A Qualitative Concept Elicitation Study to Understand Patient-Reported Symptoms and Impacts of Neisseria gonorrhoeae Infections in the United States.

BACKGROUND: Gonorrhea is the second most common bacterial sexually transmitted infection in the United States, with rising rates. Emerging antimicrobial resistance threatens public health, and vaccines are in development. This study documents patient-reported gonorrhea symptoms and health-related quality-of-life (HRQoL) impact in women who have sex with men (WSM), men who have sex with women (MSW), and men who have sex with men (MSM). METHODS: Semistructured qualitative interviews were conducted (April 2021-March 2022) among US adults with recent (≤6 months) confirmed gonorrhea. Concept saturation was achieved, confirming adequate participant numbers to meet objectives. Elicited symptom and HRQoL impacts were used to develop a conceptual disease model. Common patient-reported outcome measure (PROM) items were compared with elicited concepts. RESULTS: Thirty-two participants (15 WSM, 8 MSW, and 9 MSM) were included. Eight were asymptomatic, 6 had repeat infections, and 5 women and 4 men had complications (i.e., infertility or pelvic inflammatory disease, and urethritis, respectively).The most frequently reported symptoms were vaginal discharge (n = 11 of 12), dysuria (n = 10 of 12), and abdominal/vaginal pain (n = 9 of 12) for symptomatic WSM; dysuria (n = 5 of 6) and penile discharge (n = 4 of 6) for symptomatic MSW; and throat, testicular, or rectal pain (n = 4 of 6) for symptomatic MSM. All (symptomatic and asymptomatic) participants reported HRQoL impacts including negative emotional experiences (n = 31 of 32), and interference with sexual activity (n = 30 of 32), relationships (n = 24 of 32), and social life (n = 17 of 32). Concepts were poorly represented in existing PROMs. CONCLUSIONS: Participants reported a diverse range of symptoms and HRQoL impacts, to help inform the value of new treatment and prevention options. More research is needed to quantify patient burden and develop PROMs.

Development of an integrated conceptual model of multiple sclerosis spasticity.

PURPOSE: Spasticity is common in multiple sclerosis (MS), often leading to functional limitations and disability. We developed a conceptual model of spasticity in MS integrating expert opinion, recent literature, and experiences of clinicians and people with MS spasticity. METHODS: A conceptual model was developed based on a targeted literature review of articles published between 2014 and 2019, followed by input from clinicians, then input from participants with MS spasticity. Multidisciplinary experts on spasticity provided guidance at each step. RESULTS: Key concepts of the integrated spasticity conceptual model included: moderators; triggers; modifiers; treatment; objective manifestations; subjective experience; physical, functional, social, and emotional/psychological impacts; and long-term consequences. Participants with MS spasticity most frequently endorsed spasms, tightness, and pain as descriptors of spasticity. Some participants with MS spasticity had difficulty distinguishing spasticity from other MS symptoms (e.g. muscle weakness). Some triggers, emotional/psychological impacts, and long-term consequences of spasticity reported by participants with MS spasticity were not previously identified in the published literature. CONCLUSIONS: This conceptual model of spasticity, integrating published literature with the experience of clinicians, people with MS spasticity, and experts, demonstrates the complex, multidimensional nature of MS spasticity. This model may be used to improve clinician-patient dialogue, research, and patient care.

Many people with multiple sclerosis (MS) have spasticity, generally in the lower limbs, but this symptom is complex and multidimensional and therefore difficult to characterize.MS spasticity may be influenced by moderators, triggers, modifiers, and treatment, all of which can affect objective measures and the subjective experience of spasticity.MS spasticity can have physical, functional, social, and emotional/psychological impacts as well as long-term consequences that can affect rehabilitation and ultimately reduce health-related quality of life for people with MS.Given that people with MS may view spasticity differently than their rehabilitation providers, providers should ask patients about their spasticity, including their moderators, triggers, modifiers, experience, impacts, long-term consequences, and effects on quality of life.This conceptual model provides a framework to improve clinician-patient dialogue, research, and rehabilitation for MS spasticity.

Patient experiences with hypertrophic cardiomyopathy: a conceptual model of symptoms and impacts on quality of life.

BACKGROUND: Hypertrophic cardiomyopathy (HCM) is a primary myocardial disorder defined by left ventricular hypertrophy that cannot be explained by another cardiac or systemic disease. There is a general lack of knowledge about patients' perspectives on the symptoms and day-to-day limitations they experience as a result of HCM. We therefore sought an in-depth understanding of patients' experiences of obstructive (oHCM) and nonobstructive (nHCM) forms of the disease, including symptoms and their quality of life impacts, and to develop a conceptual model to capture them. METHODS: Development of the HCM conceptual model involved a web-based survey to capture patients' insights, a targeted literature review (which included relevant guidelines and patient advocacy websites), one-to-one interviews with clinical experts, and one-to-one qualitative concept elicitation interviews with patients. Key symptoms and their impacts most important to patients' experiences were identified and used to develop a conceptual model of the patient experience with HCM. RESULTS: The HCM symptoms reported by patient interviewees (n = 27) were largely consistent with findings from the patient web survey (n = 444), literature review, and interviews with three expert clinicians. The symptoms most commonly reported in patient interviews included tiredness (89%), shortness of breath (89%), shortness of breath with physical activity (89%), and dizziness/light-headedness (89%). Other symptoms commonly reported included chest pain (angina) (70%), chest pain (angina) with physical exertion (70%), and palpitations (fluttering or rapid heartbeat) (81%). The most commonly reported impacts of HCM symptoms on patients' lives included limitations to physical activities (78%), emotional impacts, including feeling anxious or depressed (78%), and impacts on work (63%). Symptoms and impacts were similar for both oHCM and nHCM. CONCLUSIONS: A conceptual model was developed, which identifies the core symptoms that patients with oHCM and nHCM reported as most frequent and most important: shortness of breath, palpitations, fatigue/tiredness, dizziness/light-headedness, and chest pain, as well as the impacts those symptoms have on patients' lives. This HCM conceptual model reflecting patients' experiences and perspectives was used in the development of a patient-reported outcomes instrument for use in clinical trials and it may also help inform the clinical management of HCM.

Evaluation of the psychometric properties of the Early Morning Symptoms of COPD Instrument (EMSCI).

Background: Early morning respiratory symptoms impact quality of life and are often the most troublesome for patients with COPD. Reduction in symptoms and their impact are important treatment outcomes for COPD. The Early Morning Symptoms of COPD Instrument (EMSCI) is a daily diary designed to collect patients' report of the occurrence, severity, and impact of morning COPD symptoms. Methods: To assess the psychometric properties of the EMSCI, a split-half sample of data from a COPD clinical trial where participants completed the EMSCI daily was used for conducting descriptive statistics, factor analyses, and Rasch model analyses to examine item performance and inform scoring. Once the final scoring algorithm was determined, data from the second split-half sample were used to examine the properties of the EMSCI. Test-retest reliability was assessed using intraclass correlation coefficient (ICC). Correlations with other study assessments were used to evaluate convergent and known-groups validity. Results: Data from 1,663 patients with COPD aged 40-93 years were analyzed. Factor analysis and Rasch analysis confirmed a one-factor structure for the 6 individual symptom items. Item analyses supported the generation of 4 scores. All scores demonstrated good test-retest reliability: 6-item symptom severity (ICC, 0.84); overall morning symptom severity (ICC, 0.84); activity limitation (ICC, 0.85); and rescue medication (ICC, 0.62). Significant correlations between EMSCI scores, St George's Respiratory Questionnaire scores, and EXAcerbations of Chronic pulmonary disease Tool (EXACT)-Respiratory Symptoms scores supported the tool's convergent validity. Significant differences (p<0.0001) in all EMSCI domain scores were found between known-groups based on median split St George's Respiratory Questionnaire and EXACT-Respiratory Symptoms scores. Conclusion: The EMSCI consists of 4 scores: 6-item symptom severity, overall symptom severity, activity limitation, and rescue medication. The EMSCI is a reliable and valid instrument for evaluating patients' experience of early morning COPD symptoms.

PATIENT-CENTERED DECISION MAKING: LESSONS FROM MULTI-CRITERIA DECISION ANALYSIS FOR QUANTIFYING PATIENT PREFERENCES.

OBJECTIVES: Patient preferences should be a central consideration in healthcare decision making. However, stories of patients challenging regulatory and reimbursement decisions has led to questions on whether patient voices are being considered sufficiently during those decision making processes. This has led some to argue that it is necessary to quantify patient preferences before they can be adequately considered. METHODS: This study considers the lessons from the use of multi-criteria decision analysis (MCDA) for efforts to quantify patient preferences. It defines MCDA and summarizes the benefits it can provide to decision makers, identifies examples of MCDAs that have involved patients, and summarizes good practice guidelines as they relate to quantifying patient preferences. RESULTS: The guidance developed to support the use of MCDA in healthcare provide some useful considerations for the quantification of patient preferences, namely that researchers should give appropriate consideration to: the heterogeneity of patient preferences, and its relevance to decision makers; the cognitive challenges posed by different elicitation methods; and validity of the results they produce. Furthermore, it is important to consider how the relevance of these considerations varies with the decision being supported. CONCLUSIONS: The MCDA literature holds important lessons for how patient preferences should be quantified to support healthcare decision making.

The burden of endometriosis symptoms on health-related quality of life in women in the United States: a cross-sectional study.

INTRODUCTION: To examine the symptomatic burden of endometriosis on health-related quality of life (HRQL) in women in the United States (US). METHODS: A cross sectional web-based survey study was conducted among women using survey panels. The survey included study-specific questions and standardized HRQL questionnaires. Participants reviewed a list of endometriosis symptoms and selected all symptoms they had ever or were currently experiencing. For current symptoms, participants rated the severity and bothersomeness of each symptom. Participants completed the endometriosis health profile (EHP-30) core questionnaire. Descriptive analyzes were performed and multivariate regressions were run with each EHP subscale as a dependent variable to examine the impact of symptoms while controlling for age and comorbid conditions. RESULTS: Mean age of the 1269 women was 34.3 ± 0.3; 78% were white. At least 75% reported having ever experienced: pelvic pain/cramping during their menstrual period, anxiety/stress, lower back pain or fatigue/weariness/anemia. EHP-30 scores ranged from 33.6 (95% CI: 31.4, 35.8) (social support) to 37.8 (95% CI: 35.5, 40.1) (control and powerlessness), indicating moderate HRQL impact. For all but one domain and one symptom, EHP-30 scores were significantly higher (worse) for women who had individual endometriosis-related symptoms than for those who did not. EHP-30 scores consistently deteriorated with each increase in the number of symptoms experienced and by increasing perceived disease severity. Pelvic pain/cramping during menstrual period, irregular periods and general abdominal pain were significantly associated with the EHP-30 domain scores in the regression models. CONCLUSION: Experiencing endometriosis symptoms is associated with lower HRQL. Importantly, as symptom severity and number of symptoms increase, HRQL further deteriorates.

Evaluation of COPD Treatments: A Multicriteria Decision Analysis of Aclidinium and Tiotropium in the United States.

BACKGROUND: Comparisons of the use of aclidinium bromide and tiotropium bromide for the treatment of chronic obstructive pulmonary disease often concentrate on key end points (exacerbations) at the expense of other benefits and risks. Multicriteria decision analysis (MCDA) can help overcome this by using stakeholder preferences to combine multiple end points into an overall value estimate. OBJECTIVES: To evaluate the use of aclidinium bromide twice daily via Pressair™ (AstraZeneca Pharmaceuticals LP, Wilmington, DE) and of tiotropium once daily via HandiHaler® (Boehringer Ingelheim Pharmaceuticals, Inc., Ridgefield, CT) from the perspective of patients using MCDA. METHODS: Literature reviews and clinician engagement were used to identify value criteria. Performance of criteria was estimated from a clinical trial and clinician opinion. Scores and swing weights came from six clinicians who, during a 2-day workshop, reflected their patients' preferences. Scenario and sensitivity analyses were used to explore uncertainty in model designs and inputs. RESULTS: Fourteen criteria, covering clinical effectiveness, safety, and convenience of the treatments of chronic obstructive pulmonary disease, were identified. Exacerbations and device preloading were identified as the most important to patients; the least important was rescue medication use. Tiotropium's higher overall clinical effectiveness score was offset by aclidinium's better performance on safety and convenience outcomes. The MCDA generated a -42 (worst performance) to 100 (best performance) scale. The net impact of benefits over risks of aclidinium (38.5) exceeded that of tiotropium (13.2), and patients preferred aclidinium 79.7% of the time. CONCLUSIONS: When considering clinical benefits and risks, aclidinium and tiotropium generate similar value to patients, but when convenience criteria are considered, aclidinium may be preferred. Further work is required to replicate these results, including eliciting preferences directly from patients.

Amplifying Each Patient's Voice: A Systematic Review of Multi-criteria Decision Analyses Involving Patients.

BACKGROUND: Qualitative methods tend to be used to incorporate patient preferences into healthcare decision making. However, for patient preferences to be given adequate consideration by decision makers they need to be quantified. Multi-criteria decision analysis (MCDA) is one way to quantify and capture the patient voice. The objective of this review was to report on existing MCDAs involving patients to support the future use of MCDA to capture the patient voice. METHODS: MEDLINE and EMBASE were searched in June 2014 for English-language papers with no date restriction. The following search terms were used: 'multi-criteria decision*', 'multiple criteria decision*', 'MCDA', 'benefit risk assessment*', 'risk benefit assessment*', 'multicriteri* decision*', 'MCDM', 'multi-criteri* decision*'. Abstracts were included if they reported the application of MCDA to assess healthcare interventions where patients were the source of weights. Abstracts were excluded if they did not apply MCDA, such as discussions of how MCDA could be used; or did not evaluate healthcare interventions, such as MCDAs to assess the level of health need in a locality. Data were extracted on weighting method, variation in patient and expert preferences, and discussion on different weighting techniques. RESULTS: The review identified ten English-language studies that reported an MCDA to assess healthcare interventions and involved patients as a source of weights. These studies reported 12 applications of MCDA. Different methods of preference elicitation were employed: direct weighting in workshops; discrete choice experiment surveys; and the analytical hierarchy process using both workshops and surveys. There was significant heterogeneity in patient responses and differences between patients, who put greater weight on disease characteristics and treatment convenience, and experts, who put more weight on efficacy. The studies highlighted cognitive challenges associated with some weighting methods, though patients' views on their ability to undertake weighting tasks was positive. CONCLUSION: This review identified several recent examples of MCDA used to elicit patient preferences, which support the feasibility of using MCDA to capture the patient voice. Challenges identified included, how best to reflect the heterogeneity of patient preferences in decision making and how to manage the cognitive burden associated with some MCDA tasks.

Evaluation of the psychometric properties of the Nighttime Symptoms of COPD Instrument.

BACKGROUND: Nighttime symptoms can negatively impact the quality of life of patients with chronic obstructive pulmonary disease (COPD). The Nighttime Symptoms of COPD Instrument (NiSCI) was designed to measure the occurrence and severity of nighttime symptoms in patients with COPD, the impact of symptoms on nighttime awakenings, and rescue medication use. The objective of this study was to explore item reduction, inform scoring recommendations, and evaluate the psychometric properties of the NiSCI. METHODS: COPD patients participating in a Phase III clinical trial completed the NiSCI daily. Item analyses were conducted using weekly mean and single day scores. Descriptive statistics (including percentage of respondents at floor/ceiling and inter-item correlations), factor analyses, and Rasch model analyses were conducted to examine item performance and scoring. Test-retest reliability was assessed for the final instrument using the intraclass correlation coefficient (ICC). Correlations with assessments conducted during study visits were used to evaluate convergent and known-groups validity. RESULTS: Data from 1,663 COPD patients aged 40-93 years were analyzed. Item analyses supported the generation of four scores. A one-factor structure was confirmed with factor analysis and Rasch analysis for the symptom severity score. Test-retest reliability was confirmed for the six-item symptom severity (ICC, 0.85), number of nighttime awakenings (ICC, 0.82), and rescue medication (ICC, 0.68) scores. Convergent validity was supported by significant correlations between the NiSCI, St George's Respiratory Questionnaire, and Exacerbations of Chronic Obstructive Pulmonary Disease Tool-Respiratory Symptoms scores. CONCLUSION: The results suggest that the NiSCI can be used to determine the severity of nighttime COPD symptoms, the number of nighttime awakenings due to COPD symptoms, and the nighttime use of rescue medication. The NiSCI is a reliable and valid instrument to evaluate these concepts in COPD patients in clinical trials and clinical practice. Scoring recommendations and steps for further research are discussed.

Saying sorry: shifting obligation after conciliatory acts satisfies perpetrator group members.

How are intergroup conciliatory acts (apologies and reparations) evaluated by members of the perpetrator group offering them? This research tests whether these outcomes can be predicted by obligation shifting: the perception that a conciliatory act has shifted the onus away from the perpetrators and onto the victim group. Four experiments in different contexts examined 3 possible outcomes for members of the perpetrator group: satisfaction with the act, negative feelings toward the victims, and support for future assistance. Across all 4 experiments, perceptions of obligation shifting predicted satisfaction with conciliatory acts, as did the perception that the ingroup's image had improved. Furthermore, obligation shifting alone related to more negative feelings about the victims and predicted reduced support for further acts of assistance. Image improvement perceptions did not show these effects, and sometimes were related to less negative feelings about the victims. Directly manipulating impressions of obligation shifting and image improvement (Experiment 3) showed these relationships were causal. When there were differences between types of acts on the 3 outcome variables, obligation shifting and image perceptions mediated these relationships. The negative implications of obligation shifting, as well as the more encouraging role of image improvement perceptions, are discussed.

Ingroup glorification, moral disengagement, and justice in the context of collective violence.

What aspects of ingroup identification can lead people to resist justice for the victims of their ingroup's mistreatment? In three studies carried out in the United States and United Kingdom, in which participants read reports of mistreatment of prisoners and civilians by coalition troops in the Iraq war, ingroup glorification, but not ingroup attachment or other individual-difference variables, was a key predictor of lesser demands for justice, but only when the perpetrators belonged to the ingroup. This effect of glorification was mediated by two moral disengagement mechanisms focusing on the outgroup: minimization of the emotional suffering of the victims' families and explicit dehumanization of the victim group. These findings further reinforce the difference between glorification and other forms of ingroup identification, demonstrating that glorification is problematic in maintaining and fostering intergroup relations because of its connection to moral disengagement.