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Background: There are limited tools to measure the burden of disease and effectiveness of medical/surgical interventions in patients with cryptoglandular fistulas. The aim of this study was to explore concepts that are relevant and important to patients with complex cryptoglandular fistulas (CCF) and to develop a patient-centred, disease-specific, patient-reported outcome measure (PROM) to assess symptom burden and impacts of CCF. Methods: A targeted literature review was conducted, followed by one-to-one telephone interviews with five colorectal surgeons (USA, n = 3; UK, n = 1; Spain, n = 1) and 20 US adult patients with CCF to inform the development of a conceptual model and a CCF-specific PROM. The targeted literature review informed the development of the preliminary conceptual model and identified a PROM in the literature that was used as a reference to generate the draft CCF-specific PROM. The colorectal surgeon interviews provided insights on the experience of patients with CCF to refine the conceptual model, formulate probing questions for use in patient interviews, and to develop the draft CCF-specific PROM. Patients' descriptions of their experiences with symptoms and the impacts on their lives and evaluation of the draft CCF-specific PROM in concept elicitation and cognitive interviews were used to develop the final conceptual model and final CCF-specific PROM. Results: Ten symptoms (odour, pain during bowel movement, abscess, post-operative pain, discharge/drainage/leakage, anal/perianal pain, inflammation/swelling, skin irritation, bleeding and itchiness) and 11 impacts (discomfort, inability to exercise, embarrassment, difficulty sitting, worry about disease, adapted life to maintain hygiene, negatively impacted social life/isolation, inability to perform daily activities, reduced interest in sex, negatively impacted intimate relationships and negatively impacted mood) were reported as most salient by patients. The patient experience, clinician perspective, and literature review provided input to item generation. Evaluation of relevance and patient understanding through cognitive interviews with patients provided evidence for the content validity of the new patient-reported outcome measure: the 20-item Complex Cryptoglandular Fistula Questionnaire™ (CCFQ-20™). Conclusion: The CCFQ-20™ is a new clinician-guided, patient-validated, disease-specific patient-reported outcome measure that measures disease impact and quality of life in patients with CCF. [ABSTRACT FROM AUTHOR]

The EA-QOL questionnaire measures quality-of-life specifically for children born with esophageal atresia (EA) aged 8–18 and was completed in Sweden and Germany. This study aimed to describe an international collaborative initiative to establish a semantically equivalent linguistic version of the EA-QOL questionnaires in 12 new countries. The 24-item EA-QOL questionnaire was translated into the target languages and the translated questionnaire was evaluated through cognitive debriefing interviews with children with EA aged 8–18 and their parents in each new country. Participants rated an item as to whether an item was easy to understand and sensitive/uncomfortable to answer. They could choose not to reply to a non-applicable/problematic item and provide open comments. Data were analyzed using predefined psychometric criteria; item clarity ≥80%, item sensitive/uncomfortable to answer ≤20%, item feasibility(missing item responses ≤5%). Decision to improve any translation was made by native experts–patient stakeholders and the instrument developer. Like in Sweden and Germany, all items in the cross-cultural analysis of child self-report (ntot = 82, 4–10 children/country) met the criteria for item clarity in all 12 new countries, and in parent-report (ntot = 86, 5–10 parents/country) in 8/12 countries. All items fulfilled the criteria for sensitive/uncomfortable to answer (child-report 1.2–9.9%; parent-report 0–11.6%) and item feasibility. Poor translations were resolved. Hence, this study has established semantically equivalent linguistic versions of the EA-QOL questionnaire for use in children aged 8–18 with repair of EA in and across 14 countries. [ABSTRACT FROM AUTHOR]

Background: There is considerable burden of illness in hereditary angioedema (HAE). However, instruments to assess health-related quality of life (HRQoL) in HAE are limited. The Angioedema Quality of Life Questionnaire (AE-QoL) was developed to measure HRQoL in patients with recurrent angioedema; the validity of the AE-QoL in patients with HAE is described. Methods: To identify disease-related experiences with a focus on the impact of HAE on HRQoL, interviews were conducted with a group of clinician experts and patients with HAE from Canada, France, Germany, Spain, the United Kingdom, and the United States, along with a targeted literature review. Concepts were mapped to the AE-QoL to assess item relevance, interpretation, and conceptual coverage. Cognitive interviews assessed item clarity and relevance. A psychometric validation was performed using data from a phase 3 trial. Results: Interviews were conducted with seven clinicians and 40 adult patients. Patients reported 35 unique impacts of HAE on their lives, the most frequent being on work/school, social relationships, physical activities, and emotions, particularly fear/worrying and anxiety. Saturation for these impacts was reached, and all concepts covered in the AE-QoL were reported during the interviews. Patients agreed that the questionnaire items and response options were clear and relevant, and the 4-week recall period was appropriate. The psychometric validation included data from 64 patients. For AE-QoL total scores, excellent internal consistency (Cronbach's alpha > 0.90), test-retest reliability (intraclass coefficient > 0.80), convergent validity with the Sheehan Disability Scale (r = 0.663), divergent validity with the EQ-5D-5L index (r = 0.292) and EQ-VAS (r = 0.337), and known-groups validity (p < 0.0001; ɳ2 = 0.56) were demonstrated. Conclusions: Qualitative and psychometric analyses showed that the AE-QoL is a reliable and valid instrument for measuring HRQoL in adult patients with HAE from six countries. [ABSTRACT FROM AUTHOR]

Introduction: Treatment decisions in older adults with acute myeloid leukemia (AML) are challenging, particularly for those who are not candidates for intensive chemotherapy (IC), and the trade-offs patients, their families and physicians consider when choosing a treatment option are not well understood. This qualitative research explored the value of extending survival and the treatment decision-making process from a multi-stakeholder perspective. Methods: Overall, 28 patients with AML (≥ 65 years old, unsuitable for IC), 25 of their relatives and 10 independent physicians from the US, UK and Canada took part in one-on-one, 60-minute qualitative interviews. Results: Across all stakeholders, improved health-related quality of life (HRQoL), extended survival and relief of AML symptoms were recognized as most important in AML treatment decision-making. However, extending survival in 'good health' was more important than extending survival alone, particularly because of the extra time it gives patients and their relatives together, and allows patients to achieve important goals. Patients' limited understanding of available treatment options, paired with incorrect perceptions of treatment side effects, impacted their involvement in the treatment decision-making process. Patients and physicians perceived physicians to have the most influence in the decision-making process despite their priorities not always aligning. Conclusion: These findings illustrate the importance of having structured discussions which explicitly assess patients' goals and their understanding and expectations of treatments and also the need for patient friendly resources about the lived experience of AML and available treatment options. These measures will help to ensure that patients are fully involved in the shared decision-making process. [ABSTRACT FROM AUTHOR]

Background: Hemophagocyti.c lymphohistiocytosis (HLH) is a rare and severe disorder characterized by abnormal activation of the immune system. Primary HLH causes prolonged fever, spleen and liver enlargement, and organ dysfunction, usually in infancy and early childhood and is fatal if left untreated. As effective treatment options emerge, such as emapalumab-lzsg, Health Technology Assessment bodies around the world will assess them in terms of cost-effectiveness. This study was designed to estimate quality of life weights (utilities) for such analyses. Methods: Vignettes were developed describing HLH treatment related health states. Health states included active HLH, HLH plus neurological symptoms, receiving chemotherapy, undergoing stem cell transplant (SCT), graft versus host disease (GVHD), cure and end of life care. The vignettes were based on information from in depth interviews with clinical specialists; and qualitative research with four parents of children with primary HLH aged between 1 and 18 years old. The vignettes were then assessed in time trade off (TTO) interviews with members of the UK general public in one on one face to face interviews with trained, experienced interviewers. Preference data were analysed using the generalised estimating equations framework. Results: Detailed qualitative data captured the substantial burden of this disease for young children. One hundred participants completed the TTO interviews. The utility score for Active HLH was estimated as 0.32 (95% CI, 0.24 to 0.39). Values for other states were HLH plus neurological symptoms (0.27, 95%CI 0.18–0.35), receiving chemotherapy (0.26, 95%CI 0.17–0.34), undergoing SCT (0.18, 95%CI 0.07–0.28), GVHD (0.07, 95%CI -0.04-0.17), cure (0.72, 95%CI 0.67–0.77) and end of life care (− 0.17, 95%CI -0.27- -0.07). Conclusions: This study has estimated utility weights for seven different HLH related states which are based on detailed input from carers and physicians and have good face validity. There are few other options for collecting these data in an ultra-rare setting. [ABSTRACT FROM AUTHOR]

Background Due to the climate crisis, it is increasingly evident that countries will have to decarbonize. Healthcare, which has a large carbon footprint and uses vast quantities of resources, will have to undergo significant transformation. In this research, we sought the ideas of leading thinkers in the field, to address the question of how health systems can provide high-quality care in a carbon-constrained world. Methods Semi-structured, qualitative in-depth interviews with 15 healthcare thought leaders from Australia, the UK, the USA and New Zealand. The interviews were transcribed and analysed by matrix display and thematic analysis. Results 'Green' initiatives such as improving energy efficiency and implementing travel plans will be insufficient to achieve the scale of decarbonization required. According to the thought leaders in our study, it is likely that greater carbon and resource savings will come from thinking much more broadly about innovative models of care and using 'new' sources of 'value' such as 'people' and 'relationships'. Conclusions Using human resources and human interactions as low-carbon sources of value in healthcare are promising models. [ABSTRACT FROM AUTHOR]

Background: Epidemiologic evidence suggests that fruit consumption may lower, the risk of cardiovascular diseases through blood pressure (BP)-lowering effects; little is known on the independent effect of raw fruit and fruit juice on BP. Objective: The objective was to quantify associations of raw fruit and fruit juice consumption with BP by using cross-sectional data from the INTERnational study on MAcro/micronutrients and blood Pressure (INTERMAP) of 4680 men and women aged 40-59 y from Japan, China, the United Kingdom, and the United States. Design: During 4 visits, 8 BP, four 24-h dietary recalls, and two 24-h urine samples were collected. Country-specific multivariate-controlled linear regression coefficients, including adjustment for urinary sodium excretion, were estimated and pooled, weighted by inverse of their variance. Results: The average total raw fruit consumption varied from a mean ± SD of 52 ± 65 g/1000 kcal in the United States to 68 ± 70 g/1000 kcal in China. Individual raw fruit intake was not associated with BP in pooled analyses for all countries or in participants from Western countries, although a positive association with diastolic BP was observed in East Asian participants (per 50 g/1000 kcal; 0.37 mm Hg; 95% CI: 0.02, 0.71). Positive relationships with diastolic BP were found for citrus fruit intake in Western consumers (per 25 g/1000 kcal; 0.47 mm Hg; 95% CI: 0.12, 0.81) and for apple intake in East Asian consumers (0.40 mm Hg; 95% CI: 0.03, 0.78). Among East Asian banana consumers, banana intake was inversely associated with diastolic BP (- 1.01 mm Hg; 95% CI: -1.88, -0.02). Fruit juice intake, which was negligible in Asia, was not related to BP in Western countries. Conclusion: Consistent associations were not found between raw fruit and fruit juice consumption of individuals and BP. This observational study was registered at www.clinicaltrials.gov as NCT00005271. [ABSTRACT FROM AUTHOR]

Background Although on the decline, smoking-related fires remain a leading cause of fire death in the United States and United Kingdom and account for over 10% of fire-related deaths worldwide. This has prompted lawmakers to enact legislation requiring manufacturers to implement reduced ignition propensity (RIP) safety standards for cigarettes. The current research evaluates how implementation of RIP safety standards in different countries influenced smokers' perceptions of cigarette self-extinguishment, frequency of extinguishment, and the impact on consumer smoking behaviors, including cigarettes smoked per day and planning to quit. Methods Participants for this research come from Waves 3 through 8 of the International Tobacco Control (ITC) Four Country Survey conducted longitudinally from 2004 through 2011 in the United States, United Kingdom, Australia, and Canada. Results Perceptions of cigarette self-extinguishment and frequency of extinguishment increased concurrently with an increase in the prevalence of RIP safety standards for cigarettes. Presence of RIP safety standards was also associated with a greater intention to quit smoking, but was not associated with the number of cigarettes smoked per day. Intention to quit was higher among those who were more likely to report that their cigarettes self-extinguish sometimes and often, but we found no evidence of an interaction between frequency of extinguishment and RIP safety standards on quit intentions. Conclusions Overall, because these standards largely do not influence consumer smoking behavior, RIP implementation may significantly reduce the number of cigarette-related fires and the associated death and damages. Further research should assess how implementation of RIP safety standards has influenced smoking-related fire incidence, deaths, and other costs associated with smoking-related fires. [ABSTRACT FROM AUTHOR]