Psychometric Validation of the Modified Functional Scale for the Assessment and Rating of Ataxia (f-SARA) in Patients With Spinocerebellar Ataxia.

This study aimed to generate evidence to support psychometric validity of the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA) among patients with spinocerebellar ataxia (SCA). Psychometric measurement properties and minimal change thresholds of the f-SARA were evaluated using data from a cohort of SCA subjects (recruited at Massachusetts General Hospital [MGH]; n = 33) and data from a phase 3 trial of troriluzole in adults with SCA (NCT03701399 [Study 206]; n = 217), including a subset of patients with the SCA3 genotype (n = 89). f-SARA item ceiling effects were absent within the MGH cohort, while floor effects were present. Excellent internal consistency reliability was demonstrated (αtotal = 0.90; αitems-removed = 0.86-0.90), and item-to-total correlations were strong (r = 0.82-0.91, per item). High test-retest reliability was demonstrated with intraclass correlation coefficients of 0.91 (total) and 0.73-0.92 (items). Convergent and divergent validity was supported, with strong correlations observed between the f-SARA and similarly constructed scales (FARS-FUNC, BARS, PROM-ADL, and FARS-ADL; all p < 0.001) and weaker correlations observed among measures of differing constructs. Mean item and total scores increased with disease severity (by FARS-FUNC quartile; p < 0.001). A 1-point threshold for meaningful changes was supported as 0.5 × SD = 0.89, SEM = 1.12, and mean changes from baseline for patients classified as "improved," "no change," or "deteriorated" were -0.68, 0.02, and 0.58, respectively. Similar trends were observed in Study 206 all-SCA and SCA3 cohorts. The measurement properties of the f-SARA provide evidence of its psychometric validity, responsiveness, and suitability as a clinical outcome measure in patients with SCA, including those with SCA3.

Cost-effectiveness of rimegepant oral lyophilisate compared to best supportive care for the acute treatment of migraine in the UK.

AIMS: Migraine is the most common disabling headache disorder and is characterized by recurrent throbbing head pain and symptoms of photophobia, phonophobia, nausea, and vomiting. Rimegepant 75 mg, an oral lyophilisate calcitonin gene-related peptide antagonist, is the first treatment approved for both the acute and preventative treatment of migraine, and the first acute therapy approved in over 20-years. The objective was to assess the cost-utility of rimegepant compared with best supportive care (BSC) in the UK, for the acute treatment of migraine in the adults with inadequate symptom relief after taking at least 2 triptans, or for whom triptans are contraindicated or not tolerated. MATERIALS AND METHODS: A de novo model was developed to estimate incremental costs and quality-adjusted life years (QALYs), structured as a decision tree followed by Markov model. Patients received rimegepant or BSC for a migraine attack and were assessed for response (pain relief at 2-h). Responders and non-responders followed different pain trajectories over 48-h cycles. Non-responders discontinued treatment while responders continued treatment for subsequent attacks, with a proportion discontinuing over time. Data sources included a post-hoc pooled analysis of the phase 3 acute rimegepant trials (NCT03235479, NCT03237845, NCT03461757), and a long-term safety study (NCT03266588). The analysis was conducted from the perspective of the UK National Health Service and Personal Social Services over a 20-year time horizon. RESULTS: Rimegepant resulted in an incremental cost-utility ratio (ICUR) of £10,309 per QALY gained vs BSC, which is cost-effectiveness at a willingness to pay threshold of £30,000/QALY. Rimegepant generated +0.44 incremental QALYs and higher incremental lifetime costs (£4,492). Improved QALYs for rimegepant were a result of less time spent with severe and moderate headache pain. CONCLUSION: This study highlights the economic value of rimegepant which was found to be cost-effective for the acute treatment of migraine in adults unsuitable for triptans.

A Comprehensive Assessment of Cleft Lip and Palate Websites and Patient Education Information.

OBJECTIVE: The objective of this study is to evaluate online educational resources on cleft lip and palate teams. DESIGN: A Cross-Sectional Study. SETTING: An International, Multi-Institutional Study. PARTICIPANTS: All American Cleft Palate and Craniofacial Association-approved teams with websites. INTERVENTIONS: None. MAIN OUTCOME MEASURES: Websites were assessed for patient education and support content. Affiliation between presence of materials and U.S. Newsweek Top 100 Hospitals was also assessed. RESULTS: 187 teams were included. Presence of educational videos were available in 29.4% of websites, educational infographics in 18.2%, written materials in 66.8%, perioperative instructions in 19.3%, diagnosis information in 34.8% and treatment information in 63.1%. Information on team members were available on 77.0% of websites, resources in languages other than English in 38.5%, and support group resources in 25.7%. Cleft lip and palate diagnosis information was significantly associated with geographic region, with the largest impact from the West region (P = .03). There was a significant difference between teams affiliated with U.S. Newsweek Top 100 hospitals: Top 100 hospitals had a higher presence of educational infographics, perioperative instructions, definitions, diagnosis, and treatment (P < .01). There was no significant difference between presence of educational videos (P = .37). CONCLUSION: While many websites had basic educational materials, very few included detailed information on peri-operative planning, as well as additional forms of information including videos, infographics, and non-English languages. Providing comprehensive patient education materials online is an important supplement for patients with cleft lip and palate and should be prioritized by cleft teams.

It's not all White: Implicit Racial Bias in Imagery Used in Plastic Surgery Resident Education.

OBJECTIVE: Plastic surgery education relies heavily on images featuring patient skin tone; while images can be useful representations, it is highly susceptible to implicit bias, a known contributor to healthcare disparities. Using skin tone as a proxy, this study evaluates patient representation in images used in the American Society of Plastic Surgery Resident Education Curriculum. DESIGN: Color photographs, graphics, and videos featured in the American Society of Plastic Surgery "Course Materials" for each module were categorized using the Fitzpatrick scale (I-II, III-IV, or V-VI) by six reviewers. Proportional data and average number ± standard deviation of photos and graphics for each category were reported. Significant difference between Fitzpatrick I to II and V to VI was investigated via a one-way analysis of variance with a Tukey's post-test to adjust for multiple comparisons. RESULTS: An average of 1861 photographs and 237 graphics were assessed with 82% (1518 ± 25.11) of photos and 97% (231 ± 24.45) of graphics categorized as Fitzpatrick I to II. A one-way analysis of variance with a Tukey's post-test demonstrates a statistical difference between images and graphics categorized as Fitzpatrick I to II and Fitzpatrick V to VI (p < 0.001). CONCLUSIONS: Our data reveals an opportunity to improve racial representation in resident education. When 76% of patients in the United States are white and 13% are Black, our findings demonstrate both an unequal and unrepresentative distribution of photos and graphics of non-white patients. Residency is a formative time in a surgeon's career and therefore, exposure to accurate representation of a diverse patient population is of the utmost importance.

Assessment of Health Information Technology-Related Outpatient Diagnostic Delays in the US Veterans Affairs Health Care System: A Qualitative Study of Aggregated Root Cause Analysis Data.

Importance: Diagnostic delay in the outpatient setting is an emerging safety priority that health information technology (HIT) should help address. However, diagnostic delays have persisted, and new safety concerns associated with the use of HIT have emerged. Objective: To analyze HIT-related outpatient diagnostic delays within a large, integrated health care system. Design, Setting, and Participants: This cohort study involved qualitative content analysis of safety concerns identified in aggregated root cause analysis (RCA) data related to HIT and outpatient diagnostic delays. The setting was the US Department of Veterans Affairs using all RCAs submitted to the Veterans Affairs (VA) National Center for Patient Safety from January 1, 2013, to July 31, 2018. Main Outcomes and Measures: Common themes associated with the role of HIT-related safety concerns were identified and categorized according to the Health IT Safety framework for measuring, monitoring, and improving HIT safety. This framework includes 3 related domains (ie, safe HIT, safe use of HIT, and using HIT to improve safety) situated within an 8-dimensional sociotechnical model accounting for interacting technical and nontechnical variables associated with safety. Hence, themes identified enhanced understanding of the sociotechnical context and domain of HIT safety involved. Results: Of 214 RCAs categorized by the terms delay and outpatient submitted during the study period, 88 were identified as involving diagnostic delays and HIT, from which 172 unique HIT-related safety concerns were extracted (mean [SD], 1.97 [1.53] per RCA). Most safety concerns (82.6% [142 of 172]) involved problems with safe use of HIT, predominantly sociotechnical factors associated with people, workflow and communication, and a poorly designed human-computer interface. Fewer safety concerns involved problems with safe HIT (14.5% [25 of 172]) or using HIT to improve safety (0.3% [5 of 172]). The following 5 key high-risk areas for diagnostic delays emerged: managing electronic health record inbox notifications and communication, clinicians gathering key diagnostic information, technical problems, data entry problems, and failure of a system to track test results. Conclusions and Relevance: This qualitative study of a national RCA data set suggests that interventions to reduce outpatient diagnostic delays could aim to improve test result management, interoperability, data visualization, and order entry, as well as to decrease information overload.

The burden of treatment-resistant depression: A systematic review of the economic and quality of life literature.

BACKGROUND: Major depressive disorder (MDD) is a global public health concern. In particular, treatment-resistant depression (TRD) represents a key unmet need in the management of MDD. A systematic review of the epidemiological and economic literature on the burden associated with an increasing number of treatment steps due to TRD/non-response within an MDD episode was performed to quantify the burden of TRD. METHODS: Studies were identified in the PubMed/Medline databases through April 27th, 2017. Articles were limited to full-length peer-reviewed journal publications with no date restrictions. Economic and patient health-related quality of life (HRQoL) data on non-response by the number of treatment steps were quantified and, where appropriate, compared across studies; otherwise, comparative data within studies were reported. RESULTS: The 12 studies on economic burden found an association between increasing levels of TRD/non-response and elevations in direct and indirect costs. Likewise, the 19 studies studying HRQoL burden found that increasing levels of TRD/non-response correlated with reduced patient HRQoL and health status. LIMITATIONS: TRD is defined inconsistently, which results in notable heterogeneity between published studies and poses methodological challenges for between-study comparisons. It is unknown if the increased economic and patient HRQoL burden are due to factors associated with TRD/non-response in addition to those due to depression persistence or severity. CONCLUSIONS: A consistent trend was observed such that medical costs increased and patient HRQoL and health status decreased by increasing level of TRD/non-response within an MDD episode. These findings highlight the need for improved therapies for TRD to help reduce disease burden.

An Innovative Simulation-based Community-engaged Intervention for Training Research Assistants in Culturally Appropriate Informed Consent.

BACKGROUND: Enrolling under-represented groups in biomedical research remains challenging owing to limited health literacy, historic medical abuses, and mistrust. Addressing such barriers requires scrutiny of informed consent processes and training of research assistants (RAs). Applying simulation methods that are well-established in medical education, Simulation-based Community-engaged Research Intervention for Informed Consent Protocol Testing and Training (SCRIIPTT) engages community members as community advisors (CAs) to train RAs in implementing culturally appropriate approaches, such as addressing difficult issues related to race and power dynamics, into the informed consent training process. OBJECTIVES: To present SCRIIPTT's development, implementation, and preliminary findings. METHODS: Over 12 months, our community-academic partnership 1) introduced SCRIIPTT to the greater community using the Truth & Reconciliation Model, which acknowledges the wrongs of research and offers an apology; 2) deepened understanding of community concerns; 3) developed the intervention; 4) conducted a pilot; and 5) disseminated findings. RESULTS: SCRIIPTT included a comprehensive training manual, scenarios for simulated informed consent encounters, and a 37-item checklist to measure performance. Outcomes include CA and RA satisfaction surveys, and RA performance rating. The checklist adequately measured observable behaviors. RAs reported important gains in knowledge, self-confidence, and cultural competence in conducting informed consent. CAs reported positive experiences, being fully engaged and valued as members of the research team and participants during simulation encounters. CONCLUSIONS: This pilot study demonstrated the feasibility of SCRIIPTT, with preliminary evidence of effectiveness and acceptability. SCRIIPTT offers an innovative approach for community engagement in research training and advancing the skills of RAs in culturally appropriate informed consent.

Spirituality and Willingness to Participate in Health-Related Research Among African Americans.

African Americans remain underrepresented in health-related research. We examined the association between spirituality using the Self-Rating Spirituality Scale (range 6-24) and self-reported willingness to participate in health-related research studies among African Americans. Covariates included gender, education level, employment status, and previous research experience. Adjusted associations were calculated with logistic regression models, with multiple imputation to account for missing data. Results from the logistic regression model show that each one-point increase in the Self-Rating Spirituality Scale was associated with a 24% increase in the odds of being very likely to participate in research (OR: 1.24, 95% CI: 1.07-1.44). Those with less than a college degree (OR: 3.59, 95% CI: 1.51-8.54), who were unemployed (OR: 2.34, 95% CI: 1.03-5.33), and had previous research experience (OR: 2.92, 95% CI: 1.22-6.99) reported increased willingness to participate. This work offers new insight for developing recruitment initiatives within African American spiritual communities.

The economic burden of overactive bladder in the United States: A systematic literature review.

AIMS: Overactive bladder (OAB) affects up to 17% of the United States (US) population. This study aimed to synthesize estimates of direct and indirect costs of OAB in the US and compare costs among those with and without OAB. METHODS: A systematic review was performed using MEDLINE/PubMed and Embase, from 2003 to 2016, following PRISMA guidelines. The target population was adults with idiopathic OAB or urge urinary incontinence from the US. Data were extracted on study and patient characteristics, all-cause and OAB-specific direct costs, resource use, and indirect costs. Costs were inflated to a common price year of 2016 USD. RESULTS: Eighteen studies were included. Mean insurer paid all-cause total direct healthcare costs ranged from 8168 to 15 569 USD, and OAB-specific costs ranged from 656 to 860 USD per-patient annually. Estimates of the incremental costs for OAB patients compared to non-OAB comparators ranged from 43% to 117%. One study estimated total annual indirect costs of OAB at 11 134 USD per-patient. CONCLUSIONS: The range of direct healthcare costs reported for managing patients with OAB varied, but was relatively small given the differing contributing data sources, study designs, and cost definitions. Direct costs were consistently higher among patients with OAB versus non-OAB comparisons, from a 1.4- to >2-fold increase annually. OAB-specific costs made up a small proportion of all-cause costs, highlighting the clinical and economic impact of OAB-related conditions such as falls, urinary tract infection, and depression. Few studies were identified that examined the indirect costs of OAB in the US.

Assessing the Relationship between Performance on the University of California Performance Skills Assessment (UPSA) and Outcomes in Schizophrenia: A Systematic Review and Evidence Synthesis.

OBJECTIVE: To perform a systematic review of the published literature to evaluate how functional capacity, as measured by the University of California at San Diego (UCSD) Performance-based Skills Assessment (UPSA), relates to other functional measures and real-world outcomes among individuals with schizophrenia. METHODS: The MEDLINE® and Embase® databases were searched to identify joint evaluations with UPSA and key functional outcomes (functional scale measures; generic or disease-specific, health-related quality of life [HRQoL]; or real-world outcomes [residential status; employment status]) in patients with schizophrenia. Pearson correlations were estimated between UPSA scores, HRQoL, other functional scale measures, and real-world outcomes, for outcomes described in at least six studies. RESULTS: The synthesis included 76 studies that provided 73 unique data sets. Quantitative assessment between the Specific Level of Function (SLOF) (n=18) scores and UPSA scores demonstrated a moderate borderline-significant correlation (0.45, p=0.06). Quantitative analysis of the relationship between the Global Assessment of Functioning (GAF) (n=11) and the Multidimensional Scale of Independent Functioning (MSIF) (n=6) scales revealed moderate and small nonsignificant Pearson correlations of -0.34 (p=0.31) and 0.12 (p=0.83), respectively. There was a small borderline-significant correlation between UPSA score and residential status (n=36; 0.31; p=0.08), while no correlation was found between UPSA score and employment status (n=19; 0.04; p=0.88). CONCLUSION: The SLOF was the most often used functional measure and had the strongest observed correlation with the UPSA. Although knowledge gaps remain, evidence from this review indicates that there is a quantitative relationship between functional capacity and real-world outcomes in individuals with schizophrenia.

Attention Deficit Hyperactivity Disorder: Is There an App for That? Suitability Assessment of Apps for Children and Young People With ADHD.

BACKGROUND: Attention-deficit/hyperactivity disorder (ADHD) is a complex highly comorbid disorder, which can have a huge impact on those with ADHD, their family, and the community around them. ADHD is currently managed using pharmacological and nonpharmacological interventions. However, with advances in technology and an increase in the use of mobile apps, managing ADHD can be augmented using apps specifically designed for this population. However, little is known regarding the suitability and usability of currently available apps. OBJECTIVE: The aim of this study was to explore the suitability of the top 10 listed apps for children and young people with ADHD and clinicians who work with them. It is hypothesized that mobile apps designed for this population could be more suitably designed for this population. METHODS: The top 10 listed apps that are specifically targeted toward children and young people with ADHD in the United Kingdom were identified via the Google Play (n=5) and iTunes store (n=5). Interviews were then undertaken with 5 clinicians who specialize in treating this population and 5 children and young people with ADHD themselves, to explore their opinions of the 10 apps identified and what they believe the key components are for apps to be suitable for this population. RESULTS: Five themes emerged from clinician and young people interviews: the accessibility of the technology, the importance of relating to apps, addressing ADHD symptoms and related difficulties, age appropriateness, and app interaction. Three additional themes emerged from the clinician interviews alone: monitoring symptoms, side effects and app effect on relationships, and the impact of common comorbid conditions. The characteristics of the apps did not appear to match well with the views of our sample. CONCLUSIONS: These findings suggest that the apps may not be suitable in meeting the complex needs associated with this condition. Further research is required to explore the value of apps for children and young people with ADHD and their families and, in particular, any positive role for apps in the management of ADHD in this age group. A systematic review on how technology can be used to engage this population and how it can be used to help them would be a useful way forward. This could be the platform to begin exploring the use of apps further.

ADHD: Is There an App for That? A Suitability Assessment of Apps for the Parents of Children and Young People With ADHD.

BACKGROUND: Attention-deficit hyperactivity disorder (ADHD) is a highly comorbid disorder that can impact significantly on the individual and their family. ADHD is managed via pharmacological and nonpharmacological interventions. Parents also gain support from parent support groups, which may include chat rooms, as well as face-to-face meetings. With the growth of technology use over recent years, parents have access to more resources that ever before. A number of mobile apps have been developed to help parents manage ADHD in their children and young people. Unfortunately many of these apps are not evidence-based, and little is known of their suitability for the parents or whether they are helpful in ADHD management. OBJECTIVE: The aim of this study was to explore the (1) parents' views of the suitability of the top ten listed apps for parents of children and young people with ADHD and (2) the views of clinicians that work with them on the suitability and value of the apps. METHODS: The top 10 listed apps specifically targeted toward the parents of children and young people with ADHD were identified via the Google Play (n=5) and iTunes store (n=5). Interviews were then undertaken with 7 parents of children or young people with ADHD and 6 clinicians who specialize in working with this population to explore their opinions of the 10 apps identified and what they believe the key components are for apps to be suitable and valuable for this population. RESULTS: Four themes emerged from clinician and parent interviews: (1) the importance of relating to the app, (2) apps that address ADHD-related difficulties, (3) how the apps can affect family relationships, and (4) apps as an educational tool. Two additional themes emerged from the clinician interviews alone: monitoring ADHD symptoms and that apps should be practical. Parents also identified an additional theme: the importance of the technology. Overall, the characteristics of the current top 10 listed apps did not appear to match well to the views of our sample. CONCLUSIONS: Findings suggest that these apps may not fully meet the complex needs of this parent population. Further research is required to explore the value of apps with this population and how they can be tailored to their very specific needs.

A preventative lifestyle intervention for older adults (lifestyle matters): a randomised controlled trial.

Objectives: to test whether an occupation-based lifestyle intervention can sustain and improve the mental well-being of adults aged 65 years or over compared to usual care, using an individually randomised controlled trial. Participants: 288 independently living adults aged 65 years or over, with normal cognition, were recruited from two UK sites between December 2011 and November 2015. Interventions: lifestyle Matters is a National Institute for Health and Care Excellence recommended multi-component preventive intervention designed to improve the mental well-being of community living older people at risk of decline. It involves weekly group sessions over 4 months and one to one sessions. Main outcome measures: the primary outcome was mental well-being at 6 months (mental health (MH) dimension of the SF-36). Secondary outcomes included physical health dimensions of the SF-36, extent of depression (PHQ-9), quality of life (EQ-5D) and loneliness (de Jong Gierveld Loneliness Scale), assessed at 6 and 24 months. Results: data on 262 (intervention = 136; usual care = 126) participants were analysed using intention to treat analysis. Mean SF-36 MH scores at 6 months differed by 2.3 points (95 CI: -1.3 to 5.9; P = 0.209) after adjustments. Conclusions: analysis shows little evidence of clinical or cost-effectiveness in the recruited population with analysis of the primary outcome revealing that the study participants were mentally well at baseline. The results pose questions regarding how preventive interventions to promote well-being in older adults can be effectively targeted in the absence of proactive mechanisms to identify those who at risk of decline. Trial Registration: ISRCTN67209155.

Psychometric Development of the Research and Knowledge Scale.

BACKGROUND: Many research participants are misinformed about research terms, procedures, and goals; however, no validated instruments exist to assess individual's comprehension of health-related research information. We propose research literacy as a concept that incorporates understanding about the purpose and nature of research. OBJECTIVES: We developed the Research and Knowledge Scale (RaKS) to measure research literacy in a culturally, literacy-sensitive manner. We describe its development and psychometric properties. RESEARCH DESIGN: Qualitative methods were used to assess perspectives of research participants and researchers. Literature and informed consent reviews were conducted to develop initial items. These data were used to develop initial domains and items of the RaKS, and expert panel reviews and cognitive pretesting were done to refine the scale. We conducted psychometric analyses to evaluate the scale. SUBJECTS: The cross-sectional survey was administered to a purposive community-based sample (n=430) using a Web-based data collection system and paper. MEASURES: We did classic theory testing on individual items and assessed test-retest reliability and Kuder-Richardson-20 for internal consistency. We conducted exploratory factor analysis and analysis of variance to assess differences in mean research literacy scores in sociodemographic subgroups. RESULTS: The RaKS is comprised of 16 items, with a Kuder-Richardson-20 estimate of 0.81 and test-retest reliability 0.84. There were differences in mean scale scores by race/ethnicity, age, education, income, and health literacy (all P<0.01). CONCLUSIONS: This study provides preliminary evidence for the reliability and validity of the RaKS. This scale can be used to measure research participants' understanding about health-related research processes and identify areas to improve informed decision-making about research participation.

On Edge: the impact of race-related vigilance on obesity status in African-Americans.

OBJECTIVE: Nearly half of African-Americans are classified as obese. Perceived racism has been associated with obesity, yet the internal experiences of racism have received little attention. African Americans who face racism may "ready themselves" to cope through survival strategies, including race-related vigilance. This study explores the association between race-related vigilance and obesity in African Americans. DESIGN AND METHODS: The Reactions to Race module of the Behavioral Risk Factor Surveillance Survey (years 2002-2010) was used. Our sample size consisted of 12,214 African-Americans. Race-related vigilance was assessed as: "How often do you think about your race?" and classified as: never, < daily, daily, and > daily. Obesity was dichotomized as body mass index (BMI) ≥30 kg/m2 vs. < 30 kg/m2 using self-reported weight and height. Multivariable logistic models assessed the association between race-related vigilance and obesity. RESULTS: Seventeen percent of respondents reported thinking about their race > daily; 14% daily; 31% < daily, and 39% reported never thinking about their race. Compared to those who reported never thinking about their race, the adjusted odds of obesity were 0.91, 95% CI: 0.72-1.15 among those thinking about their race daily. CONCLUSIONS: Frequently thinking about one's race was a risk factor for obesity in African-Americans in this study. Internalized impacts of racism captured through race-related vigilance may be particularly detrimental to African-Americans, driving their risk for obesity.