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The article focuses on a study which examines cardiac biomarkers as well as the health-related quality of life in patients with light chain (AL) amyloidosis, which is a rare and often fatal disease caused by misfolded light chains, that form soluble toxic aggregates that deposit as fibrils in tissue and organs. Information about disease progression and cardiac response to treatment is presented.

BACKGROUND: Immunoglobulin light-chain amyloidosis (AL amyloidosis) is a rare and often fatal disease for which there is currently no treatment approved by the US Food and Drug Administration or the European Medicines Agency. Treatment options, which are typically based on therapies for multiple myeloma and are used off-label, are associated with substantial adverse events (AEs). Because the severity of AEs is often determined by clinicians, evaluations of treatment tolerability may not fully consider patients' own experience with treatment. OBJECTIVES: To explore the prevalence of AEs and treatment tolerability problems as reported by patients who received therapies for AL amyloidosis, and to examine the effects of AEs on treatment continuation and on health-related quality of life (HRQOL). METHODS: Patients with AL amyloidosis were recruited for this noninterventional, longitudinal, online survey. The patients responded to survey items regarding demographics, disease characteristics, most recent AL amyloidosis treatment, and HRQOL. The study analyses are based on data collected during the 6-month follow-up survey and are restricted to patients who completed the baseline and 6-month surveys and received treatment for AL amyloidosis within 6 months before the follow-up survey. RESULTS: A total of 100 patients met the inclusion criteria and were included in the study. The patients self-reported having a variety of AEs, which ranged in severity. Overall, 69.4% of patients had problems tolerating their treatment in the past 6 months, of whom 22% discontinued at least 1 therapy. In addition, approximately 33% of patients reduced their AL amyloidosis treatment because of AEs. Most often reported AEs included fatigue (83%), shortness of breath (53%), nausea (52%), and diarrhea (51%). Overall, 50% of the patients reported that their treatment was moderately well-tolerated and 41% said it was very well-tolerated. Those whose treatment was not well-tolerated had significantly worse HRQOL than patients whose treatment was well-tolerated. CONCLUSIONS: Patient-reported experiences should be considered by clinicians when making treatment- related decisions. More research is needed to explore additional factors that may contribute to treatment discontinuation in patients with AL amyloidosis. [ABSTRACT FROM AUTHOR]

Background: Light chain (AL) amyloidosis is a rare disease characterized by misfolded amyloid protein deposits in tissues and vital organs, and little is known about the burden of AL amyloidosis on health-related quality of life. This study aimed to quantify the burden of AL amyloidosis in terms of health-related quality of life in a diverse, community-based sample of AL amyloidosis patients.Results: The SF-36v2® Health Survey (SF-36v2), a widely used generic measure of health-related quality of life (using physical and mental summary scales and subscales assessing eight aspects of functioning and well-being), was administered as an online survey of AL amyloidosis patients with AL amyloidosis (ClinicalTrials.gov, NCT02574676 ; n = 341). Compared with adjusted general population sample norms, health-related quality of life of AL amyloidosis patients was significantly worse across all SF-36v2 scales and summary measures based on analysis of variance (p < 0.05 for all). The largest decrement in AL amyloidosis patients was related to General Health (Δ = 9.7; p < 0.001). With the exception of Bodily Pain and Mental Health, differences were also clinically meaningful based on established clinically minimal important differences. The burden of AL amyloidosis overall and in key subgroups tended to be greater on physical health than on mental health. Stratified analyses indicated additional burden among patients with recently diagnosed disease and those with cardiac involvement than among their respective counterparts.Conclusion: Understanding the burden of AL amyloidosis highlights the unmet need for treatment, helps physicians identify ancillary treatments and services geared towards improving patients' functioning, well-being, and overall health-related quality of life. These findings also help to support the use of health-related quality of life end points as important outcome measures in current and future treatment studies.Trial Registration: ClinicalTrials.gov, NCT02574676 . Registered October 5, 2015. [ABSTRACT FROM AUTHOR]

This article focuses on the development, implementation, and evaluation of a branded smoking cessation campaign piloted in four cities in the United States. The development of the EX brand and messaging strategies were based on existing public health literature and extensive formative research. Market segmentation was specifically employed to create messages that would resonate with a defined target audience, smokers who were thinking about quitting smoking. This approach led to the creation of a campaign with an empathetic and hopeful tone that was designed to change relevant beliefs about smoking and to increase smokers' self-efficacy in regard to quitting smoking. As part of a comprehensive evaluation, telephone surveys were conducted in three pilot cities to measure awareness of and receptivity to the EX brand. Awareness of EX varied across the three markets based on the media buy. Receptivity to the EX brand was high across the three samples comprised of three different racial/ethnic populations. High receptivity was also found among the target audience: smokers who were thinking about quitting smoking. Findings from this pilot campaign helped to inform the launch of a national smoking cessation campaign. [ABSTRACT FROM AUTHOR]

Objectives: To develop, implement, and assess the efficacy of a comprehensive, evidence-based smoking cessation program for entertainment industry workers and their families. Methods: Study participants were recruited from 5 outpatient medical clinics and a worksite setting. Tobacco use data were collected during the initial counseling visit and at 6-month follow-up. Univariate and multivariate regressions were used in analysis. Results: More than 50% of participants (n=470) self-reported 7-day abstinence at follow-up. The majority of participants used combination cessation medications, with more than 50% still using at least 1 medication at 6 months. Conclusions: This evidence-based smoking cessation program using behavioral counseling and combination pharmacotherapy was successful with entertainment industry workers. [ABSTRACT FROM AUTHOR]

Background: Mass media campaigns can be an effective strategy to increase quitting activity among smokers, particularly when aired in the context of other anti-tobacco efforts. Design: A longitudinal study using data collected from smokers identified in a random-digit-dial survey of adults in Grand Rapids MI, prior to the campaign and approximately 6 months after the launch of the campaign. Setting/participants: Adult smokers who were interviewed in the fall of 2006 and agreed to participate in a follow-up interview approximately 6 months later (n=212). Intervention: A pilot mass media campaign, branded EX®, which used empathy to encourage smokers to “relearn” life without cigarettes, and focused on disassociating smoking from common activities that would otherwise function as smoking cues, such as driving or drinking coffee. The campaign averaged 100 targeted rating points per week on television. Main outcome measures: Primary outcome measures were five campaign-related cognitions and confidence in quitting. Secondary outcome measures were quitting behaviors. Results: This 2007 analysis suggests that the campaign generated a high level of awareness of EX, with 62% of the sample demonstrating confirmed awareness and 79% reporting aided awareness. Awareness of EX was associated with significant change in two of five campaign-related cognitions. Awareness was not associated with confidence in quitting or having made a quit attempt. Conclusions: These findings demonstrate that a branded, empathetic media campaign that offers smokers practical advice on how to approach quitting can change cognitions related to successful cessation over a relatively short time period. [Copyright &y& Elsevier]

Background: Children with sickle cell disease (SCD) experience a multiplex of disease-related symptoms and complications, including vaso-occlusive crises (VOCs), episodes characterized by extreme pain., Methods: A cross-sectional observational survey examined the health-related quality of life (HRQoL) and school experiences of children with SCD 2 months-11 years, burden experienced by their caregivers, and associations between these outcomes and VOC frequency. Caregivers (N=167) of children with SCD in the US completed the Infant-Toddler Quality of Life-Short Form 47 (ITQoL-SF47) for children 2 months-4 years, the Child Health Questionnaire-Parent Form 50 (CHQ-PF50) and PROMIS Pain Interference and Sleep Disturbance Parent Proxy short forms for children 5-11 years, and a study-specific survey of school experiences., Results: Children with SCD 2 months-4 years had lower ITQoL-SF47 scores (ie, worse HRQoL, p<0.001) than a normative sample of children; across domains, differences ranged from 18.73-45.03 points and exceeded minimal important difference (MID) thresholds. Except for the behavior domain, children with SCD 5-11 years had lower scores on all CHQ-PF50 domains than the normative sample (p<0.001); differences ranged from 6.78-36.37 points and exceeded MID thresholds. Children with more frequent VOCs had lower HRQoL and worse school experiences than children with less frequent VOCs (p<0.05, except for behavior domains). The largest differences based on VOC frequency were observed for overall health and bodily pain/discomfort among children 2 months-4 years (differences=40.88 and 32.50 points, respectively), and bodily pain and role/social limitations due to physical health among children 5-11 years (differences=38.99 and 37.80, respectively). Caregivers of children with more frequent VOCs experienced greater burden than caregivers of children with less frequent VOCs, though specific areas of impact (eg, caregiver emotions, time) differed across child age groups., Conclusion: VOC frequency is negatively associated with HRQoL, highlighting the burden experienced by children with SCD and their caregivers., Competing Interests: GPY, JP, SL, and SI are employees of Novartis Pharmaceuticals Corporation. GPY and JP also own stock in the company. AC has received consultancy and research funding from Novartis Pharmaceuticals Corporation. He also reports grants for clinical trial and/or consultancy from Global Blood Therapeutics, Agios, Vertex, and Bluebird Bio, outside the submitted work. AAR and KLM are employees of QualityMetric Incorporated, LLC, and received funding from Novartis Pharmaceuticals Corporation to conduct this research. After the study was conducted, QualityMetric acquired ownership of the HealthActCHQ family of surveys, which includes the CHQ-PF50 and the ITQoL-SF47. The authors report no other conflicts of interest in this work., (© 2023 Campbell et al.)

Purpose: The purpose of this secondary data analysis was to examine the association between hematologic response and health-related quality of life (HRQoL) among patients with light-chain (AL) amyloidosis., Patients and Methods: Data for this secondary analysis were collected through a non-interventional, longitudinal, online self-report survey of patients with AL amyloidosis. Patients completed an initial online survey, with follow-up surveys administered 1, 6, 12, 18, and 24 months after completion of the initial survey. The online survey included an assessment of patients' most recent self-reported hematologic response status. Eight domains and 2 summary components of HRQoL were evaluated with the SF-36v2 ® Health Survey. A series of logistic regression models were used to examine the association between self-reported hematologic response at 24 months (dichotomized as new or maintained complete hematologic response; less than a complete response) and change in HRQoL from baseline to 24 months (dichotomized as meaningful worsening; improvement or preservation)., Results: For all measured domains of HRQoL except physical functioning, there was no statistically significant relationship between meaningful worsening in HRQoL and hematologic response status at 24 months. Patients without a complete hematologic response had an odds of experiencing meaningful worsening of HRQoL that was similar to that of patients with a complete hematologic response., Conclusion: Among patients with AL amyloidosis, change in HRQoL was generally not associated with hematologic response. Achieving a complete hematologic response does not necessarily mean that a patient will experience increased or stable HRQoL. When defining treatment success, it is important to recognize that clinical markers such as hematologic response may not fully encapsulate the patient experience., Competing Interests: AC is an employee of and TPQ is a former employee of Prothena Bioscience Inc and both are stockholders of Prothena Corporation plc. AAR, KLM, and MKC are employees of QualityMetric Incorporated, LLC, and received funding from Prothena to conduct this research. QualityMetric owns the copyright to and receives licensing fees for the use of the SF-36v2® Health Survey. The authors report no other conflicts of interest in this work., (© 2023 Rizio et al.)

Purpose: Patients with transthyretin amyloidosis (ATTR) experience a wide variety of symptoms and impacts on health-related quality of life (HRQoL). However, the lack of an ATTR-specific patient-reported outcome (PRO) measure has made consistent measurement of HRQoL in ATTR challenging. This paper describes the development of a conceptual model and subsequent content for the Transthyretin Amyloidosis - Quality of Life Questionnaire (ATTR-QOL), an ATTR-specific PRO measure., Methods: This was a cross-sectional, non-interventional, US-based study. The study design included three stages: 1) a targeted literature review followed by qualitative data collection with patients and experts; 2) development of a conceptual model and PRO measure; and 3) review of the PRO measure using a modified Delphi method, translatability assessment, and interviews with patients and experts. Revisions were made to the measure after each round of review., Results: Forty-four patients and 29 experts participated in this study. The conceptual model included two primary concepts of interest: symptoms (cardiac, neuropathic-peripheral, neuropathic-autonomic, and other) and impacts (eg, physical, role, and mental/emotional functioning). Seventy-two items were created (32 symptoms; 40 impacts) to align with the model. A recall period of one month was selected based on participant input., Conclusion: The ATTR-QOL was created with significant patient involvement and guidance from a multidisciplinary group of experts. The mix of patient and clinical perspectives helped to ensure a balanced representation of all relevant disease experiences and clinical specialties. With further refinement from psychometric testing, the ATTR-QOL will provide a standard, comprehensive measure for all ATTR-specific research including both clinical trials and clinical practice., Competing Interests: KH, SR, and IL are employees of ARC and they also report grants from Alnylam, Akcea, and Pfizer to ARC, during the conduct of the study; In addition, KH reports that the instrument ATTR-QOL used is an intellectual property owned by ARC, and will be licensed for a fee to recoup costs. MO′C, LB, KL, KLM, and MC are employees of QualityMetric Inc (QM), which received funding from ARC to conduct the study. The authors report no other conflicts of interest in this work., (© 2023 O’Connor et al.)

Background: Light chain (AL) amyloidosis is a rare and progressive disease that affects myriad organs and systems. Patients with cardiac involvement have the highest risk of death. This report compiles findings across three cohorts of patients with AL amyloidosis to understand patterns of employment and work impacts., Methods: Data came from three cohorts recruited through patient advocacy organizations in the US. Patients in Cohort 1 completed the SF-36v2 ® Health Survey (SF-36v2), the Work Productivity and Activity Impairments - Specific Health Problem (WPAI) questionnaire, and the 12-item Kansas City Cardiomyopathy Questionnaire (KCCQ-12). The relationship between work impacts (WPAI scores) and HRQoL (SF-36v2 scores) was investigated using multivariable logistic regression and summarized according to cardiac severity using New York Heart Association (NYHA) classes estimated from KCCQ-12 scores. Changes in employment, days of missed work, and long-term disability due to AL amyloidosis were summarized for patients diagnosed in the past 24 months and stratified by NYHA class (Cohort 2). Findings were contextualized using patient interviews (Cohort 3)., Results: Work-related impacts, especially reduced productivity, were common among patients with AL amyloidosis. WPAI scores were significantly related to HRQoL (p<0.05 for all models). Among patients with cardiac involvement, the greatest degree of work impacts was observed for those in NYHA class 3 or 4. Changes in employment, missed work, and long-term disability were common among newly diagnosed patients, especially among those in NYHA class 3 or 4. Patient interviews supported the survey findings; patients described absences, reduced productivity at work, and loss of employment due to the disease and its treatment., Conclusion: Patients with AL amyloidosis, particularly those with more advanced disease, experience impacts across a range of employment-related outcomes. These findings highlight the need for more effective treatments and interventions which may improve functioning and patient outcomes, while reducing indirect costs associated with the disease., Competing Interests: TPQ is an employee and stockholder of Prothena Corporation plc. AAR, KLM, and MKW are employees of QualityMetric Incorporated, LLC, and received funding from Prothena Biosciences Ltd to conduct this research. The authors report no other conflicts of interest in this work., (© 2021 Rizio et al.)

Background: Light chain (AL) amyloidosis, a rare and life-threatening protein misfolding disorder, causes organ damage and severely impacts health-related quality of life (HRQoL). No patient-reported outcome (PRO) HRQoL measure has been validated for use in an AL amyloidosis patient population, leaving a gap for researchers conducting observational studies and clinical trials for drug development. The SF-36 Health Survey (SF-36) has been the most frequently used PRO in AL amyloidosis studies to date, and early qualitative validation studies support its use in this population. The aim of this study was to assess the psychometric properties of the SF-36 among patients with AL amyloidosis., Methods: Data from community-based (n=341) and clinic-based (n=1,438) observational studies were used to document the psychometric properties of the SF-36 in this disease population. Reliability was estimated using internal consistency (Cronbach's alpha) and test-retest reliability (intraclass correlation). Convergent validity, known-groups validity, and the ability to detect change were assessed with available criterion variables., Results: Scale reliability (Cronbach's alpha ≥0.780 for all scores) and test-retest reliability (intraclass correlation coefficients ≥0.731 for all) were acceptable. Scale convergent validity was supported by strong correlations with conceptually related measures. Mean SF-36 scores varied by response to treatment ( P <0.05 for all scores) and a self-reported measure of disease severity ( P <0.001 for all scores). Data indicate that the SF-36 is sensitive to changes in other measures over time., Conclusion: This study provided clear and consistent evidence of the psychometric properties of the SF-36 in both community-based and clinic-based samples of patients with AL amyloidosis., Competing Interests: Disclosure At the time of the original submission, MKW, MSB, and KLM were full-time employees of Optum, Inc, which publishes the SF-36v2, and received research funding from Prothena Biosciences Inc. to conduct the study. SDG was a full-time employee of the study sponsor, Prothena Biosciences Inc. at the time of the original submission. The authors report no other conflicts of interest in this work.

Background: This study examined the content validity of the SF-36v2® Health Survey (SF-36v2) in patients with AL amyloidosis using qualitative interviews with physicians and patients. The study included three distinct phases of qualitative research: concept elicitation interviews among physicians, concept elicitation interviews among patients, and cognitive debriefing interviews among patients. The concept elicitation interviews focused on areas of health-related quality of life that are affected by AL amyloidosis and may be affected by treatment, while patient cognitive debriefings aimed to confirm whether the SF-36v2 instructions, recall period, items, and response choices were comprehensive and understandable to AL amyloidosis patients., Results: Physicians discussed the importance of measuring physical functioning, general health, mental/emotional health, sleep, fatigue, and work impact; though they also reported that they do not routinely use a standard Patient-Reported Outcome (PRO) measure of health-related quality of life. Patients described social, physical, role, and emotional impacts of AL amyloidosis and various treatments. Cognitive debriefing interviews confirmed the relevance of the concepts measured by the SF-36v2 and indicated that patients found the SF-36v2 both easy to understand and complete, that the SF-36v2 instructions and items were comprehensive and understandable without change, and the response choices and recall period were appropriate for use with patients with AL amyloidosis., Conclusions: The findings support the content validity of the SF-36v2 as an appropriate measure of health-related quality of life in patients with AL amyloidosis., Competing Interests: All procedures performed in these studies involving human participants were in accordance with the 1964 Helsinki declaration and its later amendments or comparable ethical standards. All patient interview informed consents, protocols, and discussion guides were approved by the New England Independent Review Board (Patient Concept Elicitation Interviews: IRB #15–140; Patient Cognitive Debriefing Interviews: IRB# 15–435). All patients who participated in the qualitative interviews provided written consent.Not applicable.At the time of the original submission, MKW, MB, KPR, AAR, and KLM were full-time employees of Optum, Inc., which publishes the SF-36v2®, and received research funding from Prothena Biosciences Inc. to conduct the study. SDG is a full-time employee of the study sponsor, Prothena Biosciences Inc. and owns stock in Prothena Corporation plc.Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

Background: Mass media campaigns can be an effective strategy to increase quitting activity among smokers, particularly when aired in the context of other anti-tobacco efforts., Design: A longitudinal study using data collected from smokers identified in a random-digit-dial survey of adults in Grand Rapids MI, prior to the campaign and approximately 6 months after the launch of the campaign., Setting/participants: Adult smokers who were interviewed in the fall of 2006 and agreed to participate in a follow-up interview approximately 6 months later (n=212)., Intervention: A pilot mass media campaign, branded EX, which used empathy to encourage smokers to "relearn" life without cigarettes, and focused on disassociating smoking from common activities that would otherwise function as smoking cues, such as driving or drinking coffee. The campaign averaged 100 targeted rating points per week on television., Main Outcome Measures: Primary outcome measures were five campaign-related cognitions and confidence in quitting. Secondary outcome measures were quitting behaviors., Results: This 2007 analysis suggests that the campaign generated a high level of awareness of EX, with 62% of the sample demonstrating confirmed awareness and 79% reporting aided awareness. Awareness of EX was associated with significant change in two of five campaign-related cognitions. Awareness was not associated with confidence in quitting or having made a quit attempt., Conclusions: These findings demonstrate that a branded, empathetic media campaign that offers smokers practical advice on how to approach quitting can change cognitions related to successful cessation over a relatively short time period., (2010 American Journal of Preventive Medicine. Published by Elsevier Inc. All rights reserved.)

Objectives: To describe the prevalence of obesity, smoking, and both health risk factors together among adults in the United States., Design: Cross sectional analysis of a national health interview survey., Setting: United States., Participants: 29 305 adults (aged > or = 18) in 2002., Main Outcome Measures: Prevalence of adults who are obese (body mass index > or = 30), who smoke, and who are obese and smoke. Prevalence was stratified by age, sex, ethnic group, education, and income., Results: 23.5% of adults were obese, 22.7% smoked, and 4.7% smoked and were obese., Conclusions: Although the proportion of adults who smoke and are obese is relatively low, this subgroup is concentrated among lower socioeconomic groups.