Cross-Sectional Quantitative Evaluation of a Novel Patient-Reported Outcome Measure in Familial Chylomicronemia Syndrome.

Background: Familial chylomicronemia syndrome (FCS) is a rare metabolic disorder that impacts physical, emotional, social, and cognitive functioning. The FCS-Symptom and Impact Scale (FCS-SIS) patient-reported outcome (PRO) measure assesses common symptoms and impacts of FCS. This study was conducted to evaluate cross-sectional psychometric properties of the FCS-SIS and its scoring method. Methods: This multisite, cross-sectional, observational study of individuals with FCS was conducted in the United States and Canada. Participants completed a survey composed of 7 PRO measures, including the FCS-SIS, and questions about clinical characteristics and demographics. The structure of the FCS-SIS was evaluated using inter-item and item-scale correlations and internal consistency reliability. Construct, known-groups, and criterion validity were evaluated by examining associations between FCS-SIS item and composite scores and other measures included within the survey. Results: Most of the 33 participants were female (63.6%) and White (78.1%). On average, participants reported first noticing FCS symptoms at ~16 years, with abdominal pain the most frequently reported initial symptom (n=20). Participants reported 2.5 acute pancreatitis attacks on average over the past year. Average FCS-SIS symptom item scores ranged from 1.8 to 3.9 (on a 0-to-10 scale [none-to-worst-possible]) within the 24-hour recall period, with an average Symptom composite score of 2.7. The average impact item scores on the FCS-SIS ranged from 1.6 to 3.0 (on a 0-to-4 scale), with an average Impact composite score of 2.1. Inter-item correlations between the FCS-SIS Symptom items ranged from 0.32 to 0.78. Corrected item-total correlations were highly satisfactory for Impact items, ranging from 0.62 to 0.85. All a priori validity hypotheses were supported by observed correlations and score differences between known groups. Conclusion: The results of this study support the structure, reliability, and validity of the FCS-SIS, laying the psychometric groundwork for longitudinal evaluation of its utility in assessing treatment benefit in FCS clinical studies.

Changes in Clinical Management of Patients with Schizophrenia Treated with Long-Acting Injectable Antipsychotics (LAIs), Including Telepsychiatry Use, During the COVID-19 Pandemic.

Purpose: The COVID-19 pandemic substantially impacted care of patients with schizophrenia treated with long-acting injectable antipsychotics (LAIs). This study (OASIS-MAPS) examined how clinical sites adapted operations and used telepsychiatry to maintain standard of care for these patients during the pandemic. Methods: Two online surveys (initial: October-November 2020, N = 35; follow-up: July-September 2021, N = 21) were completed by a principal investigator (PI) or PI-appointed designee at sites participating in the OASIS study (NCT03919994). Survey responses were analyzed descriptively. Results: At the time of the initial survey, all 35 participating sites were using variants of telepsychiatry, with 20 sites adopting it after the pandemic started. Most sites reported no negative impacts of the pandemic on medication adherence, although approximately 20% of sites reported decreased adherence for LAIs. Twelve sites (34%) reported switching patients with schizophrenia from LAIs to oral antipsychotic medications, while 11 sites (31%) reported switching patients from shorter to longer injection interval LAIs during the pandemic. Most sites did not experience difficulties in implementing or expanding telepsychiatry services, although lower reimbursement rate for telepsychiatry and patients' lack of access to and training on relevant technologies were the most frequently reported barriers. Conclusion: Changes made by sites after the pandemic onset were viewed by almost all participants as satisfactory for maintaining standard of care. Almost all participants thought that the use of telepsychiatry services would continue after the pandemic in a hybrid manner combining telepsychiatry and office visits. Ensuring that patients have equitable access to telepsychiatry will be important in the post-pandemic future.

Translating the REACH OUT dementia caregiver intervention into a primary care setting: a pilot study.

OBJECTIVE: The current study translated the Resources for Enhancing Alzheimer's Caregiver Health: Offering Useful Treatments (REACH OUT), a skills-building stress and burden intervention, for the primary care setting and pilot the resulting intervention. METHODS: The 16-week intervention consisted of a combination of clinic-based group and one-on-one sessions offered within a medical home, geriatrics clinic. A quasi-experimental pre- and post-test study design without a control group tested the resulting intervention. Semi-structured qualitative exit interviews evaluated program satisfaction. RESULTS: Twenty-five caregivers participated in one of four intervention groups; 21 caregivers completed the intervention (attended at least five of six group sessions). Caregiver burden on standardized assessments was significantly reduced between pre- and post-intervention, specifically for physical/emotional strain and caregiving uncertainty. Significant reductions were found in the frequency of reported disruptive behaviors; increased caregiver confidence in handling behavior problem frequency, depressive symptoms, disruptive behaviors, and memory-related problems; and decreased bother with respect to behavioral problem frequency and care recipient depression. Program satisfaction was high. CONCLUSION: This work suggests that the REACH OUT program can be successfully modified for use within a primary-care medical home setting.

Antipsychotic Treatment Experiences of People with Schizophrenia: Patient Perspectives from an Online Survey.

BACKGROUND: This survey examined the experiences of people living with schizophrenia who have used oral antipsychotics (APs). METHODS: Adults with self-reported physician-diagnosed schizophrenia (N=200), who were members of an online research participation panel and reported taking one or more oral APs within the last year, completed a cross-sectional online survey that focused on direct report of their experiences regarding APs (eg, symptoms, side effects, adherence). Descriptive analyses were conducted for the total survey sample and for subgroups defined a priori by experience with specific, prevalent side effects. RESULTS: The mean age of the sample was 41.9 (SD=11.0) years, 50% of participants were female, and 32% were nonwhite. Overall ratings were positive for medication effectiveness and convenience but negative for side effects. While most participants reported that APs improved schizophrenia symptoms (92%), 27% reported APs as having done "more harm than good." Almost all participants (98%) reported experiencing side effects of APs, with the most common being anxiety (88%), feeling drowsy/tired (86%), and trouble concentrating (85%). Side effects frequently cited as either "extremely" or "very" bothersome were weight gain (56%), sexual dysfunction (55%), and trouble concentrating (54%). Over 80% reported that side effects had negatively impacted their work and social functioning (eg, social activities or family/romantic relationships). Since initiating treatment, 56% of respondents had stopped taking APs at some point (65% of these due to side effects). Side effects commonly reported as having led to stopping AP treatment were "feeling like a 'zombie'" (22%), feeling drowsy/tired (21%), and weight gain (20%). CONCLUSION: Most participants reported improvements in schizophrenia symptoms associated with the use of APs. However, most participants also reported experiencing numerous bothersome side effects that negatively impacted their work, social functioning, and treatment adherence. Results highlight the unmet need for new APs with favorable benefit-risk profiles.

Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis.

OBJECTIVE: To examine the impact on quality of life (QOL) of patients with hATTR amyloidosis with polyneuropathy treated with inotersen (Tegsedi™) versus placebo. METHODS: Data were from the NEURO-TTR trial (ClinicalTrials.gov Identifier: NCT01737398), a phase 3, multinational, randomized, double-blind, placebo-controlled study of inotersen in patients with hATTR amyloidosis with polyneuropathy. At baseline and week 66, QOL measures-the Norfolk-QOL-Diabetic Neuropathy (DN) questionnaire and SF-36v2® Health Survey (SF-36v2)-were assessed. Treatment differences in mean changes in QOL from baseline to week 66 were tested using mixed-effect models with repeated measures. Responder analyses compared the percentages of patients whose QOL meaningfully improved or worsened from baseline to week 66 in inotersen and placebo arms. Descriptive analysis of item responses examined treatment differences in specific activities and functions at week 66. RESULTS: Statistically significant mean differences between treatment arms were observed for three of five Norfolk-QOL-DN domains and five of eight SF-36v2 domains, with better outcomes for inotersen than placebo in physical functioning, activities of daily living, neuropathic symptoms, pain, role limitations due to health problems, and social functioning. A larger percentage of patients in the inotersen arm than the placebo arm showed preservation or improvement in Norfolk-QOL-DN and SF-36v2 scores from baseline to week 66. Responses at week 66 showed more substantial problems with daily activities and functioning for patients in the placebo arm than in the inotersen arm. CONCLUSION: Patients with hATTR amyloidosis with polyneuropathy treated with inotersen showed preserved or improved QOL at 66 weeks compared to those who received placebo.

Social Disparities in Unaided Quit Attempts Among Daily Current and Former Smokers: Results From the 2010-2011 Tobacco Use Supplement to the Current Population Survey.

INTRODUCTION: There are well-documented inverse relationships between smoking and smoking cessation with measures of socioeconomic status. This study used nationally representative data to examine unaided quit attempts and their sociodemographic determinants among daily current and former smokers who made a quit attempt in the last 12 months. METHODS: We used data from the 2010-2011 Tobacco Use Supplement to the Current Population Survey. We limited the analysis to current daily smokers who made a quit attempt in the past year and former smokers who were daily smokers 1 year prior to the survey (N = 8201). RESULTS: Nearly 62% (n = 5078) of the sample made an unaided quit attempt. Adjusted results indicated unaided quit attempts were more likely among males compared to females (P < .001), younger age groups compared to older age groups (P < .001), non-Hispanic blacks compared to non-Hispanic whites (P < .001), among people with lower income compared to people with higher income (P < .001), and among people with lower nicotine dependence compared to those with higher nicotine dependence (P < .001). CONCLUSIONS: Most quit attempts were unaided and there were significant sociodemographic disparities in unaided quit attempts. Considering that cessation aids enhance the likelihood of quitting, policies and programs should target populations which are more likely to attempt quitting without an aid and encourage them to use or provide subsidized cessation aids. Healthcare providers should advise their patients about approaches to quitting. IMPLICATIONS: This study used the most recent nationally representative data for the United States to examine sociodemographic disparities in unaided quitting among current and former daily smokers who made a quit attempt in the last 12 months. Most quit attempts were unaided. People who were male, younger, non-Hispanic black, had lower nicotine dependence, and those who were low income were more likely to make an unaided quit attempt. These results could be used by policy makers and program planners to develop cessation interventions directed at specific populations to improve smoking cessation rates.

Point-of-Sale Cigarette Marketing, Urge to Buy Cigarettes, and Impulse Purchases of Cigarettes: Results From a Population-Based Survey.

AIMS: Our aim was to examine the association of exposure to point-of-sale (POS) cigarette marketing for one's regular brand, as well as any brand of cigarettes, with the urge to buy cigarettes and frequency of impulse purchases of cigarettes. METHODS: Nine hundred ninety-nine smokers in Omaha, Nebraska were interviewed via telephone. Cigarette marketing was measured by asking respondents questions about noticing pack displays, advertisements, and promotions such as discounts for their regular brand as well as any brand of cigarettes in their neighborhoods stores. We measured urge to buy cigarettes with the question "When you are in a store in your neighborhood that sells tobacco products, how often do you get an urge to buy cigarettes?" We measured frequency of impulse purchases of cigarettes with the question "When you are shopping in a store in your neighborhood for something other than cigarettes, how often do you decide to buy cigarettes?" We estimated ordinary least squares linear regression models to address the study aim. RESULTS: Higher levels of POS marketing for one's regular brand and any brands of cigarettes were associated with more frequent urges to buy (P < .001 and P < .001, respectively) and impulse purchases of cigarettes (P = .01 and P = .013, respectively), after adjusting for covariates. CONCLUSION: Exposure to POS marketing for one's own brand of cigarette as well as any brand is associated with urges to buy and impulse purchases of cigarettes. IMPLICATIONS: Existing studies on the association of POS cigarette marketing with urge to buy and an impulse purchase of cigarettes only focus on cigarette pack displays, not on advertisements and promotions. Also, these studies make no distinction between marketing for the smokers' regular brand and any brand of cigarettes. This study found that Exposure to POS marketing for one's own brand of cigarette as well as any brand is associated with urges to buy and impulse purchases of cigarettes. Our findings can provide part of the evidence-base needed by the Food and Drug Administration or local authorities to regulate POS marketing.

Dieting Increases the Likelihood of Subsequent Obesity and BMI Gain: Results from a Prospective Study of an Australian National Sample.

BACKGROUND: Diet is a major determinant of obesity; however, findings from the studies examining how dieting to lose weight affects weight gain have been inconclusive. PURPOSE: Our aim was to examine the longitudinal association of frequency of dieting for weight loss with (a) obesity status and (b) body mass index (BMI) change. METHODS: We used data from Waves 9 (2009) and 10 (2010) of the Household Income and Labour Dynamics in Australia (HILDA) survey. Binominal logistic regression estimated the association of frequency of dieting in 2009 on probability of obesity in 2010. Multinomial logistic regression estimated the association of frequency of dieting in 2009 on the probability of BMI gain versus BMI maintenance and BMI loss between 2009 and 2010. The analysis sample size was 8824. RESULTS: Compared to those who were never on a diet in the previous year, the odds of obesity were 1.9, 2.9, and 3.2 times higher among those who were on a diet once, more than once, and always, respectively. Similarly, the odds of BMI gain versus BMI maintenance and also versus BMI loss were higher among those who dieted than those who did not. CONCLUSIONS: Dieting to lose weight can contribute to the risk of future obesity and weight gain. Losing weight requires a commitment to change one's lifestyle and a sustained effort to maintain a healthy diet and engage in physical activity.

Association between duration of use of pharmacotherapy and smoking cessation: findings from a national survey.

OBJECTIVE: To investigate the association of the duration of use of prescription medications and nicotine replacement therapy (NRT) with smoking cessation using a national sample of the general population in the USA, controlling for nicotine dependence and sociodemographic variables. SETTING USA PARTICIPANTS: We used data from the 2010-2011 Tobacco Use Supplement to the US Current Population Survey. We limited the analysis to current daily smokers who made a quit attempt in the past year and former smokers who were a daily smoker 1 year prior to the survey (n=8263). Respondents were asked about duration of use of prescription medication (varenicline, bupropion, other) and NRT (nicotine patch, gum/lozenges, nasal spray and inhaler) for smoking cessation. PRIMARY OUTCOME MEASURE: Successful smoking cessation. Individuals who reported to have smoked at least 100 cigarettes in their lifetime but were not smoking at all at the time of the interview and were a daily smoker 1 year prior to the interview were considered to have successfully quit smoking. RESULTS: After adjusting for daily cigarette consumption and sociodemographic covariates, we found evidence for an association between duration of pharmacotherapy use and smoking cessation (p<0.001). Adjusted cessation rates for those who used prescription medication or NRT for 5+ weeks were 28.8% and 27.8%, respectively. Adjusted cessation rates for those who used prescription medication or NRT for less than 5 weeks varied from 6.2% to 14.5%. Adjusted cessation rates for those who used only behavioural counselling and those who attempted to quit smoking unassisted were 16.1% and 16.4%, respectively. CONCLUSIONS: Use of pharmacotherapy for at least 5 weeks is associated with increased likelihood of successful smoking cessation. Results suggest that encouraging smokers who intend to quit to use pharmacotherapy and to adhere to treatment duration can help improve chances of successful cessation.