Inhibition of Crif1 protects fatty acid-induced POMC neuron-like cell-line damage by increasing CPT-1 function.

Hypothalamic proopiomelanocortin (POMC) neurons are sensors of signals that reflect the energy stored in the body. Inducing mild stress in proopiomelanocortin neurons protects them from the damage promoted by the consumption of a high-fat diet, mitigating the development of obesity; however, the cellular mechanisms behind these effects are unknown. Here, we induced mild stress in a proopiomelanocortin neuron cell line by inhibiting Crif1. In proopiomelanocortin neurons exposed to high levels of palmitate, the partial inhibition of Crif1 reverted the defects in mitochondrial respiration and ATP production; this was accompanied by improved mitochondrial fusion/fission cycling. Furthermore, the partial inhibition of Crif1 resulted in increased reactive oxygen species production, increased fatty acid oxidation, and reduced dependency on glucose for mitochondrial respiration. These changes were dependent on the activity of CPT-1. Thus, we identified a CPT-1-dependent metabolic shift toward greater utilization of fatty acids as substrates for respiration as the mechanism behind the protective effect of mild stress against palmitate-induced damage of proopiomelanocortin neurons.NEW & NOTEWORTHY Saturated fats can damage hypothalamic neurons resulting in positive energy balance, and this is mitigated by mild cellular stress; however, the mechanisms behind this protective effect are unknown. Using a proopiomelanocortin cell line, we show that under exposure to a high concentration of palmitate, the partial inhibition of the mitochondrial protein Crif1 results in protection due to a metabolic shift warranted by the increased expression and activity of the mitochondrial fatty acid transporter CPT-1.

Stroke rehabilitation pathways during the first year: A cost-effectiveness analysis from a cohort of 460 individuals.

BACKGROUND: Stroke burden challenges global health, and social and economic policies. Although stroke recovery encompasses a wide range of care, including in-hospital, outpatient, and community-based rehabilitation, there are no published cost-effectiveness studies of integrated post-stroke pathways. OBJECTIVE: To determine the most cost-effective rehabilitation pathway during the first 12 months after a first-ever stroke. METHODS: A cohort of people in the acute phase of a first stroke was followed after hospital discharge; 51 % women, mean (SD) age 74.4 (12.9) years, mean National Institute of Health Stroke Scale score 11.7 (8.5) points, and mode modified Rankin Scale score 3 points. We developed a decision tree model of 9 sequences of rehabilitation care organised in 3 stages (3, 6 and 12 months) through a combination of public, semi-public and private entities, considering both the individual and healthcare service perspectives. Health outcomes were expressed as quality-adjusted life years (QALY) over a 1-year time horizon. Costs included healthcare, social care, and productivity losses. Sensitivity analyses were conducted on model input values. RESULTS: From the individual perspective, pathway 3 (Short-term Inpatient Unit ¼ Community Clinic) was the most cost-effective, followed by pathway 1 (Rehabilitation Centre ¼ Community Clinic). From the healthcare service perspective, pathway 3 was the most cost-effective followed by pathway 7 (Outpatient Hospital ¼ Private Clinic). All other pathways were considered strongly dominated and excluded from the analysis. The total 1-year mean cost ranged between €12104 and €23024 from the individual's perspective and between €10992 and €31319 from the healthcare service perspective. CONCLUSION: Assuming a willingness-to-pay threshold of one times the national gross domestic product (€20633/QALY), pathway 3 (Short-term Inpatient Unit ¼ Community Clinic) was the most cost-effective strategy from both the individual and healthcare service perspectives. Rehabilitation pathway data contribute to the development of a future integrated care system adapted to different stroke profiles.

New thymol-derived triazole exhibits promising activity against Trichophyton rubrum.

Fungal infections have emerged worldwide, and azole antifungals are widely used to control these infections. However, the emergence of antifungal resistance has been compromising the effectiveness of these drugs. Therefore, the objective of this study was to evaluate the antifungal and cytotoxic activities of the nine new 1,2,3 triazole compounds derived from thymol that were synthesized through Click chemistry. The binding mode prediction was carried out by docking studies using the crystallographic structure of Lanosterol 14α-demethylase G73E mutant from Saccharomyces cerevisiae. The new compounds showed potent antifungal activity against Trichophyton rubrum but did not show relevant action against Aspergillus fumigatus and Candida albicans. For T. rubrum, molecules nº 5 and 8 showed promising results, emphasizing nº 8, whose fungicidal and fungistatic effects were similar to fluconazole. In addition, molecule nº 8 showed low toxicity for keratinocytes and fibroblasts, concluding that this compound demonstrates promising characteristics for developing a new drug for dermatophytosis caused by T. rubrum, or serves as a structural basis for further research.

Usage and impact of patient-reported outcomes in epilepsy.

BACKGROUND: The use of patient-reported outcomes (PRO) in clinical practice is gaining increasing attention. This study aimed to provide a critical assessment of the current state-of-the-art and beliefs about the use of PRO in the management of people with epilepsy across some European countries. METHODS: Structured interviews were conducted with European experts to collect insights about (I) the personal experience with PRO; (II) the value and impact of PRO in the decision-making process at the national level; and (III) the interest for and use of PRO by national health authorities. RESULTS: Nine neurologists (Austria, Belgium, Czechia, Denmark, France, Greece, Italy, Poland, and United Kingdom), three health economists (Portugal, Romania, and Sweden), and one epidemiologist (Slovakia) participated. They all stated that PRO are collected at their own countries in the context of clinical trials and/or specific projects. During everyday clinical practice, PRO are collected routinely/almost routinely in Austria and Sweden and only at the discretion of the treating physicians in Czechia, Denmark, France, Greece, and Portugal. There was complete consensus about the favorable impact that the PRO can have in terms of clinical outcomes, healthcare resources utilization, and general patient satisfaction. Only participants from Portugal and Sweden answered that the PRO are perceived as very important by the National Health Authorities of their respective countries. CONCLUSIONS: Differences exist in attitudes and perspectives about PRO in epilepsy across Europe. An active plan is warranted to harmonize the measurement of PRO and ensure they can be relevant to people with epilepsy and health services.

Health and well-being of the Portuguese citizens: impacts of the COVID-19.

BACKGROUND: COVID-19 pandemic placed unprecedented pressure on societies and healthcare systems around the world. Over the last years, measures imposed in almost all countries dealing with the pandemic sent the entire world into an extensive crisis and thus into a deep global recession. Since the outbreak began, many European countries have faced three/four waves of pandemic. Portugal has mainly dealt with three waves (March/April'2020; October/November'2020; January/February'2021), the third being the deadliest one. The purpose of this article is to provide evidence on the impact of the COVID-19 on health-related quality of life (HRQol) and well-being (W-B) of Portuguese citizens. It aims to (i) characterize these outcomes during the COVID-19 pandemic; (ii) compare them to pre-COVID-19 Portuguese population; and (iii) identify the social determinants that may affect these outcomes during the COVID-19 pandemic. METHODS: This study used data from a survey that collected data on HRQoL, W-B, satisfaction with life, economic and labour impacts, access to healthcare, mental and physical health, amongst others. The survey was implemented by telephone to a representative random sample of 1,255 respondents from the general adult Portuguese population, stratified by sex, age group and region. Data was collected during the end of the second national lockdown. For comparison purposes, we have also used two other representative databases from the general Portuguese population: (i) data from before the pandemic (n = 1,006); and (ii) data from a survey conducted during the first lockdown, (n = 904). RESULTS: Looking at health and access to healthcare, 4% of citizens had their surgeries postponed or cancelled because of COVID-19, more than a quarter had medical appointments or complementary exams postponed or cancelled, with 7% over 65 years old with surgeries cancelled or postponed and 32% medical appointments. COVID-19 pandemic also impacted negatively on the HRQoL of citizens, especially in the first lockdown. Half of the respondents reported feeling nervous, anxious, or on edge, about 45% of citizens felt sad or depressed. Sleeping problems were reported for almost 39% of citizens, and loneliness is reported by 29% of citizens. For about 70-85% of citizens, these feelings were more so than before the pandemic. Citizens with fair/strong economic stability were the most economically affected by the pandemic. CONCLUSIONS: We provided evidence on the impact of COVID-19 on health and W-B of Portuguese citizens. Their health was worse than before the pandemic and the access to healthcare was highly affected.

Circulating MicroRNAs as Specific Biomarkers in Atrial Fibrillation: A Meta-Analysis.

Atrial fibrillation (AF) is the most frequently occurring supraventricular arrhythmia. Although microRNAs (miRNAs) have been associated with AF pathogenesis, standard protocols for quantifying and selecting specific miRNAs for clinical use as biomarkers should be optimized. In this study, we evaluated the clinical application of miRNAs as biomarkers for the prognosis and diagnosis of AF. Literature searches were conducted on PubMed, Cochrane Library, and EMBASE. We included prospective or retrospective observational studies that had been published as of 14 February 2022; our main objective was to analyze the relationship between circulating miRNAs and AF. The data were extracted using the descriptors "Atrial fibrillation AND miRNA", "Atrial fibrillation AND diagnostic AND miRNA", and "Atrial fibrillation AND prognosis AND miRNA". No filters were applied for period delimitation, type of publication, or language. Studies using samples isolated from blood plasma and TaqMan and RT-qPCR for detecting and quantifying miRNAs were selected, and those that used atrial tissue samples were excluded. We identified 272 articles and excluded 102 duplicated articles. Two authors independently read the titles and abstracts of 170 out of 272 articles and selected 56 potential articles, 6 of which were selected for final review. Our analysis revealed a significant association between AF and miR-4798 [OR = 1.90 (95% CI 1.45-2.47)], AF and miRNA-133a [2.77 (2.73-2.82)], AF and miRNA-150 [3.77 (1.50-9.46); I2 = 70%], AF and miRNA-21 [2.23 (1.20-4.17); I2 = 99%], AF and hsa-miRNA4443 [2.32 (2.20-2.44)], and AF and miR-20a-5p [3.67 (1.42-9.49)]. The association between miRNAs and AF showed an OR of 2.51 [95% CI 1.99-3.16; I2 = 99%]. Our meta-analysis demonstrated that circulating miRNAs are potential biomarkers of AF, as they exhibit stable expression post-sample collection. In addition to regulating cellular processes, such as proliferation, differentiation, development, and cell death, miRNAs were found to be linked to arrhythmia development.

EQ-5D-5L Portuguese population norms.

OBJECTIVES: The EQ-5D-5L is a generic preference-based quality-of-life measure for which the corresponding Portuguese population value set was only recently developed. This study sought to establish EQ-5D-5L population norms for Portugal and to identify significant relationships with sociodemographic variables. METHODS: The research was based on a representative sample of Portugal's general population (n = 1006) aged 18 or older. The sample was stratified by gender, age group, and geographical region. The respondents were interviewed by telephone and asked to value their own state of health using both the EQ-5D-5L description system and the EuroQol-Visual Analogue Scale (EQ VAS). RESULTS: The estimated mean EQ-5D-5L index for Portugal's general population is 0.887 (standard error [SE] = 0.0051), and the EQ VAS score was estimated as 76.0 (SE = 0.640). One-third of the population reported being in the best health state (11111). Women, individuals 70 years old or more and people with low education or a chronic disease reported a lower EQ-5D-5L index score (p < 0.001). Residents in the Azores and the Algarve reported higher health utility scores. CONCLUSIONS: The EQ-5D-5L Portuguese population norms obtained can be used as reference scores. These norms are consistent with other countries' population norms. The findings facilitate clinical, economic, and policy decision-making processes and provide a fuller understanding of the Portuguese population's health-related quality of life.

From healthcare system to individuals through stroke rehabilitation pathways.Outcomes, information, and satisfaction along 12 months prospective cohort in Portugal.

BACKGROUND: The nature and quality of stroke survivor rehabilitation varies throughout Europe, including in Portugal, having not been widely monitored or benchmarked. OBJECTIVES: This study analyses the stroke care pathway from three perspectives: healthcare system, process, and patient. METHODS: The study uses data from a one-year single-center prospective cohort of first stroke patients, assessed at baseline, 3, 6, and 12 months. Care pathways and settings were described in terms of organizational model, funding, patient involvement, frequency and intensity and multidisciplinary team. Patient-level information and satisfaction were evaluated using a 10-point numeric rating scale. Kruskal - Wallis and post-hoc tests were used to compare EQ-5D-3 L, National Institute of Health Stroke Scale (NIHSS), Modified Rankin Scale (mRS), Barthel Index, Mini-Mental State Examination scores between pathways and settings. RESULTS: A total of 391 acute stroke patients, with a mean disability of 3.7 (mRS) and severity of 11.7 (NIHSS) participated. Six pathways and eight settings were described. A lack of compliance between guidelines and care was identified. There were significant differences in the four outcomes between the six pathways (p-values 0.007 to 0.020) suggesting inefficiency and inequalities, with an inadequate level of information and patient satisfaction. After post-hoc analysis, pathways 1 and 2 showed highest outcomes (p-values 0.001 to 0.002). Within settings, short-term units showed high scores, followed by rehabilitation center, outpatient hospital, and community clinic (p-values 0.001 to 0.040). CONCLUSION: A multilevel characterization of the post-stroke rehabilitation pathway showed a more complete perspective on stroke management which may contribute to future rehabilitation and stroke policies.

Charge effect of water-soluble porphyrin derivatives as a prototype to fight infections caused by Acinetobacter baumannii by aPDT approaches.

In the last decade, Acinetobacter baumannii has emerged as a pathogen associated with infections in intensive care units worldwide, especially due to its ability to resist an extensive list of antibiotics. In this context, porphyrins have emerged as an important strategy in photodynamic therapy, since they are a group of tetrapyrrolic compounds with important photochemical and photobiological activities. In this study, the antimicrobial photodynamic activity of meso-tetra(4-N-methyl-pyridyl)porphyrin (H2TMePyP+) and meso-tetra(4-sulfonatophenyl)porphyrin (H2TPPS‒) was evaluated against A. baumannii by minimum inhibitory concentration (MIC), anti-biofilm activity, and the interaction with antibiotics after exposure to white-light LED irradiation. The cationic derivative H2TMePyP+ was more potent (MIC = 0.61 µM) than H2TPPS‒, with anti-biofilm activity and increased the antimicrobial activity of ciprofloxacin and amikacin. Given these findings, the tetra-cationic porphyrins can be assumed as prototypes to optimize and develop new agents by promoting oxidative stress and inducing free radical production.

Healthcare, Clinical Factors and Rehabilitation Predicting Quality of Life in First-time Stroke Patients: A 12-month Longitudinal Study.

OBJECTIVES: One of the long-term rehabilitation goals in stroke survivors is to achieve the best health-related quality of life (HRQoL). This study analyzes the evolution of HRQoL one-year post-stroke to establish the main pre-stroke, clinical, health care and rehabilitation predictors. MATERIALS AND METHODS: This study uses patient-level data from a one-year single-center prospective cohort study of first stroke patients, assessed at baseline, 3, 6 and 12 months. A generalized linear model with a linear response determined independent predictors of HRQol with EQ-5D-3L and SF-6D. The model included age, gender, scholarity, monthly income, residence, occupation, National Institute of Health Stroke Scale (NIHSS), Modified Rankin Scale (mRS), Barthel Index (BI), Mini-Mental State Examination (MMSE), length of stay, door-to-neurological examination time, access, frequency, and satisfaction with rehabilitation care. RESULTS: A total of 391 acute stroke patients, with a mean disability of 3.7 and severity of 11.7 participated. A decline of HRQoL was observed from baseline to the first three months in both indexes, with an increase in HRQoL at 3 months until 12 months. Scores were significantly lower compared to corresponding population norms throughout follow-up, mostly affected by stroke severity, disability, rehabilitation access and frequency. Higher HRQoL was associated with lower mRS, NIHSS, age, length of stay, and with higher BI, MMSE, scholarity, occupation, and rehabilitation care. CONCLUSION: Clinical measures and rehabilitation care were the strongest HRQoL predictors of stroke survivors regardless of severity levels. These findings may contribute to the development of future health policies that focus on post-stroke recovery.

Reliability and validity of the One Arm Hop Test and Seated Medicine Ball Throw Test in young adults: A cross-sectional study.

BACKGROUND: Functional performance tests are inexpensive, accessible, and easy to apply tools that can be used to help practitioners in daily decision making process. The purpose of this study was to evaluate the reliability and validity of the One Arm Hop Test (OAHT) and Seated Medicine Ball Throw Test (SBMT) in young adults. METHODS: Cross-sectional study with a sample consisted of 59 young adults. The subjects performed the OAHT and SMBT in two moments separated by seven days and by two examiners. The Closed Kinetic Chain Upper Extremity Stability Test (CKCUEST) was performed at the second moment. The time in OAHT, distance in SMBT, mean number of touches, normalized score, and power of the CKCUEST were measured. Reliability was determined using Intraclass Correlation Coeficient (ICC) and Bland-Altman Plots. Validity was assessed via Pearson's Correlation Coefficient (r) between these tests and CKCUEST. RESULTS: We found good reliability of the OAHT between different raters (dominant limb - ICC = 0.83; non-dominant limb - ICC = 0.80) and moderate reliability between the same rater (dominant limb - ICC = 0.63; non-dominant limb - ICC = 0.62). In the SMBT we found good reliability inter-examiner (ICC = 0.84) and intra-examiner (ICC = 0.77). Low to moderate correlations with the CKCUEST were found (r < 0.70; p < 0.05). CONCLUSIONS: The OAHT and the SMBT show moderate/good reliability intra and inter-examiner, however these tests are poorly correlated with CKCUEST. The SMBT presented higher values of ICC than OAHT. A combination of the SMBT and CKCUEST is recommended in clinical practice.

Protein-losing enteropathy caused by Yersinia enterocolitica colitis.

A 7-month-old boy was admitted with acute gastro-enteritis accompanied by fever and hyponatraemic dehydration. The clinical course was complicated by severe hypokalaemia and hypo-albuminaemia with anasarca. Protein-losing enteropathy (PLE) owing to Yersinia enterocolitica colitis was diagnosed and was complicated by fungal sepsis owing to Kodomaea ohmeri. Colonoscopy demonstrated multiple diffuse ulcers and sub-epithelial haemorrhages extending from the rectum to the hepatic angle. He required prolonged nutritional support comprising partial parenteral feeding for 10 days, followed by a hypo-allergenic diet until 13 months of age when cow milk was tolerated. He was discharged on a normal diet and in good health at 19 months of age.Abbreviations AVPU scale: A alert, V verbally responsive, P painfully responsive, U unresponsive; CMV: cytomegalovirus; EBV: Epstein-Barr virus; HIV: human immunodeficiency virus; Ig: immunoglobulin; IBD: inflammatory bowel disease; IPEX: immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome; PICU: paediatric intensive care unit; PLE: protein-losing enteropathy.

Validity and reliability of the Portuguese version of the modified Migraine Disability Assessment.

BACKGROUND: Migraine Disability Assessment Scale (MIDAS) is a useful tool to measure headache-related disability. Modified MIDAS with 4-week recall period reduces recall bias and improves accuracy of the results. This study aimed at validating mMIDAS in Portuguese. METHODS: Studied population consisted of adult migraine patients attending a headache outpatient clinic. Reliability was assessed by internal consistency and reproducibility in a 3-week test-retest. Content validity was evaluated by two expert panels. Construct validity was tested by comparing mMIDAS-P index in socioeconomic and clinical patient groups and scale unidimensionality was evidenced by factor analysis. Criterion validity was tested using EQ-5D-5L and HADS. RESULTS: Ninety-two patients, 88% female, mean age of 44 years, participated. They had, in average, 9.7 headache days in previous month, pain averaging 7.5/10. About 69.9% were on a migraine prophylactic treatment, and 42.4% had severe disability; 29.4 and 13.0% showed, respectively, moderate/severe anxiety and depression. Content validity showed that mMIDAS-P is simple and clinically useful. It did not show to be determined by patient's sociodemographic characteristics and it was correlated with depression scale and EQ-5D-5L. Test-retest demonstrated high reproductive reliability and good internal consistency. CONCLUSION: mMIDAS-P is valid and reliable. We strongly recommend it for clinical and research use.

Quality of life under the COVID-19 quarantine.

BACKGROUND: COVID-19 has spread rapidly throughout the world, causing thousands of illnesses and deaths. To fight this pandemic, almost all governments and health authorities have focused on prevention. In March or April, most countries' officials imposed home quarantine and lockdown measures nationwide. PURPOSE: This study sought to assess health-related quality of life (HRQoL) and anxiety levels among people in Portugal under mandatory home quarantine due to the COVID-19 pandemic. The results were compared to the general Portuguese population's HRQoL before the COVID-19 outbreak. This research also aimed to understand the factors that can influence the respondents' HRQoL. METHODS: A sample of Portugal's population quarantined at home (n = 904) filled in an online survey comprising the Generalized Anxiety Disorder 7-item and the EQ-5D-5L and other questions about sociodemographic characteristics, feelings, duties and activities during the quarantine. The sample was weighted to mirror the general population's gender, age and education. Descriptive analyses and correlation coefficients were used to evaluation the respondents' anxiety and HRQoL. Generalised linear models were estimated to identify determinants of HRQoL during the COVID-19 quarantine. RESULTS: The results show that individuals quarantined at home reported higher anxiety and lower HRQoL levels and that people with more anxiety tended to have a lower HRQoL. Females and elderly individuals experienced the highest levels of anxiety and poorest HRQoL. In addition, HRQoL during the quarantine can be explained by various occupational and attitudinal variables, as well as sociodemographic variables. CONCLUSION: Individuals' mental health should be taken into consideration during pandemics or other emergency situations. Anxiety and other factors can decrease people's HRQoL, in conjunction with the pandemic's social and economic consequences.

High frequency of non-compliance with quality indicators during oral nutrition support in hospitalized patients.

BACKGROUND & AIMS: Quality indicators are important tools in clinical practice for assessing and monitoring the quality of care in nutritional therapy. Application of these indicators can improve care and may help to decrease the high frequency of malnutrition and associated hospital costs. Therefore, the purpose of the present study was to estimate the frequency of oral nutritional supplements (ONS) use and to evaluate compliance with the four available quality indicators in oral nutritional therapy (QIONT) in a university hospital. METHODS: A prospective study was conducted from November 2017 to May 2018, using data from all patients with an ONS prescription aged 18 years or older admitted to the medical clinical or surgical clinical wards. Four indicators were investigated, as recommended by the International Life Science Institute. RESULTS: Of the 727 hospitalized patients in the included wards, 214 were on ONS. The frequency of ONS prescription was 29.4%. Of the 4 QIONT evaluated, none achieved the goals: frequency of subjective global assessment and reassessment nutritional (48.1% and 5.6%, respectively); frequency of non-compliance for the indication of ONS (73.36%); and fasting over 24 h during ONS (50%). CONCLUSION: A high frequency of non-compliance (100% of QIONT) was observed in hospitalized patients on ONS in medical clinical and surgical clinical wards. These results will enable health professionals in the evaluated service to elaborate protocols to improve the evaluation and recording of the nutritional follow-up of hospitalized patients on ONS. This will help to improve the quality of nutritional care.

High Frequency of Non-Compliance with Quality Indicators of Enteral and Parenteral Nutritional Therapy in Hospitalized Patients.

Quality indicators in nutritional therapy (QINT) are measures of the effectiveness and quality of nutrition support. The purpose of this study was to evaluate the frequency of the QINT adequacy of Enteral Nutritional Therapy (EN) and/or Parenteral (PN) in hospitalized patients and identify the best indicators according to health professionals. A prospective study was performed, including data from patients aged 18 years or over admitted to clinical or surgical wards. The patients who had received EN and/or PN were followed from the first day of nutritional prescription until discharge. Twelve indicators were calculated, as recommended by the literature. Regarding professional opinion, the QINT adequacy was evaluated by observing its utility, simplicity, objectivity, and cost. Of the 727 hospitalized patients, 101 were on EN and/or PN. Regarding the 12 QINT evaluated, only 25% (3) achieved the goals: involuntary withdrawal of enteral feeding tube (0.01%); feeding tube occlusion or withdrawal per occlusion (0%); the measurement of energy and protein requirements (92%). A high frequency of non-compliance (75% of QINT) was observed in clinical and surgical patients on EN and/or PN. With knowledge of the six best indicators chosen by health professionals in this service, it will be possible to elaborate protocols according to the real-life situation in the institution.

Health-related quality of life in hereditary transthyretin amyloidosis polyneuropathy: a prospective, observational study.

BACKGROUND: Hereditary Transthyretin Amyloidosis Polyneuropathy is a rare life-threatening neurologic disease that imposes considerable mortality and it is associated with progressive related disabilities. In this study, we aimed to assess the effect of the disease across health-related quality of life dimensions, in both carriers of the mutation and patients, to compare health-related quality of life with general population, as well as to explore health-related quality of life prognostic factors among patients, including disease progression and treatment. METHODS: This study was a multi-institutional, longitudinal, prospective, observational study of hereditary Transthyretin Amyloidosis Polyneuropathy Portuguese adult subjects (621 asymptomatic carriers and 733 symptomatic patients) enrolled in the Transthyretin Amyloidosis Outcomes Survey. Health-related quality of life was captured with the preference-based instrument EQ-5D-3 L. For general population the dataset included all subjects enrolled in a representative national study (n = 1500). Different econometric models were specified; multivariate probit, generalized linear model and generalized estimating equations model; including demographic and clinical covariates. RESULTS: Hereditary Transthyretin Amyloidosis Polyneuropathy patients have their health status severely impaired in all quality of life dimensions and more anxiety/depression problems were found among asymptomatic carriers. No differences on utility were found between carriers and general population (p = 0.209). Among patients, the utility value is estimated to be 0.51 (0.021), a decrement of 0.27 as compared with general population utility. Higher disease duration, advanced disease stage and not receiving treatment are associated with impaired health-related quality of life. No differences were found between genders (p = 0.910) or between late (≥50 years) and early-onset patients (p = 0.254). The utility estimate ranged from 0.63 (0.009) in stage I to 0.01 (0.005) in stage IV. CONCLUSIONS: Hereditary Transthyretin Amyloidosis Polyneuropathy symptoms and progressive associated disabilities substantially decrease patient's health-related quality of life. Clinical strategies focused on health-related quality of life preservation such as close follow-up of asymptomatic carriers, prompt diagnosis and adequate, early treatment would benefit patient's long-term outcomes, slowing the progressive decline in health-related quality of life.

Conduta terapêutica em pacientes com doença pulmonar obstrutiva crônica: monoterapia ou terapia cominada? / Therapy for chronic obstructive pulmonary disease patients: monotherapy or combination therapy?

Objetivo: Comparar a dupla terapia broncodilatadora com glicopirrônio mais indacaterol à monoterapia com glicopirrônio em pacientes portadores de doença pulmonar obstrutiva crônica. Métodos: Estudo clínico prospectivo, unicêntrico, controlado, cruzado, randomizado e duplo-cego realizado com 14 pacientes com diagnóstico de doença pulmonar obstrutiva crônica grau II. Os participantes receberam cada um dos tratamentos durante 30 dias. Antes de cada terapia, realizou-se período de wash-out por 7 dias, com broncodilador de curta ação. Antes e após cada intervenção, os pacientes passaram por exame de espirometria e responderam ao questionário COPD Assessment Test. Resultados: Observou-se melhora na função pulmonar medida por meio do volume expiratório forçado no primeiro segundo de 19mL (±36) para a monoterapia e 87mL (±33) para a terapia dupla. O ganho foi de 67mL (p=0,042) da associação dos medicamentos em relação ao glicopirrônio isolado. A melhora na qualidade de vida, medida a partir das pontuações do questionário, foi de 4,7 (±8,9) pontos para a monoterapia e 5,2 (±11) pontos para a dupla terapia (p=0,08). Conclusão: Ambos os tratamentos demonstram melhora na função pulmonar dos pacientes.

Objective: To compare dual bronchodilator therapy (Glycopyrronium with Indacaterol) versus Glycopyrronium monotherapy in patients with chronic obstructive pulmonary disease. Methods: This was a prospective, unicentric, controlled, crossover, randomized, and double-blind clinical trial with 14 patients diagnosed with grade II chronic obstructive pulmonary disease. The participants received each treatment during the period of 30 days. Before each therapy, a 7-day wash-out period with a short-acting bronchodilator was instituted. Before and after each intervention, the patients underwent spirometry and answered the COPD Assessment Test questionnaire. Results: An improvement in pulmonary function measured by forced expiratory volume during the first second of 19mL (±36) for monotherapy, and 87mL (±33) for dual therapy was observed. The gain was of 67mL (p=0.042) in the association of the drugs in relation to Glycopyrronium alone. The mean improvement in quality of life measured from the questionnaire scores was 4.7 (±8.9) points for monotherapy and 5.2 (± 11) points for dual therapy (p=0.08). Conclusion: Both treatments show improvement in the patients' pulmonary function.

[Erratum to "Does Aortic Valve Replacement Surgery Improve Patients' Quality of Life?", by Pedro COELHO, Lara N. FERREIRA, Clara VITAL, José FRAGATA. Acta Med Port 2018 Jul-Aug;31(7-8):399-408]. / Errata a "A Cirurgia de Substituição Valvular Aórtica Melhora a Qualidade de Vida dos Doentes?" Pedro COELHO, Lara N. FERREIRA, Clara VITAL, José FRAGATA. Acta Med Port 2018 Jul-Aug;31(7-8):399-408.

Article published with errors: https://actamedicaportuguesa.com/revista/index.php/amp/article/view/10241 On page 405, Table 3, where it reads: On bold, the value 34.4 in "limitação do Desempenho" concerning the column "Seguimento a 6 meses" It should read: On bold, the value of 37,9. On page 406, Figure 4, where it reads: The y-axis of index SF-6D values are designed between 0 and 50. It should read: The y-axis of index SF-6D values between are designed between 0 and 1.

Artigo publicado com erros: https://actamedicaportuguesa.com/revista/index.php/amp/article/view/10241 Na página 405, Tabela 3 onde se lê: A negrito o valor 34,4 na dimensão "limitação do Desempenho" referente à coluna "Seguimento a 6 meses"Deverá ler-se: A negrito o valor 37,9. Na página 406, Figura 4, onde se lê:O eixo do índice SF-6D com valores entre 0 a 50.Deverá ler-se:O eixo do índice SF-6D com valores entre 0 a 1.

A hybrid modelling approach for eliciting health state preferences: the Portuguese EQ-5D-5L value set.

BACKGROUND: The EQ-5D is a generic preference-based quality of life measure considered useful for supporting clinical and policy decisions by providing utility values that can easily be converted into quality-adjusted life years to be integrated in cost-utility economic evaluations. Although the three-level classification system of the EuroQol questionnaire (EQ-5D-3L) is still the most popular preference-based instrument used worldwide, several studies reported a ceiling effect on this version, especially in healthy and/or young individuals. In 2009, the EuroQol Group introduced a five-level EQ-5D, which expands the descriptive system from three to five levels within the same five dimensions. For this version to be used in health economic evaluation, societal values need to be assigned to the 3125 health states generated by this instrument. OBJECTIVES: The aims of this study were to elicit the EQ-5D-5L health state preferences from the general Portuguese population and to derive the Portuguese value set for the EQ-5D-5L. METHODS: A representative sample of the Portuguese general population aged above 18 years was stratified by age and gender (n = 1451). Between October 2015 and July 2016, 28 interviewers carried out a series of 1-h-long computer-assisted personal interviews following the EuroQol Valuation Technology protocol. Each interview included the valuation of ten health states using the composite time trade-off (cTTO) and seven pairs of discrete choice experiments (DCEs). A standardized tool for quality control was used to assess the quality of the data as well as direct supervision and cross-examination of 10% of the global sample size. Data from both cTTO and DCE valuation tasks were modelled using a censored heteroskedastic hybrid model. RESULTS: Interviewers complied with the quality control protocol in providing high-quality valuation data. The hybrid econometric model had consistent and significant parameters. The derived societal values for the Portuguese population ranged from - 0.603 to 1. CONCLUSION: This study provided the Portuguese value set for the EQ-5D-5L on the basis of a hybrid econometric model using cTTO and DCE data. These results represent the preferences of the Portuguese population and are recommended to inform health decision-making in Portugal.