"You Take This Day by Day, Come What May": A Qualitative Study of the Psychosocial Impacts of Living with Duchenne Muscular Dystrophy.

INTRODUCTION: Studies have reported health-related quality-of-life impacts of Duchenne muscular dystrophy (DMD); however, further research is needed to understand how those with DMD experience their condition and how psychosocial impacts evolve over time in response to disease progression. This qualitative study explores the social and emotional implications of key transitions, challenges and adaptations throughout the disease course from the perspective of patients and family caregivers. METHODS: Semi-structured interviews were conducted with men and boys with DMD, and/or their caregivers, in the USA. Thematic analysis was used to examine patterns in data collected across the interviews. RESULTS: Nineteen participants were included. Three major themes were identified: (1) barriers to participation are multifaceted; (2) an emotional journey shaped by 'inevitable progression;' (3) family provides critical tangible and emotional support. This study illustrates that psychosocial impacts of DMD are shaped by knowledge of the condition's natural history alongside other factors including the extent of social barriers, personal growth and adaptation, and family support. CONCLUSIONS: Findings provide insight into the strength and resilience with which individuals and their families respond to daily challenges and major clinical milestones and highlight the relative importance of loss of upper limb function as a transition in DMD affecting health-related quality-of-life.

Comparing Preferences for Disease Profiles: A Discrete Choice Experiment from a US Societal Perspective.

OBJECTIVES: There is increasing interest in expanding the elements of value to be considered when making health policy decisions. To help inform value frameworks, this study quantified preferences for disease attributes in a general public sample and examined which combination of attributes (disease profiles) are considered most important for research and treatment. METHODS: A discrete choice experiment (DCE) was conducted in a US general population sample, recruited through online consumer panels. Respondents were asked to select one of a set of health conditions they believed to be most important, characterized by attributes defined by a previous qualitative study: onset age; cause of disease; life expectancy; caregiver requirement; symptom burden (characterized by the Health Utilities Index with varying levels of ambulation independence, dexterity limitations, and degree of pain and discomfort); and disease prevalence. A fractional factorial DCE design was implemented using R, and 60 choice sets were generated (separated into blocks of 10 per participant). Data were analyzed using a mixed-logit regression model, and results used to assess the likelihood of preferring disease profiles. Based on individual attribute preferences, overall preferences for disease profiles, including a profile aligned with Duchenne muscular dystrophy (DMD), were compared. RESULTS: Fifty-two percent of respondents (n = 537) were female, and 70.6% were aged 18-54 years. Attributes considered most important were those related to life expectancy (odds ratio [OR], 95% confidence interval [CI] 1.88 [1.56-2.27] for a 50% reduction in remaining life expectancy vs no impact), and symptom burden (OR [95% CI] 1.84 [1.47-2.31] for severe vs mild burden). Greater importance was also found for pediatric onset, caregiver requirement, and diseases affecting more people. As an example of disease profile preferences, a DMD-like pediatric inherited disease with 50% reduction in life expectancy, extensive caregiver requirement, severe symptom burden, and 1:5000 prevalence had 2.37-fold higher odds of being selected as important versus an equivalent disease with adult onset and no life expectancy reduction. CONCLUSIONS: Of disease attributes included in this DCE, respondents valued higher prevalence of disease, life expectancy and symptom burden as most important for prioritizing research and treatment. Based on expressed attribute preferences, a case study of an inherited pediatric disease involving substantial reductions to length and quality of life and requiring caregiver support has relatively high odds of being identified as important compared to diseases reflecting differing attribute profiles. These findings can help inform expansions of value frameworks by identifying important attributes from the societal perspective.

Estimating health state utilities in Duchenne muscular dystrophy using the health utilities index and EQ-5D-5L.

BACKGROUND: The progression of Duchenne muscular dystrophy (DMD) is characterized by loss of ambulation, respiratory insufficiency, cardiomyopathy, and early mortality. DMD profoundly impacts health-related quality-of-life (HRQoL). However, few health state utility data exist; published utilities tend to be derived from small samples for a limited number of health states and are often based on caregiver-reported patient health status. This study estimated utility values for varied clinical and functional health states in DMD, based on patient-reported health status. METHODS: Individuals with DMD in the US aged 12-40 years completed the EQ-5D (5-level) and Health Utilities Index (HUI) preference-based instruments. Based on responses to a clinical questionnaire, participants self-classified into functional health states according to level of lower and upper limb function, use of respiratory support, and presence of cardiomyopathy. Mean [standard deviation (SD)] utility and EQ-5D visual analogue scale (VAS) scores were estimated according to health state; and median (interquartile range) attribute levels calculated to understand which domains of health are most severely affected in DMD. RESULTS: Of 63 males with DMD, mean (SD) age was 19.8 (6.1) years and 11 (17.5%) were ambulatory. Mean (SD) utility values were 0.92 (0.08; HUI2), 0.84 (0.20; HUI3), and 0.84 (0.13; EQ-5D) for ambulatory patients without cardiomyopathy (n = 10). For non-ambulatory patients with moderately impaired upper limb function, night and daytime ventilation without cardiomyopathy, mean (SD) utilities were 0.49 (0.07) for the HUI2, 0.16 (0.15) for the HUI3 and 025 (0.14) for the EQ-5D. Mean (SD) VAS scores for the same health states were 91 (9) and 83 (21), respectively. In addition to impairments in mobility/ambulation, and self-care, attributes like usual activities and pain also showed notable effects of DMD. CONCLUSIONS: In DMD, although a relationship between disease progression and HRQoL is observed, there is large variability in utility within functional health states, and across instruments. Utility values for less severe non-ambulatory health states described by level of upper limb function are novel. These utility values, derived based on direct patient feedback rather than from caregiver report, are relevant to individuals of varying functional statuses and augment scarce DMD-specific utility data.

Exploring the relationship between North Star Ambulatory Assessment and Health Utilities Index scores in Duchenne muscular dystrophy.

BACKGROUND: The North Star Ambulatory Assessment (NSAA) documents motor performance in ambulatory individuals with Duchenne muscular dystrophy (DMD). Health Utilities Index (HUI) scores, reflecting preferences for health-related quality-of-life (HRQoL) implications of health states, are commonly estimated within trials. This study sought to characterize the relationship between the NSAA score and utility in DMD. METHODS: Family members serving as proxy respondents for placebo-treated ambulatory individuals with DMD (NCT01254019; BioMarin Pharmaceuticals Inc) completed the HUI and the NSAA (score range, 0-34). Mean change over time on these measures was estimated, and the correlation between changes in NSAA score and a) HUI utility; b) HUI3 ambulation and HUI2 mobility attribute scores, over 48 weeks was calculated. RESULTS: Baseline mean (range) age was 8.0 years (5-16; n = 61) and mean (standard deviation [SD]) scores were 0.87 (0.13; HUI2), 0.82 (0.19; HUI3), and 21.0 (8.1; NSAA). Mean (SD) change over 48 weeks was -0.05 (0.14; HUI2), -0.06 (0.19; HUI3), and -2.9 (4.7; NSAA). Weak positive correlations were observed between baseline NSAA score and HUI utility (HUI2: r = 0.29; HUI3: r = 0.17) and for change over 48 weeks (HUI2: r = 0.16; HUI3: r = 0.15). Stronger correlations were observed between change in NSAA score and the HUI3 ambulation (r = 0.41) and HUI2 mobility (r = 0.41) attributes. CONCLUSIONS: Among ambulatory individuals with DMD, NSAA score is weakly correlated with HUI utility, suggesting that motor performance alone does not fully explain HRQoL. Stronger relationships were observed between HUI ambulation and mobility attributes, and NSAA. Although unidimensional measures like the NSAA are informative for documenting disease-specific health impacts, they may not correlate well with measures of overall health status; requiring use in conjunction with other patient-reported and preference-based outcomes.

Health Utilities Index Mark 3 scores for children and youth: Population norms for Canada based on cycles 5 (2016 and 2017) and 6 (2018 and 2019) of the Canadian Health Measures Survey.

Background: Utility scores are an important tool for evaluating health-related quality of life. Utility score norms have been published for Canadian adults, but no nationally representative utility score norms are available for children and youth. Data and methods: Health Utilities Index Mark 3 (HUI3) data from two recent cycles of the Canadian Health Measures Survey (i.e., 2016 and 2017, and 2018 and 2019) were used to provide utility score norms for children aged 6 to 11 years and adolescents aged 12 to 17 years. Children younger than 14 years answered the HUI3 under the supervision of an adult, while older children answered without supervision. Utility scores were reported as a weighted average (95% confidence intervals [CIs]) and median values (interquartile range). Utility scores were stratified by sociodemographic and medical characteristics of the child or adolescent. Regression analyses were used to identify predictors of utility scores. All results were weighted using sampling weights provided by Statistics Canada. Results: Among the 2,297,136 children aged 6 to 11 years and the 2,329,185 adolescents aged 12 to 17 years in the weighted sample, the average utility scores were 0.95 (95% CI: 0.94 to 0.95) and 0.89 (95% CI: 0.87 to 0.90), respectively. Approximately 60% of the children and 34% of the adolescents had a utility score of 1.00. Analyses identified several factors associated with utility scores (e.g., age, chronic condition and income levels), although differences were observed between children and adolescents. Interpretation: This study provides utility score estimates based on a nationally representative sample of Canadian children and youth. Further research examining the determinants of utility scores of children and adolescents is warranted.

Generic Health-Related Quality of Life Utility Measure for Preschool Children (Health Utilities Preschool): Design, Development, and Properties.

OBJECTIVES: Health Utilities Preschool (HuPS) was developed to fill the need for a generic preference-based measure (GPM) applicable in early childhood. A GPM has all the properties for higher-order summary measures, such as quality-adjusted life-years, required to inform important policy decisions regarding health and healthcare services. METHODS: Development was in accordance with published standards for a GPM, statistical procedures, and modeling. HuPS incorporates key components of 2 existing measurement systems: Health Status Classification System for Preschool Children and Health Utilities Index Mark 3 (HUI3). The study included a series of 4 measurement surveys: definitional, adaptational, quantificational, and evaluational health-related quality of life (HRQL). HuPS measurements were evaluated for reliability, validity, interpretability, and acceptability. RESULTS: Definitional measurements identified 8 Health Status Classification System for Preschool Children attributes in common with HUI3 (vision, hearing, speech, ambulation, dexterity, emotion, cognition, and pain and discomfort), making the HUI3 scoring equation commensurate with HuPS health states. Adaptational measurements informed the content of attribute-level descriptions (n = 35). Quantificational measurements determined level scoring coefficients. HRQL scoring inter-rater reliability (intraclass correlation coefficient = 0.79) was excellent. Continuity of HRQL scoring with HUI3 was reliable (intraclass correlation coefficient = 0.80, P < .001) and valid (mean absolute difference = 0.016, P = .396). CONCLUSIONS: HuPS is an acceptable, reliable, and valid GPM. HRQL scoring is continuous with HUI3. Continuity expands the applicability of GPM (HUI3) scoring to include subjects as young as 2 years of age. Widespread applications of HuPS would inform important health policy and management decisions as HUI3 does for older subjects.

Improving the Hearing Status Discrimination of the Health Utilities Index, Mark 3: Design of the Hearing Status Classification System.

OBJECTIVE: Redesign the health status classification system of the Health Utilities Index, Mark 3 (HUI-3) "hearing" attribute to improve its discrimination of hearing-impaired health states. STUDY DESIGN: Domain and item selection from a previously generated item set guided by a domain and item importance survey, structural independence, and cognitive interviews with patients. SETTING: Tertiary referral center. PARTICIPANTS: Patients with a range of hearing loss severities, etiologies, and treatment experiences participated in the domain and item importance survey (n = 108) and hour-long cognitive interviews (n = 10). MAIN OUTCOME AND MEASURES: Subattributes and levels for the novel Hearing attribute. Domain and item importance was scored on a seven-point Likert scale (1, not at all important; 7, extremely important). RESULTS: Mean domain importance was highest for "speech in noise" (6.21; 95% confidence interval, 5.98-6.43) and lowest for "benefits of hearing loss" (3.46; 95% confidence interval, 3.03-3.89). Domains with moderate or greater ( r ≥ 0.5) domain importance Pearson correlation or construct overlap that interfered with structural independence were combined into single subattributes. Iterative adjustments to instructions, items, and phrasing were guided by cognitive interviews to derive the final instrument with seven subattributes: speech, environmental sounds, localization, listening effort, tinnitus, music, and assistive devices. The novel hearing attribute defines 25,920 unique hearing states. CONCLUSION: The novel HUI-hearing is part of a comprehensive health utility instrument designed for individuals with hearing loss. Pending derivation of a hearing single attribute utility function and evaluation of measurement properties, our innovative approach could be used to improve health utility measurement for impairments described by any of the other HUI-3 attributes.

Factors associated with the health-related quality of life among people with Duchenne muscular dystrophy: a study using the Health Utilities Index (HUI).

BACKGROUND: Data on health state utility in Duchenne muscular dystrophy (DMD) are few. This study estimated mean utility values by age, ambulatory status and over time, and investigated which aspects of health-related quality-of-life (HRQoL) are most strongly associated with utility in DMD. METHODS: Data from placebo-treated ambulant boys with DMD with exon 51 skip amenable mutations, (NCT01254019), were included. Ambulatory function assessments were conducted at baseline and every 12 weeks for the trial duration. Family member proxies completed the Health Utility Index (HUI) at baseline, 24 and 48 weeks; and HUI3 and HUI2 utility values were summarized. Changes in HUI attribute level over time, and predictors of changes in utility, were explored. RESULTS: Sixty-one boys (mean [range] age of 8.0 [5-16] years) were included in the analysis. Mean baseline utilities were 0.82 (HUI3) and 0.87 (HUI2); and utilities were 0.35 (HUI3) and 0.55 (HUI2) after loss of ambulation (LOA, where applicable). Over the follow-up period mean utility declined more among the older versus younger boys. Pain accounted for the highest proportion of variability (42%) in change in HUI3 utility from baseline to week 48, while for HUI2, self-care (39%) did. After LOA, change in ambulation levels explained 88% of the decline in mean HUI3 utility and change in mobility levels explained 66% of the decline in mean HUI2 utility. CONCLUSIONS: Utility values among this sample were higher than previously published estimates. In younger boys utility remained relatively stable, but older boys and those losing ambulation experienced important declines over follow-up.

Societal perspectives on disease and treatment attributes characterizing rare diseases: a qualitative study from the United States.

PURPOSE: Under a societal perspective, disease and treatment attributes that the general public deem important should be considered within value frameworks. The objective was to investigate how members of the general public value attributes beyond health gains and healthcare system expenditures; and better understand their perspectives regarding the importance of attributes typically characterizing rare genetic diseases like Duchenne muscular dystrophy (DMD). METHODS: Qualitative interviews were conducted to elicit feedback on the importance of disease and treatment attributes from general public participants from three US cities. Participants ranked attributes (scale, 1-10) in terms of importance for future research, reported their rationale for ranking, and provided feedback specific to rare diseases. Interview transcripts were coded using NVivo for thematic analysis. RESULTS: The 33 participants (median age, 51 years; 48.5% male) ranked disease severity (mean [median] ranking, 8.7 [9.0]), treatment availability (8.7 [9.0]), and impact on life expectancy (8.4 [9.0]), as most important. The impact on the family, need for equity, and intrinsic value of life were frequently provided rationales. While rare disease as an attribute received a relatively low ranking (6.1 [7.0]), 88% of participants prioritized disease profiles including attributes of severity, health related quality of life (HRQoL) impact, limited lifespan and young age at onset. CONCLUSION: Attributes including disease severity, impact on life expectancy and HRQoL, and treatment availability were all highly important to members of the general public. These findings support the growing evidence regarding the importance of expanding value assessments to include attributes considered important from a societal perspective.

Parent-reported health status of preterm survivors in a Canadian cohort.

OBJECTIVES: Health status (HS)/ health-related quality of life measures, completed by self or proxy, are important outcome indicators. Most HS literature on children born preterm includes adolescents and adults with limited data at preschool age. This study aimed to describe parent-reported HS in a large national cohort of extreme preterm children at preschool age and to identify clinical and sociodemographic variables associated with HS. METHODS: Infants born before 29 weeks' gestation between 2009 and 2011 were enrolled in a prospective longitudinal national cohort study through the Canadian Neonatal Network (CNN) and the Canadian Neonatal Follow-Up Network (CNFUN). HS, at 36 months' corrected age (CA), was measured with the Health Status Classification System for Pre-School Children tool completed by parents. Information about HS predictors was extracted from the CNN and CNFUN databases. RESULTS: Of 811 children included, there were 79, 309 and 423 participants in 23-24, 25-26 and 27-28 weeks' gestational age groups, respectively. At 36 months' CA, 78% had a parent-reported health concern, mild in >50% and severe in 7%. Most affected HS attributes were speech (52.1%) and self-care (41.4%). Independent predictors of HS included substance use during pregnancy, infant male sex, Score for Neonatal Acute Physiology-II, bronchopulmonary dysplasia, severe retinopathy of prematurity, caregiver employment and single caregiver. CONCLUSION: Most parents expressed no or mild health concerns for their children at 36 months' CA. Factors associated with health concerns included initial severity of illness, complications of prematurity and social factors.

Correction: Associations between continuity of primary and specialty physician care and use of hospital-based care among community-dwelling older adults with complex care needs.

[This corrects the article DOI: 10.1371/journal.pone.0234205.].

Construct Validity of the Health Utilities Index Mark 2 and Mark 3 to Measure Health-Related Quality of Life of Residents in Long-Term Care Facilities.

BACKGROUND AND PURPOSE: The purpose is to evaluate the construct validity of two generic health measures, the Health Utilities Index Mark 2 (HUI2) and Mark 3 (HUI3) in residents of long-term care (LTC) facilities, using a convergent/divergent validity approach, with the Functional Independence Measure (FIM), Quality of Life-Alzheimer's Disease (QOL-AD) and Resident Assessment Instrument (RAI) 2.0 as comparators. METHODS: Health status of 130 residents with dementia residing in one of seven LTC facilities was evaluated by their Healthcare Aides who were their primary care assistants. A priori hypotheses on the magnitude and direction of the correlations were formulated by two clinician/researchers and a researcher familiar with the measures and this population. Predicted and observed correlations were compared. RESULTS: Mean overall HUI2 (0.48, SD 0.16) and HUI3 scores (0.31, SD 0.27) were indicative of severe disability. Of the 208 a priori hypotheses, 39.9% (n = 83) matched the observed correlations, 29.8% were underestimated and 19.7% were overestimated by one category. CONCLUSIONS: Findings support the use of the HUI2 and HUI3 in measuring health-related quality of life in dementia-related research to complement disease-specific measures.

Canadian French translation and linguistic validation of the health-related quality of life utility measure for pre-school children.

BACKGROUND: There is a need to perform a Canadian French translation and linguistic validation of the health-related quality of life utility measure for pre-school children (HuPS) conceptually equivalent to the original Canadian English version. RESEARCH DESIGN AND METHODS: The translation process consisted of forward and back translations. The linguistic validation was performed with the parents of preschool children during face-to-face cognitive debriefing interviews. The whole process was done in accordance with academic standards and the guidance of the Food and Drug Administration (FDA) for patient-reported outcome instruments. RESULTS: The results of back translations indicated that 89% of the sentences were identical or almost identical to the original English-language wording. The review of the back translations led to a change in 13 sentences out of 91 from the reconciled forward translation, while the linguistic validation process with 13 parents led to 14 additional changes. Preliminary reliability validation results indicate a Cronbach's alpha of 0.73. CONCLUSION: The translation and linguistic testing processes were successful in creating a valid HuPS in Canadian French (HuPS-CF). This translation should be the subject of reliability and validity studies in a wide variety of clinical and general populations before to use in research projects.

A Scoring Algorithm for Deriving Utility Values from the Neuro-QoL for Patients with Multiple Sclerosis.

INTRODUCTION: The Neuro-QoL is a standardized approach to assessing health-related quality of life in people with neurological conditions, including multiple sclerosis (MS). Item banks were developed with item response theory (IRT) methodology so items are calibrated along a continuum of each construct. The purpose of this study was to develop a preference-based scoring algorithm for the Neuro-QoL to derive utilities that could be used in economic modeling. METHODS: With input from neurologists, 6 Neuro-QoL domains were selected based on relevance to MS and used to define health states for a utility elicitation study in the United Kingdom. General population participants and individuals with MS valued the health states and completed questionnaires (including Neuro-QoL short forms). The Neuro-QoL Utility Scoring System (NQU) was derived based on multi-attribute utility theory using data from the general population sample. Single-attribute disutility functions for 6 Neuro-QoL domains were estimated using isotonic regression with linear interpolation and then combined with a multiplicative model. NQU validity was assessed using MS participant data. RESULTS: Interviews were completed with 203 general population participants (50.2% female; mean age = 45.0 years) and 62 participants with MS (62.9% female; mean age = 46.1 years). Mean (SD) NQU scores were 0.94 (0.06) and 0.82 (0.13) for the general population and MS samples, respectively. The NQU demonstrated known-groups validity by differentiating among subgroups categorized based on level of disability. The NQU demonstrated convergent validity via correlations with generic measures (0.66 and 0.63 with EQ-5D-5L and Health Utilities Index Mark 3, respectively; both P < 0.001). DISCUSSION: With the NQU, utilities can be derived from any MS treatment group, subgroup, or patient sample who completes items from 6 Neuro-QoL domains. Because the Neuro-QoL is frequently used with MS patients, the NQU greatly expands the options for quantifying outcomes in cost-utility analyses conducted to inform allocation of resources for MS treatment.

Effect of Access to After-Hours Primary Care on the Association Between Home Nursing Visits and Same-Day Emergency Department Use.

PURPOSE: Previous work has demonstrated that home care patients have an increased risk of visiting the emergency department after a home nursing visit on the same day. We investigated whether this association is modified by greater access to after-hours primary care. METHODS: We conducted a population-based case-crossover study of home care patients in Ontario, Canada in 2014-2016. Emergency department visits after 5:00 pm were selected as case periods and matched, within the same patient, to control periods within the previous week. The association between home nursing visits and same-day emergency department visits was estimated with conditional logistic regression. Access to after-hours primary care, measured on the patient and practice level, was tested for effect modification using an interaction term approach. Analysis was performed separately for all emergency department visits and a less urgent subset not admitted to hospital. RESULTS: A total of 11,840 patients contributed cases to the analysis. Patients with a history of after-hours primary care use had a smaller increased risk of a same-day after-hours emergency department visit (OR = 1.18; 95% CI, 1.06-1.30) compared with patients with no after-hours care (OR = 1.31; 95% CI, 1.25-1.39). The modifying effect was stronger among emergency department visits not admitted to hospital (OR = 1.11; 95% CI, 0.97-1.28 vs OR = 1.41; 95% CI, 1.31-1.51). CONCLUSION: Greater access to after-hours primary care reduced the risk of less-urgent emergency department use associated with home nursing visits. These findings suggest increasing access to after-hours primary care could prevent some less-urgent emergency department visits.

Associations between continuity of primary and specialty physician care and use of hospital-based care among community-dwelling older adults with complex care needs.

OBJECTIVE: While research suggests that higher continuity of primary and specialty physician care can improve patient outcomes, their effects have rarely been examined and compared concurrently. We investigated associations between continuity of primary and specialty physician care and emergency department visits and hospital admissions among community-dwelling older adults with complex care needs. METHODS: We conducted a retrospective cohort study of home care patients in Ontario, Canada, from October 2014 to September 2016. We measured continuity of primary and specialty physician care over the two years prior to a home care assessment and categorized them into low, medium, and high groups using terciles of the distribution. We used Cox regression models to concurrently test the associations between continuity of primary and specialty care and risk of an emergency department visit and hospital admission within six months of assessment, controlling for potential confounders. We examined interactions between continuity of care and count of chronic conditions, count of physician specialties seen, functional impairment, and cognitive impairment. RESULTS: Of 178,686 participants, 49% had an emergency department visit during follow-up and 27% had a hospital admission. High vs. low continuity of primary care was associated with a reduced risk of an emergency department visit (HR = 0.90 (0.89-0.92)) as was continuity of specialty care (HR = 0.93 (0.91-0.95)). High vs. low continuity of primary care was associated also with a reduced risk of a hospital admission (HR = 0.94 (0.92-0.96)) as was continuity of specialty care (HR = 0.92 (0.90-0.94)). The effect of continuity of specialty care was moderately stronger among patients who saw four or more physician specialties. CONCLUSION: Higher continuity of primary physician and specialty physician care had independent, protective effects of similar magnitude against emergency department use and hospital admissions. Improving continuity of specialty care should be a priority alongside improving continuity of primary care in complex, older adult populations with significant specialist use.

Physician Home Visit Patterns and Hospital Use Among Older Adults with Functional Impairments.

BACKGROUND: Home-based primary care has been associated with reductions in hospital use among homebound older adults, but population-based studies on the general home visit patterns of primary care physicians are lacking. OBJECTIVE: We examined the association between the provision of home visits by primary care physicians and subsequent use of hospital-based care among their older adult patients with extensive functional impairments. DESIGN: Population-based retrospective cohort study. SETTING: The setting was Ontario, Canada, from October 2014 to September 2016. PARTICIPANTS: Older adults (aged ≥65 years) with extensive functional impairments receiving publicly funded home care. MEASUREMENTS: We measured the provision of home visits by a patient's most responsible primary care physician during the year before a comprehensive home care assessment. Physician home visit patterns were measured as the proportion of the total outpatient visits in a year that were home visits, categorized with quartiles. Multivariable, multilevel negative binomial regression models examined the associations between physician-level home visit provision and patient emergency department visits and hospital admissions over the 6 months following the home care assessment. RESULTS: There were 49,613 patients in the cohort who were linked to 8,096 unique primary care physicians. A total of 69.1% of physicians provided at least one home visit in a year, with the median proportion of home visits to total visits ranging from 0.057% to 3.19% across quartiles. Patients whose physicians were in the highest home visit provision quartile had lower rates of emergency department visits (incidence rate ratio [IRR] = 0.93; 95% confidence interval [CI] = 0.90-0.96) and hospital admissions (IRR = 0.89; 95% CI = 0.85-0.93) compared with patients whose physician did not do home visits. CONCLUSION: Home care patients with extensive functional impairments whose physicians provided higher levels of home visits had fewer emergency department visits and hospital admissions. Expanding home visits by primary care physicians could reduce hospital use by older adults living with functional impairments in the community.

Health-Related Quality of Life Changes Associated With Hearing Loss.

Importance: Utility is a single-value, preference-based measure of health-related quality of life that represents the desirability of a health state relative to being dead or in perfect health. Clinical, funding, and policy decisions rely on measured changes in utility. The benefit of hearing loss treatments may be underestimated because existing utility measures fail to capture important changes in quality of life associated with hearing loss. Objective: To develop a comprehensive profile of items that describe how quality of life is associated with hearing loss and its treatments that can be used to generate hearing-related quality of life measures, including a novel utility measure. Design, Setting, and Participants: This qualitative study, performed from August 1, 2018, to August 1, 2019, in tertiary referral centers, comprised a systematic literature review, focus groups, and semistructured interviews. The systematic review evaluated studies published from 1982 to August 1, 2018. Focus groups included 8 clinical experts experienced in the measurement, diagnosis, treatment, and rehabilitation of hearing loss. Semistructured interviews included 26 adults with hearing loss recruited from an institutional data set and outpatient hearing aid and otology clinics using stratified convenience sampling to include individuals of diverse ages, urban and rural residency, causes of hearing loss, severity of hearing loss, and treatment experience. Main Outcomes and Measures: A set of items and subdomains that collectively describe the association of hearing loss with health-related quality of life. Results: The literature search yielded 2779 articles from the MEDLINE, Embase, Cochrane, PsycINFO, and CINAHL databases. Forty-five studies including 1036 individuals (age range, 18-84 years) were included. The focus group included 4 audiologists and 4 otologists. Hour-long semistructured interviews were conducted with 26 individuals (13 women; median age, 54 years; range, 25-83 years) with a broad range of hearing loss causes, configurations, and severities. From all 3 sources, a total of 125 items were generated and organized into 29 subdomains derived from the World Health Organization's International Classification of Functioning, Disability and Health. Conclusions and Relevance: The association of hearing loss with quality of life is multidimensional and includes subdomains that are not considered in the estimation of health utility by existing utility measures. The presented comprehensive profile of items can be used to generate or evaluate measures of hearing-related quality of life, including utility measures.

Characterizing health state utilities associated with Duchenne muscular dystrophy: a systematic review.

BACKGROUND: Preferences for health states for Duchenne muscular dystrophy (DMD) are necessary to assess costs and benefits of novel therapies. Because DMD progression begins in childhood, the impact of DMD on health-related quality-of-life (HRQoL) affects preferences of both DMD patients and their families. The objective of this review was to synthesize published evidence for health state utility from the DMD patient and caregiver perspectives. METHODS: A systematic review was performed using MEDLINE and Embase, according to best practices. Data were extracted from studies reporting DMD patient or caregiver utilities; these included study and patient characteristics, health states considered, and utility estimates. Quality appraisal of studies was performed. RESULTS: From 888 abstracts, eight publications describing five studies were identified. DMD utility estimates were from preference-based measures presented stratified by ambulatory status, ventilation, and age. Patient (or patient-proxy) utility estimates ranged from 0.75 (early ambulatory DMD) to 0.05 (day-and-night ventilation). Caregiver utilities ranged from 0.87 (for caregivers of adults with DMD) to 0.71 (for caregivers of predominantly childhood patients). Both patient and caregiver utilities trended lower with higher disease severity. Variability in utilities was observed based on instrument, respondent type, and country. Utility estimates for health states within non-ambulatory DMD are under reported; nor were utilities for DMD-related health states such as scoliosis or preserved upper limb function identified. CONCLUSION: Published health state utilities document the substantial HRQoL impacts of DMD, particularly with disease progression. Additional research in patient utilities for additional health states, particularly in non-ambulatory DMD patients, is warranted.

DIVERT-Collaboration Action Research and Evaluation (CARE) Trial Protocol: a multiprovincial pragmatic cluster randomised trial of cardiorespiratory management in home care.

INTRODUCTION: Home care clients are increasingly medically complex, have limited access to effective chronic disease management and have very high emergency department (ED) visitation rates. There is a need for more appropriate and targeted supportive chronic disease management for home care clients. We aim to evaluate the effectiveness and preliminary cost effectiveness of a targeted, person-centred cardiorespiratory management model. METHODS AND ANALYSIS: The Detection of Indicators and Vulnerabilities of Emergency Room Trips (DIVERT) - Collaboration Action Research and Evaluation (CARE) trial is a pragmatic, cluster-randomised, multicentre superiority trial of a flexible multicomponent cardiorespiratory management model based on the best practice guidelines. The trial will be conducted in partnership with three regional, public-sector, home care providers across Canada. The primary outcome of the trial is the difference in time to first unplanned ED visit (hazard rate) within 6 months. Additional secondary outcomes are to identify changes in patient activation, changes in cardiorespiratory symptom frequencies and cost effectiveness over 6 months. We will also investigate the difference in the number of unplanned ED visits, number of inpatient hospitalisations and changes in health-related quality of life. Multilevel proportional hazard and generalised linear models will be used to test the primary and secondary hypotheses. Sample size simulations indicate that enrolling 1100 home care clients across 36 clusters (home care caseloads) will yield a power of 81% given an HR of 0.75. ETHICS AND DISSEMINATION: Ethics approval was obtained from the Hamilton Integrated Research Ethics Board as well as each participating site's ethics board. Results will be submitted for publication in peer-reviewed journals and for presentation at relevant conferences. Home care service partners will also be informed of the study's results. The results will be used to inform future support strategies for older adults receiving home care services. TRIAL REGISTRATION NUMBER: NCT03012256.