Impact of Chorea on Self-care Activity, Employment, and Health-care Resource Use in Patients with Huntington's Disease.

Background: Chorea is recognized as a prototypic motor feature of Huntington's disease (HD), but its effect on health-related quality of life (HRQoL) has not been fully explored. This study describes the impact of chorea on HRQoL in patients with HD. Objective: To determine the impact of HD-related chorea on employment, self-care activities, activities of daily living, and health-care resource utilization (HCRU). Methods: Data were drawn from the Adelphi HD Disease Specific Programme, a real-world point-in-time survey of 144 neurologists and 427 patients in the United States between July and October 2017. HD patients with and without chorea were identified and examined for differences in employment status, reasons for employment changes, self-care activities, and modifications to cope with involuntary movements. Bivariate tests and inverse probability weighted regression adjustment methods were used to determine differences in outcomes between patients with and without chorea. Results: HD patients with (n=287) and without (n=140) chorea were identified. Patients with chorea were less likely to be employed full-time (16.7% vs 25.7%; P<0.04) and more likely to be on long-term sick leave (17.4% vs 5.0%; P<0.01). The onset of motor symptoms in HD-related chorea patients coincided with a change in employment status (42.7% vs 20.8%; P<0.01). Among those still working (n=145), more than two-fifths of patients with chorea required changes to their workplace and required these changes more frequently (45% vs 17%; P<0.001). HD patients with chorea required aid to help them get around significantly more frequently than those without chorea (55% vs 34%; P<0.001). Discussion: These results demonstrate that HD patients with chorea experienced greater negative impact to employment, self-care activities, and HCRU than patients without chorea experienced. These patients were more likely to stop working due to motor, cognitive, and behavioral symptoms; require modifications in the home and workplace; and need more assistance from caregivers than patients without chorea. Conclusions: Patients with HD-related chorea have greater detriments to emotional, interpersonal, and professional functioning that could be improved by reducing chorea.

Tetrabenazine Treatment Patterns and Outcomes for Chorea Associated with Huntington Disease: A Retrospective Chart Review.

BACKGROUND: Huntington disease (HD) is a neurodegenerative disorder characterized by motor impairments (including chorea), along with behavioral, psychiatric, and cognitive symptoms. Tetrabenazine was the first US Food and Drug Administration (FDA)-approved treatment for chorea related to HD. OBJECTIVE: To examine pharmacologic treatment patterns among patients using tetrabenazine, including reasons for treatment initiation, non-initiation, dose adjustments, and discontinuation, and to quantify the burden of chorea based on healthcare resource utilization. METHODS: In this retrospective patient chart review, neurologists were recruited from the Medefield (http://www.medefield.com) opt-in panel, and selected ≤5 medical charts based on the criteria provided and abstracted data on demographics, disease history, healthcare resource use, and treatment patterns. RESULTS: 138 neurologists participated and 512 HD patient charts were reviewed. Among these patients, 26.4% did not initiate tetrabenazine. Most HD patients (66.5%) received a tetrabenazine dose ≤50 mg. The most common reasons for stopping upward titration were optimal chorea control (55.5%), intolerability of higher doses (31.2%), and reaching the maximum recommended dosage despite suboptimal chorea control (11.4%). Chorea severity and non-persistence to tetrabenazine were associated with increased emergency room visits, hospitalizations, and days hospitalized. CONCLUSIONS: Although tetrabenazine was the sole FDA-approved treatment for HD chorea until April 2017, more than one-quarter of respondents never initiated therapy. Tetrabenazine dosing was lower than predicted, and many patients experienced adverse symptoms of intolerability at high doses. New safer and more tolerable treatment options, such as deutetrabenazine, may improve treatment outcomes and reduce healthcare resource use.

Understanding How Chorea Affects Health-Related Quality of Life in Huntington Disease: An Online Survey of Patients and Caregivers in the United States.

BACKGROUND: Chorea is the hallmark motor feature of Huntington disease (HD) and can negatively impact daily functioning and health-related quality of life (HRQoL). OBJECTIVE: The objective of this study was to evaluate how chorea impacts HRQoL and overall functioning among HD patients participating on the PatientsLikeMe website ( www.PatientsLikeMe.com ). METHODS: A survey was provided to HD participants and/or their caregivers via PatientsLikeMe (9 February 2017-22 March 2017), comprising multiple-choice and open-ended questions designed to assess how chorea impacts HRQoL and overall functioning, and the importance of treating chorea. The HDQLIFE measurement system was used to evaluate patient-reported outcomes of chorea and compare Anxiety and Stigma scores in participants with high chorea versus those with low chorea [HDQLIFE Chorea scores ≥ 60 (n = 45) vs. < 60 (n = 38)]. RESULTS: A total of 115 participants (n = 35 caregivers; n = 80 individuals with HD) were included in this study. Among those experiencing chorea (n = 83, 74% of respondents), 66% indicated it was 'Very Important' to manage chorea; however, only 47% agreed that their current medication regimen helped manage their movements. In general, respondents reported that chorea negatively affected HRQoL [HDQLIFE Chorea mean score (standard deviation): 59.3 (6.1)]. Consistent with this, significantly higher Anxiety (P = 0.0423) and stigma (P < 0.0001) scores were observed among respondents with high chorea than in those with low chorea. CONCLUSIONS: These results highlight the negative impact of chorea on HRQoL and overall functioning in individuals with HD. Better chorea treatment options are needed to successfully manage symptoms and to help improve HRQoL in these individuals, and patient experiences of anxiety and stigma should be considered in treatment plans.

Physician perceptions of pharmacologic treatment options for chorea associated with Huntington disease in the United States.

OBJECTIVE: To survey neurologists and obtain clinical perceptions of tetrabenazine for the treatment of chorea in patients with Huntington disease (HD). METHODS: Board-certified/board-eligible neurologists, in practice for ≥5 years, who had treated treat ≥3 HD patients in the past 2 years, were recruited from an online physician panel to participate in a cross-sectional, web-based survey. Respondents provided information about themselves, their practice, approaches to HD chorea management and perceptions of available treatments. RESULTS: Two hundred neurologists responded to the survey. Based on clinician responses, the most common reasons to treat chorea are impairment in activities of daily living (54%) and quality of life (41%). Although tetrabenazine was the only approved treatment for chorea in HD patients at the time of this analysis, it was only prescribed to ∼50% of patients with HD-related chorea. More than half of physicians perceive tetrabenazine as having minimal or no effectiveness in improving chorea. More than 40% of physicians consider tetrabenazine to be a non-optimal treatment, and 51% of physicians agree that they are unable to titrate to efficacious doses due to adverse side effects or tolerability concerns. Physicians report that side effects leading to dose interruptions (33%) and reductions (30%) occur in their patients "often" or "almost always". The most common side effects that led to insufficient dosing and disruptions in titration were sedation and somnolence (41%), depression (24%) and anxiety (22%). CONCLUSIONS: Many physicians who treat HD-related chorea report that tolerability issues with tetrabenazine impact their ability to effectively use tetrabenazine in their clinical practice.

Retrospective Analysis of Healthcare Resource Use, Treatment Patterns, and Treatment-related Events in Patients with Huntington's Disease-associated Chorea Initiated on Tetrabenazine.

BACKGROUND: Huntington's disease (HD) is a multifaceted neurodegenerative disorder characterized by involuntary movements, specifically chorea, as well as behavioral and psychiatric disturbance, and cognitive dysfunction. Tetrabenazine was the first approved treatment for chorea, although tolerability concerns exist. OBJECTIVES: To characterize demographic and clinical characteristics of HD patients with chorea based on tetrabenazine use and examine treatment persistence with tetrabenazine in a real-world setting. METHODS: Patients with a claim for HD-associated chorea (ICD-9-CM code 333.4) between 1/1/08 and 9/30/15 were selected from the MarketScan® Commercial and Medicare Supplemental databases. The first diagnosis date during the study period was considered the index date, with ≥6 months of continuous medical and prescription coverage before and after the index date. Treatment persistence was defined as the number of days from initiation to discontinuation or end of follow-up period. Discontinuation was defined as a gap in therapy of ≥60 days. RESULTS: 1644 patients met selection criteria (mean age ± standard deviation: 54.5 ± 15.5), of which 151 (9.2%) were treated with tetrabenazine during the study period. The average (median) daily dose of tetrabenazine during the treatment period was 45.5 (42.3) mg/day. A total of 41.8% (59/141) of HD patients who initiated tetrabenazine experienced a ≥60-day gap in tetrabenazine therapy, with a median time to discontinuation of 293.5 days. During the 6-month post-index period after HD diagnosis, HD patients incurred higher all-cause healthcare costs ($20 204) vs the 6-month pre-index period ($6057), driven by higher hospitalization and pharmacy costs. CONCLUSIONS: A small percentage of HD patients with chorea were treated with tetrabenazine and discontinuation rates were high among those receiving treatment, with a median time to discontinuation of 9 months.

Patient and parent preferences for immunoglobulin treatments: a conjoint analysis.

OBJECTIVES: The purpose was to quantify patient and parent preferences for administration attributes of immunoglobulin (IG) treatments; and determine which administration attributes were most important to users of IG treatment and whether patients and parents have similar preferences for administration attributes. METHODS: US adult patients and parents of children with a self-reported physician diagnosis of a primary immunodeficiency disorder completed a best-practice web-enabled choice-format conjoint survey that presented a series of 12 choice questions, each including a pair of hypothetical IG-treatment profiles. After reviewing current therapies, each profile was defined by mode of administration, frequency, location, number of needle sticks, and treatment duration. Before answering the choice questions, respondents were told to assume all treatments worked equally well. Choice questions were based on a D-efficient experimental design. Preference weights for attribute levels were estimated using random-parameters logit for each sample (adult patients and parents). Tests were performed to determine potential interactions among the administration attributes. All respondents provided online informed consent. RESULTS: In total, 252 patients and 66 parents completed the choice questions appropriately. Overall, both groups preferred a home setting, monthly frequency, fewer needle sticks, and shorter treatment durations of IG treatment relative to alternative choices (p<0.05). Mode of administration was the least important attribute to both samples; however, parents strongly preferred self-administration to an appointment with a healthcare professional (p<0.05), whereas patients slightly preferred self-administration but were indifferent to the two modes. LIMITATIONS: Respondents evaluate hypothetical treatments and differences can arise between stated and actual choices. CONCLUSIONS: Considering the hypothetical treatments evaluated, IG treatments that provide the option of a home setting, monthly frequency, fewer needle sticks, and shorter treatment durations may address the needs of both patients and parents. Patients and parents have different preferences for administration attributes of IG treatments.

Pain documentation and predictors of analgesic prescribing for elderly patients during emergency department visits.

CONTEXT: Inappropriate pain documentation is likely to be an important contributor to the poor management of pain in elderly patients in the emergency department (ED). Failure to assess pain limits ability to treat pain. OBJECTIVES: The objectives of this study were to examine the relationship between visit characteristics of elderly patients and pain score documentation in the ED, and to determine predictors of analgesic use in the ED. METHODS: This was a cross-sectional analysis of documented ED visits by elderly patients from the National Hospital Ambulatory Medical Care Survey (2003-2006). The study included 5661 ED visits by patients aged 65 years and older, representing an estimated 18 million ED visits during the four-year study period. Univariate logistic regression was used to analyze associations among independent variables and documentation of pain. Multivariate logistic regression was used to determine whether nonopioid and opioid analgesic prescribing disparities existed and were associated with pain level. RESULTS: Pain score documentation was found to be suboptimal in the elderly population in this study, with only 75% of visits having documented pain scores. Older age, self-pay, patients residing in the Western region of the United States, and emergent ED visits were associated with decreased pain score documentation. Documentation of pain score was associated with increased odds of an analgesic prescription and opioid analgesic prescription. Odds of prescribing an opioid increased significantly with increasing level of pain severity. CONCLUSION: ED pain score documentation is suboptimal in the elderly population. Disparity in the use of analgesic prescriptions and opioid analgesics exists and may result in patients not receiving analgesics. Improving pain assessment and documentation, changes in attitude toward analgesic prescribing, and recognition of ethnic, racial, and age differences in patients with pain have the potential to contribute to effective management of pain in the ED.

The variation in patient care rendered by physicians and nonphysician clinicians in an ambulatory care setting.

This study compared three medical care services provided by a physician, a nonphysician clinician (NPC), and an NPC with a physician in ambulatory settings while measuring the trend in patients with essential hypertension and allergic rhinitis seen by these clinicians for the years 1999 through 2006. Multivariate analysis of variance and Z-test were used to analyze the data, while post hoc comparisons were performed using Scheffe test. There was a significant increase in the proportion of patients seen by an NPC with a physician for both diagnoses compared with the other two types of clinicians. The combination type prescribed significantly more medications than the other types, while there was moderate to substantial agreement in the choice of medications. Clinician type emerged as a prominent variable to explain the differences in how counseling services are provided, medications are prescribed, and diagnostic and screening services are ordered for patients with essential hypertension or allergic rhinitis in ambulatory settings.