for many persons with HD, although other methods may also be needed. Clinicians and researchers should develop strategies to assist people with HD to complete HRQOL surveys.

Evaluating cognition in individuals with Huntington disease: Neuro-QoL cognitive functioning measures

PurposeCognitive functioning impacts health-related quality of life (HRQOL) for individuals with Huntington disease (HD). The Neuro-QoL includes two patient-reported outcome (PRO) measures of cognition—Executive Function (EF) and General Concerns (GC). These measures have not previously been validated for use in HD. The purpose of this analysis is to evaluate the reliability and validity of the Neuro-QoL Cognitive Function measures for use in HD.MethodsFive hundred ten individuals with prodromal or manifest HD completed the Neuro-QoL Cognition measures, two other PRO measures of HRQOL (WHODAS 2.0 and EQ5D), and a depression measure (PROMIS Depression). Measures of functioning The Total Functional Capacity and behavior (Problem Behaviors Assessment) were completed by clinician interview. Objective measures of cognition were obtained using clinician-administered Symbol Digit Modalities Test and the Stroop Test (Word, Color, and Interference). Self-rated, clinician-rated, and objective composite scores were developed. We examined the Neuro-QoL measures for reliability, convergent validity, discriminant validity, and known-groups validity.ResultsExcellent reliabilities (Cronbach’s alphas ≥ 0.94) were found. Convergent validity was supported, with strong relationships between self-reported measures of cognition. Discriminant validity was supported by less robust correlations between self-reported cognition and other constructs. Prodromal participants reported fewer cognitive problems than manifest groups, and early-stage HD participants reported fewer problems than late-stage HD participants.ConclusionsThe Neuro-QoL Cognition measures provide reliable and valid assessments of self-reported cognitive functioning for individuals with HD. Findings support the utility of these measures for assessing self-reported cognition.