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Objectives: Depression is a major contributor of young people's burden of disease. In this study we aim to estimate the effect of elevated depressive symptoms on physical health-related quality of life. Design: We used self-reported information from the prospective BELLA cohort study, which included adolescents selected from the general population in Germany. The baseline assessment (2003-2006) and the 1-, 2-, and 6-year follow-up waves provide the data basis. Participants: The baseline study population consisted of 1,460 adolescents between the ages of 12 and 17 who, according to their caregivers, did not suffer from depression. Variables: The primary outcome, as measured by the physical component score (PCS) of the SF-36 at a 6-year follow-up (range: 0-100), is physical health-related quality of life. The exposure of interest is depressive symptoms, as measured by the Center for Epidemiological Studies Depression Scale for Children (CES-DC) at baseline, 1-year follow-up and 2-year follow-ups (range: 0-60). We dichotomized the exposure into subthreshold (≤15) and elevated depressive symptoms (>15). For the main analyses we considered a cumulative index for elevated depressive symptoms across the three time points (range: 0-3). Considered confounders are sex, age, socioeconomic status, migrant background, social support, anxiety symptoms, physical activity, chronic diseases, and sleeping problems. Statistical methods: We used multiple imputation to account for missing values. Within each imputed dataset, we applied inverse probability weighting (IPW) to estimate the effect of the cumulative index for elevated depressive symptoms at baseline, 1- and 2-year follow-up on physical health-related quality of life at 6-year follow-up. We derived 95% confidence intervals by bootstrapping. Results: After adjusting with IPW, the effect of the cumulative index per one unit increase of elevated depressive symptoms on the physical component score was -1.71 (95% CI: -3.51 to -0.04). The adjusted effect estimates of single exposure of elevated depressive symptoms on physical health-related quality of life were -0.83 (95% CI: -3.69 to 1.87) at baseline, -2.96 (95% CI: -4.94 to -0.52) at 1-year follow-up and -1.32 (95% CI: -3.85 to 1.15) at 2-year follow-up. Conclusion: Findings suggest that elevated depressive symptoms during adolescence decrease physical health-related quality of life in young adulthood.
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BACKGROUND: Cognitive screening tools are widely used in clinical practice to screen for age-related cognitive impairment and dementia. These tools' test scores are known to be influenced by age and education, leading to routine correction of raw scores for these factors. Despite these corrections being common practice, there is evidence suggesting that corrected scores may perform worse in terms of discrimination than raw scores. OBJECTIVE: To address the ongoing debate in the field of dementia research, we assessed the impact of the corrections on discrimination, specificity, and sensitivity of the Montreal Cognitive Assessment test in Italy, both for the overall population and across age and education strata. METHODOLOGY: We created a realistic model of the resident population in Italy in terms of age, education, cognitive impairment and test scores, and performed a simulation study. RESULTS: We confirmed that the discrimination performance was higher for raw scores than for corrected scores in discriminating patients with cognitive impairment from individuals without (areas under the curve of 0.947 and 0.923 respectively). With thresholds determined on the overall population, raw scores showed higher sensitivities for higher-risk age-education groups and higher specificities for lower-risk groups. Conversely, corrected scores showed uniform sensitivity and specificity across demographic strata, and thus better performance for certain age-education groups. CONCLUSION: Raw and corrected scores show different performances due to the underlying causal relationships between the variables. Each approach has advantages and disadvantages, the optimal choice between raw and corrected scores depends on the aims and preferences of practitioners and policymakers.
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OBJECTIVE: Class imbalance is sometimes considered a problem when developing clinical prediction models and assessing their performance. To address it, correction strategies involving manipulations of the training dataset, such as random undersampling or oversampling, are frequently used. The aim of this article is to illustrate the consequences of these class imbalance correction strategies on clinical prediction models' internal validity in terms of calibration and discrimination performances. METHODS: We used both heuristic intuition and formal mathematical reasoning to characterize the relations between conditional probabilities of interest and probabilities targeted when using random undersampling or oversampling. We propose a plug-in estimator that represents a natural correction for predictions obtained from models that have been trained on artificially balanced datasets ("naïve" models). We conducted a Monte Carlo simulation with two different data generation processes and present a real-world example using data from the International Stroke Trial database to empirically demonstrate the consequences of applying random resampling techniques for class imbalance correction on calibration and discrimination (in terms of Area Under the ROC, AUC) for logistic regression and tree-based prediction models. RESULTS: Across our simulations and in the real-world example, calibration of the naïve models was very poor. The models using the plug-in estimator generally outperformed the models relying on class imbalance correction in terms of calibration while achieving the same discrimination performance. CONCLUSION: Random resampling techniques for class imbalance correction do not generally improve discrimination performance (i.e., AUC), and their use is hard to justify when aiming at providing calibrated predictions. Improper use of such class imbalance correction techniques can lead to suboptimal data usage and less valid risk prediction models.
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Método de Monte Carlo , Humanos , Calibragem , Curva ROC , Modelos Estatísticos , Área Sob a Curva , Simulação por Computador , Modelos Logísticos , Algoritmos , Medição de Risco/métodosRESUMO
Sometimes treatment effects are absent in a subgroup of the population. For example, penicillin has no effect on severe symptoms in individuals infected by resistant Staphylococcus aureus , and codeine has no effect on pain in individuals with certain polymorphisms in the CYP2D6 enzyme. Subgroups where a treatment is ineffective are often called negative control populations or placebo groups. They are leveraged to detect bias in different disciplines. Here we present formal criteria that justify the use of negative control populations to rule out unmeasured confounding and mechanistic (direct) causal effects. We further argue that negative control populations, satisfying our formal conditions, are available in many settings, spanning from clinical studies of infectious diseases to epidemiologic studies of public health interventions. Negative control populations can also be used to rule out placebo effects in unblinded randomized experiments. As a case study, we evaluate the effect of mobile stroke unit dispatches on functional outcomes at discharge in individuals with suspected stroke, using data from a large trial. Our analysis supports the hypothesis that mobile stroke units improve functional outcomes in these individuals.
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Staphylococcus aureus Resistente à Meticilina , Acidente Vascular Cerebral , Humanos , Viés , Estudos Epidemiológicos , CausalidadeRESUMO
INTRODUCTION: In patients with acute intracerebral haemorrhage (ICH) and elevated systolic blood pressure (BP), guidelines suggest that systolic BP reduction to <140 mmHg should be rapidly initiated. Compared with conventional care, Mobile Stroke Units (MSUs) allow for earlier ICH diagnosis through prehospital imaging and earlier BP lowering. PATIENTS AND METHODS: ICH patients were prospectively evaluated as a cohort of the controlled B_PROUD-study in which MSU availability alone determined MSU dispatch in addition to conventional ambulance. We used inverse probability of treatment weighting to adjust for confounding to estimate the effect of additional MSU dispatch in ICH patients. Outcomes of interest were 7-day mortality (primary), systolic BP (sBP) at hospital arrival, dispatch-to-imaging time, largest haematoma volume, anticoagulation reversal, length of in-hospital stay, 3-month functional outcome. RESULTS: Between February 2017 and May 2019, MSUs were dispatched to 95 (mean age: 72 ± 13 years, 45% female) and only conventional ambulances to 78 ICH patients (mean age: 71 ± 12 years, 44% female). After adjusting for confounding, we found shorter dispatch-to-imaging time (mean difference: -17.75 min, 95% CI: -27.16 to -8.21 min) and lower sBP at hospital arrival (mean difference = -16.31 mmHg, 95% CI: -30.64 to -6.19 mmHg) in the MSU group. We found no statistically significant difference for the other outcomes, including 7-day mortality (adjusted odds ratio: 1.43, 95% CI: 0.68 to 3.31) or favourable outcome (adjusted odds ratio = 0.67, 95% CI: 0.27 to 1.67). CONCLUSIONS: Although MSU dispatch led to sBP reduction and lower dispatch-to-imaging time compared to conventional ambulance care, we found no evidence of better outcomes in the MSU dispatch group.
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BACKGROUND: Machine learning models promise to support diagnostic predictions, but may not perform well in new settings. Selecting the best model for a new setting without available data is challenging. We aimed to investigate the transportability by calibration and discrimination of prediction models for cognitive impairment in simulated external settings with different distributions of demographic and clinical characteristics. METHODS: We mapped and quantified relationships between variables associated with cognitive impairment using causal graphs, structural equation models, and data from the ADNI study. These estimates were then used to generate datasets and evaluate prediction models with different sets of predictors. We measured transportability to external settings under guided interventions on age, APOE ε4, and tau-protein, using performance differences between internal and external settings measured by calibration metrics and area under the receiver operating curve (AUC). RESULTS: Calibration differences indicated that models predicting with causes of the outcome were more transportable than those predicting with consequences. AUC differences indicated inconsistent trends of transportability between the different external settings. Models predicting with consequences tended to show higher AUC in the external settings compared to internal settings, while models predicting with parents or all variables showed similar AUC. CONCLUSIONS: We demonstrated with a practical prediction task example that predicting with causes of the outcome results in better transportability compared to anti-causal predictions when considering calibration differences. We conclude that calibration performance is crucial when assessing model transportability to external settings.
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Disfunção Cognitiva , Modelos Estatísticos , Humanos , Prognóstico , Disfunção Cognitiva/diagnóstico , Benchmarking , CalibragemRESUMO
BACKGROUND: Migraine is a very common headache disorder on the population level, characterized by symptomatic attacks (activity). For many people with migraine, the migraine symptoms intermittently or permanently cease during their lifetime (inactive migraine). The current diagnostic classification of migraine considers two states: active migraine (having migraine symptoms within the last year) and not having active migraine (including both individuals with inactive migraine and those who never had migraine). Defining a state of inactive migraine that has gone into remission may better capture the trajectories of migraine across the lifespan and contribute to a better understanding of its biological processes. We aimed to quantify the prevalence of never, active, and inactive migraine separately, using modern prevalence and incidence estimation methodology to better describe the complexity of migraine trajectories at the population level. METHODS: Using a multistate modeling approach, data from the Global Burden of Disease (GBD) study, and results from a population-based study, we estimated the transition rates by which individuals moved between migraine disease states and estimated prevalences of never, active and inactive migraine. We used data from the GBD project and a hypothetical cohort of 100,000 people with a starting age of 30 and 30 years of follow-up, both in Germany and globally, stratified by sex. RESULTS: In Germany, the estimated rate of transition from active to inactive migraine (remission rate) increased after the age of 22.5 in women and 27.5 in men. The pattern for men in Germany was similar to the one observed on the global level. The prevalence of inactive migraine among women reaches 25.7% in Germany and 16.5% globally at age 60. For men, the inactive migraine prevalence estimates at the same age were 10.4% in Germany and 7.1% globally. CONCLUSIONS: Considering an inactive migraine state explicitly reflects a different epidemiological picture of migraine across the lifecourse. We have demonstrated that many women of older ages may be in an inactive migraine state. Many pressing research questions can only be answered if population-based cohort studies collect information not only on active migraine but also on inactive migraine states.
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Transtornos da Cefaleia , Transtornos de Enxaqueca , Masculino , Humanos , Feminino , Pessoa de Meia-Idade , Adulto , Prevalência , Alemanha/epidemiologia , Carga Global da Doença , Transtornos de Enxaqueca/epidemiologiaRESUMO
BACKGROUND: The Berlin-based B_PROUD study was designed to assess the effect of mobile stroke unit (MSU) dispatch among ischemic stroke and transient ischemic attack (TIA) patients without contraindications to reperfusion treatments. However, a large proportion of patients for whom the MSU was dispatched did not ultimately receive MSU care. We estimated the causal effect of additional MSU care on 3-month functional outcomes among B_PROUD patients for whom an MSU was dispatched. METHODS: We used data from the B_PROUD study (1 February 2017-8 May 2019). Given the presence of exposure-outcome unmeasured confounding, we used the front-door formula to identify the distribution of modified Rankin scale (mRS) outcomes under two hypothetical interventions: (1) receiving additional MSU care and (2) only receiving conventional care. We considered the time from dispatch to thrombolysis as the full mediator and adjusted for exposure-mediator and mediator-outcome confounding. We used a parametric estimator to estimate the common odds ratio (cOR) and 95% bootstrapped confidence intervals (CI). RESULTS: We included in total 768 ischemic stroke/TIA patients with MSU dispatch. The MSU was canceled for 180 (23%) patients, whereas 588 (77%) received MSU care. The unadjusted association between the care group and mRS favored conventional care (cOR = 1.7; 95% CI = 1.2, 2.3); however, after applying the front-door formula, the mRS distribution favored MSU care (cOR = 0.88; 95% CI = 0.81, 0.96). CONCLUSIONS: Receiving MSU care was associated with better functional outcomes than conventional care only, compatible with the hypothesized beneficial effect of MSU care on poststroke outcomes, among stroke and TIA patients without contraindications to reperfusion treatments.
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Ataque Isquêmico Transitório , AVC Isquêmico , Acidente Vascular Cerebral , Humanos , Ataque Isquêmico Transitório/epidemiologia , Ataque Isquêmico Transitório/terapia , Ataque Isquêmico Transitório/complicações , Terapia Trombolítica/efeitos adversos , Acidente Vascular Cerebral/epidemiologia , Acidente Vascular Cerebral/terapia , Unidades Móveis de Saúde , AVC Isquêmico/complicações , Resultado do TratamentoRESUMO
Background: Modified Rankin Scale (mRS) scores are used to measure functional outcomes after stroke. Researchers create horizontal stacked bar graphs (nicknamed "Grotta bars") to illustrate distributional differences in scores between groups. In well-conducted randomized controlled trials, Grotta bars have a causal interpretation. However, the common practice of exclusively presenting unadjusted Grotta bars in observational studies can be misleading in the presence of confounding. We demonstrated this problem and a possible solution using an empirical comparison of 3-month mRS scores among stroke/TIA patients discharged home versus elsewhere after hospitalization. Patients and methods: Using data from the Berlin-based B-SPATIAL registry, we estimated the probability of being discharged home conditional on prespecified measured confounding factors and generated stabilized inverse probability of treatment (IPT) weights for each patient. We visualized mRS distributions by group with Grotta bars for the IPT-weighted population in which measured confounding was removed. We then used ordinal logistic regression to quantify unadjusted and adjusted associations between being discharged home and the 3-month mRS score. Results: Of 3184 eligible patients, 2537 (79.7%) were discharged home. In the unadjusted analyses, those discharged home had considerably lower mRS compared with patients discharged elsewhere (common odds ratio, cOR = 0.13, 95% CI: 0.11-0.15). After removing measured confounding, we obtained substantially different mRS distributions, visually apparent in the adjusted Grotta bars. No statistically significant association was found after confounding adjustment (cOR = 0.82, 95% CI: 0.60-1.12). Discussion and conclusion: The practice of presenting only unadjusted stacked bar graphs for mRS scores together with adjusted effect estimates in observational studies can be misleading. IPT weighting can be implemented to create Grotta bars that account for measured confounding, which are more consistent with the presentation of adjusted results in observational studies.
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Acidente Vascular Cerebral , Humanos , Acidente Vascular Cerebral/diagnóstico , Modelos Logísticos , Hospitalização , Alta do Paciente , ProbabilidadeRESUMO
Pneumococcal conjugate vaccines (PCVs) protect against diseases caused by Streptococcus pneumoniae, such as meningitis, bacteremia, and pneumonia. It is challenging to estimate their population-level impact due to the lack of a perfect control population and the subtleness of signals when the endpoint-such as all-cause pneumonia-is nonspecific. Here we present a new approach for estimating the impact of PCVs: using least absolute shrinkage and selection operator (LASSO) regression to select variables in a synthetic control model to predict the counterfactual outcome for vaccine impact inference. We first used a simulation study based on hospitalization data from Mexico (2000-2013) to test the performance of LASSO and established methods, including the synthetic control model with Bayesian variable selection (SC). We found that LASSO achieved accurate and precise estimation, even in complex simulation scenarios where the association between the outcome and all control variables was noncausal. We then applied LASSO to real-world hospitalization data from Chile (2001-2012), Ecuador (2001-2012), Mexico (2000-2013), and the United States (1996-2005), and found that it yielded estimates of vaccine impact similar to SC. The LASSO method is accurate and easily implementable and can be applied to study the impact of PCVs and other vaccines.
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Infecções Pneumocócicas , Pneumonia , Humanos , Lactente , Teorema de Bayes , Infecções Pneumocócicas/epidemiologia , Infecções Pneumocócicas/prevenção & controle , Vacinas Pneumocócicas/administração & dosagem , Pneumonia/epidemiologia , Pneumonia/prevenção & controle , Streptococcus pneumoniae , Estados Unidos , Vacinas ConjugadasRESUMO
BACKGROUND: Lower global disability and higher quality of life among ischemic stroke patients was found to be associated with the dispatch of mobile stroke units (MSUs) among patients eligible for recanalizing treatments in the Berlin_Prehospital Or Usual Delivery of stroke care (B_PROUD) study. The current study assessed the cost-utility and cost-effectiveness of additional MSU dispatch using data from this prospective, controlled, intervention study. METHODS: Outcomes considered in the economic evaluation included quality-adjusted life years (QALYs) derived from the 3-level version of EQ-5D (EQ-5D-3L) and modified Rankin Scale (mRS) scores for functional outcomes 3-months after stroke. Costs were prospectively collected during the study by the MSU provider (Berlin Fire Brigade) and the B_PROUD research team. We focus our results on the societal perspective. As we aimed to determine the economic consequences of the intervention beyond the study's follow-up period, both care costs and QALYs were extrapolated over 5 years. RESULTS: The additional MSU dispatch resulted in an incremental 40,984 per QALY. The best-case scenario and the worst-case scenario yielded additional costs of, respectively, 24,470.76 and 61,690.88 per QALY. In the cost-effectiveness analysis, MSU dispatch resulted in incremental costs of 81,491 per survival without disability. The best-case scenario and the worst-case scenario yielded additional costs of, respectively, 44,455.30 and 116,491.15 per survival without disability. INTERPRETATION: Among patients eligible for recanalizing treatments in ischemic stroke, MSU dispatch was associated with both higher QALYs and higher costs and is cost-effective when considering internationally accepted thresholds ranging from an additional 40,000 to 80,000 per QALY. ANN NEUROL 2023;93:942-951.
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AVC Isquêmico , Acidente Vascular Cerebral , Humanos , Análise Custo-Benefício , Qualidade de Vida , Estudos Prospectivos , Acidente Vascular Cerebral/terapiaRESUMO
Importance: Diagnostic incidence data for syndromes associated with frontotemporal lobar degeneration (FTLD) in multinational studies are urgent in light of upcoming therapeutic approaches. Objective: To assess the incidence of FTLD across Europe. Design, Setting, and Participants: The Frontotemporal Dementia Incidence European Research Study (FRONTIERS) was a retrospective cohort study conducted from June 1, 2018, to May 31, 2019, using a population-based registry from 13 tertiary FTLD research clinics from the UK, the Netherlands, Finland, Sweden, Spain, Bulgaria, Serbia, Germany, and Italy and including all new FTLD-associated cases during the study period, with a combined catchment population of 11â¯023â¯643 person-years. Included patients fulfilled criteria for the behavioral variant of frontotemporal dementia (BVFTD), the nonfluent variant or semantic variant of primary progressive aphasia (PPA), unspecified PPA, progressive supranuclear palsy, corticobasal syndrome, or frontotemporal dementia with amyotrophic lateral sclerosis (FTD-ALS). Data were analyzed from July 19 to December 7, 2021. Main Outcomes and Measures: Random-intercept Poisson models were used to obtain estimates of the European FTLD incidence rate accounting for geographic heterogeneity. Results: Based on 267 identified cases (mean [SD] patient age, 66.70 [9.02] years; 156 males [58.43%]), the estimated annual incidence rate for FTLD in Europe was 2.36 cases per 100â¯000 person-years (95% CI, 1.59-3.51 cases per 100â¯000 person-years). There was a progressive increase in FTLD incidence across age, reaching its peak at the age of 71 years, with 13.09 cases per 100 000 person-years (95% CI, 8.46-18.93 cases per 100 000 person-years) among men and 7.88 cases per 100 000 person-years (95% CI, 5.39-11.60 cases per 100 000 person-years) among women. Overall, the incidence was higher among men (2.84 cases per 100â¯000 person-years; 95% CI, 1.88-4.27 cases per 100â¯000 person-years) than among women (1.91 cases per 100â¯000 person-years; 95% CI, 1.26-2.91 cases per 100â¯000 person-years). BVFTD was the most common phenotype (107 cases [40.07%]), followed by PPA (76 [28.46%]) and extrapyramidal phenotypes (69 [25.84%]). FTD-ALS was the rarest phenotype (15 cases [5.62%]). A total of 95 patients with FTLD (35.58%) had a family history of dementia. The estimated number of new FTLD cases per year in Europe was 12â¯057. Conclusions and Relevance: The findings suggest that FTLD-associated syndromes are more common than previously recognized, and diagnosis should be considered at any age. Improved knowledge of FTLD incidence may contribute to appropriate health and social care planning and in the design of future clinical trials.
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Esclerose Lateral Amiotrófica , Demência Frontotemporal , Degeneração Lobar Frontotemporal , Masculino , Humanos , Feminino , Idoso , Demência Frontotemporal/epidemiologia , Incidência , Estudos Retrospectivos , Degeneração Lobar Frontotemporal/epidemiologia , Síndrome , Europa (Continente)/epidemiologiaRESUMO
BACKGROUND AND OBJECTIVES: In Italy, approximately 650 individuals receive a diagnosis of primary progressive aphasia (PPA) every year. Unfortunately, the frequency with which patients are referred to speech-language services is suboptimal, likely due to skepticism regarding the value of speech-language therapy in the context of neurodegeneration. MATERIALS AND METHODS: We conducted a virtual survey of speech and language therapists (SLTs) across Italy, to collect information about the assessment, intervention and management of patients with PPA. To ensure that as many SLTs as possible received the survey, the Italian Federation of SLTs (Federazione Logopedisti Italiani, FLI) aided in disseminating the survey. RESULTS: In total, 336 respondents participated in the online survey, 140 of whom had previous experience with PPA patients. Respondents indicated having seen a total of 428 PPA patients in the previous 24 months (three patients on average, range: 0-40). SLTs who reported never working with PPA identified underdiagnoses, low referral rates and the rarity of the clinical syndrome as major reasons for their lack of experience with PPA. SLTs with experience working with PPA indicated that patients may not have accessed services because of service dysfunction and geographical barriers. Respondents reported using informal interviews during assessments and tests developed for post-stroke aphasia, while impairment-based/restitutive interventions were utilised most often. CONCLUSION: Findings may serve to inform health policy organisations regarding the current shortcomings and needed recommendations for improving the care of individuals with PPA in Italy. Improving awareness of the utility of rehabilitation among SLTs and other clinical service providers may serve to facilitate access to intervention, which in turn will serve to better support individuals living with PPA. WHAT THIS PAPER ADDS: What is already known on the subject Speech and language therapists (SLTs) play a crucial role in the assessment, diagnosis and treatment of people with primary progressive aphasia (PPA). However, the frequency with which individuals with PPA are referred for speech and language services is suboptimal due to skepticism regarding the value of speech and language therapy in the context of neurodegeneration, the scarcity of SLTs with expertise in the treatment of PPA and the lack of awareness of the SLT role amongst referrers. What this paper adds to existing knowledge In recognition of the lack of published information on the provision of speech and language therapy services and clinicians' approaches to the assessment and treatment of individuals with PPA in Italy, we conducted an online survey to evaluate the current referral patterns for speech and language therapy services and to examine the current barriers to access these services for individuals with PPA in Italy. What are the potential or actual clinical implications of this work? The data presented here support that SLTs view treatment as useful for individuals with PPA and other professional figures and may serve to improve access to intervention, which in turn will serve to better support individuals living with PPA. The results highlight the need to inform health policy organisations about current gaps and aid in developing recommendations for improving the care of individuals with PPA, in order to understand how SLTs can best support individuals with PPA and their families.
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Afasia Primária Progressiva , Terapia da Linguagem , Fonoterapia , Humanos , Afasia Primária Progressiva/diagnóstico , Afasia Primária Progressiva/terapia , Terapia da Linguagem/métodos , Encaminhamento e Consulta , Fala , Fonoterapia/métodos , Inquéritos e Questionários , Acessibilidade aos Serviços de Saúde , ItáliaRESUMO
OBJECTIVE: To determine the effect of additional mobile stroke unit (MSU) dispatch on functional outcomes among the full spectrum of stroke patients, regardless of subtype or potential contraindications to reperfusion therapies. METHODS: We used data from the nonrandomized Berlin-based B_PROUD study (02/2017 to 05/2019), in which MSUs were dispatched based solely on availability, and the linked B-SPATIAL stroke registry. All patients with final stroke or transient ischemic attack (TIA) diagnoses were eligible. The intervention under study was the additional dispatch of an MSU, an emergency physician-staffed ambulance equipped to provide prehospital imaging and thrombolytic treatment, compared to conventional ambulance alone. The primary outcome was the 3-month modified Rankin Scale (mRS) score, and the co-primary outcome was a 3-tiered disability scale. We identified confounders using directed acyclic graphs and obtained adjusted effect estimates using inverse probability of treatment weighting. RESULTS: MSUs were dispatched to 1,125 patients (mean age: 74 years, 46.5% female), while for 1,141 patients only conventional ambulances were dispatched (75 years, 49.9% female). After confounding adjustment, MSU dispatch was associated with more favorable 3-month mRS scores (common odds ratio [cOR] = 0.82; 95% confidence interval [CI]: 0.71-0.94). No statistically significant association was found with the co-primary outcome (cOR = 0.86; 9% CI: 0.72-1.01) or 7-day mortality (OR = 0.94; 95% CI: 0.59-1.48). INTERPRETATION: When considering the entire population of stroke/TIA patients, MSU dispatch improved 3-month functional outcomes without evidence of compromised safety. Our results are relevant for decision-makers since stroke subtype and treatment eligibility are unknown at time of dispatch. ANN NEUROL 2023;93:50-63.
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Ataque Isquêmico Transitório , Acidente Vascular Cerebral , Humanos , Feminino , Idoso , Masculino , Ataque Isquêmico Transitório/diagnóstico , Ataque Isquêmico Transitório/terapia , Terapia Trombolítica/métodos , Acidente Vascular Cerebral/terapia , Acidente Vascular Cerebral/tratamento farmacológico , Unidades Móveis de Saúde , AmbulânciasRESUMO
Cognitive screening tests such as the Mini-Mental State Examination are widely used in clinical routine to predict cognitive impairment. The raw test scores are often corrected for age and education, although documented poorer discrimination performance of corrected scores has challenged this practice. Nonetheless, test correction persists, perhaps due to the seemingly counterintuitive nature of the underlying problem. We used a causal framework to inform the long-standing debate from a more intuitive angle. We illustrate and quantify the consequences of applying the age-education correction of cognitive tests on discrimination performance. In an effort to bridge theory and practical implementation, we computed differences in discrimination performance under plausible causal scenarios using Open Access Series of Imaging Studies (OASIS)-1 data. We show that when age and education are causal risk factors for cognitive impairment and independently also affect the test score, correcting test scores for age and education removes meaningful information, thereby diminishing discrimination performance.
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Disfunção Cognitiva , Humanos , Disfunção Cognitiva/diagnóstico , Testes Neuropsicológicos , Escolaridade , Testes de Estado Mental e Demência , CogniçãoRESUMO
Data-driven algorithms are currently deployed in several fields, leading to a rapid increase in the importance algorithms have in decision-making processes. Over the last years, several instances of discrimination by algorithms were observed. A new branch of research emerged to examine the concept of "algorithmic fairness." No consensus currently exists on a single operationalization of fairness, although causal-based definitions are arguably more aligned with the human conception of fairness. The aim of this article is to investigate the degree of this alignment in a case study inspired by a recent ruling of an Italian court on the reputational-ranking algorithm used by a food delivery platform. I relied on the documentation of the legal dispute to discuss the applicability, intuitiveness and appropriateness of causal models in evaluating fairness, with a specific focus on a causal-based fairness definition called "counterfactual fairness." I first describe the details of the dispute and the arguments presented to the court, as well as the court's final decision, to establish the context of the case study. Then, I translate the dispute into a formal simplified problem using a causal diagram, which represents the main aspects of the data generation process in the case study. I identify the criteria used by the court in ruling that the algorithm was unfair and compare them with the counterfactual fairness definition. The definition of counterfactual fairness was found to be well aligned with the human conception of fairness in this case study, using the court order rationale as a gold standard.
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INTRODUCTION: The incidence of Frontotemporal Lobar Degeneration (FTLD)-related disorders and their characteristics are not well known. The "FRONTotemporal dementia Incidence European Research Study" (FRONTIERS) is designed to fill this gap. METHODS: FRONTIERS is a European prospective, observational population study based on multinational registries. FRONTIERS comprises 11 tertiary referral centers across Europe with long-lasting experience in FTLD-related disorders and comprehensive regional referral networks, enabling incidence estimation over well-defined geographical areas. ENDPOINTS: The primary endpoints are (1) the incidence of FTLD-related disorders across Europe; (2) geographic trends of FTLD-related disorders; (3) the distribution of FTLD phenotypes in different populations and ethnicities in Europe; (4) inheritance of FTLD-related disorders, including the frequencies of monogenic FTLD as compared to overall disease burden; and (5) implementation of data banking for clinical and biological material. EXPECTED IMPACTS: FRONTIERS will improve the understanding of FTLD-related disorders and their epidemiology, promoting appropriate public health service policies and treatment strategies.
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Demência Frontotemporal , Degeneração Lobar Frontotemporal , Estudos de Coortes , Demência Frontotemporal/epidemiologia , Demência Frontotemporal/genética , Degeneração Lobar Frontotemporal/epidemiologia , Degeneração Lobar Frontotemporal/genética , Humanos , Incidência , Estudos ProspectivosRESUMO
BACKGROUND: Neurological conditions are highly prevalent and disabling, in particular in the elderly. The Italian population has witnessed sharp ageing and we can thus expect a rising trend in the incidence, prevalence and disability of these conditions. METHODS: We relied on the Global Burden of Disease 2019 study to extract Italian data on incidence, prevalence and years lived with a disability (YLDs) referred to a broad set of neurological disorders including, brain and nervous system cancers, stroke, encephalitis, meningitis, tetanus, traumatic brain injury, and spinal cord injury. We assessed changes between 1990 and 2019 in counts and age-standardized rates. RESULTS: The most prevalent conditions were tension-type headache, migraine, and dementias, whereas the most disabling were migraine, dementias and traumatic brain injury. YLDs associated with neurological conditions increased by 22.5%, but decreased by 2.3% in age-standardized rates. The overall increase in prevalence and YLDs counts was stronger for non-communicable diseases with onset in old age compared to young to adult-age onset ones. The same trends were in the opposite direction when age-standardized rates were taken into account. CONCLUSIONS: The increase in YLDs associated with neurological conditions is mostly due to population ageing and growth: nevertheless, lived disability and, as a consequence, impact on health systems has increased. Actions are needed to improve outcome and mitigate disability associated with neurological conditions, spanning among diagnosis, treatment, care pathways and workplace interventions.
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Carga Global da Doença , Doenças do Sistema Nervoso , Adulto , Idoso , Saúde Global , Humanos , Incidência , Itália/epidemiologia , Doenças do Sistema Nervoso/epidemiologia , PrevalênciaRESUMO
India faces 0.5 million malaria cases annually, including half of all Plasmodium vivax malaria cases worldwide. This case-control study assessed socioeconomic determinants of urban malaria in coastal Mangaluru, Karnataka, southwestern India. Between June and December 2015, we recruited 859 malaria patients presenting at the governmental Wenlock Hospital and 2190 asymptomatic community controls. We assessed clinical, parasitological, and socioeconomic data. Among patients, p. vivax mono-infection (70.1%) predominated. Most patients were male (93%), adult (median, 27 years), had no or low-level education (70.3%), and 57.1% were daily labourers or construction workers. In controls (59.3% male; median age, 32 years; no/low-level education, 54.5%; daily labourers/construction workers, 41.3%), 4.1% showed asymptomatic Plasmodium infection. The odds of malaria was reduced among those who had completed 10th school grade (aOR, 0.3; 95% CI, 0.26-0.42), lived in a building with a tiled roof (aOR, 0.71; 95% CI, 0.53-0.95), and reported recent indoor residual spraying (aOR, 0.02; 95% CI, 0.01-0.04). In contrast, migrant status was a risk factor for malaria (aOR, 2.43; 95% CI, 1.60-3.67). Malaria in Mangaluru is influenced by education, housing condition, and migration. Indoor residual spraying greatly contributes to reducing malaria in this community and should be promoted, especially among its marginalised members.
Assuntos
Inseticidas , Malária , Adulto , Estudos de Casos e Controles , Escolaridade , Feminino , Qualidade Habitacional , Humanos , Índia/epidemiologia , Malária/epidemiologia , MasculinoRESUMO
Percutaneous endoscopic gastrostomy (PEG) is the standard procedure for feeding severely dysphagic patients with amyotrophic lateral sclerosis (ALS). It is associated with prolonged survival and improvement in quality of life. Nasal inspiratory pressure during a sniff (SNIP) is a respiratory test used extensively in ALS for the assessment of inspiratory muscle strength. In this study, we aimed to investigate the role of SNIP at baseline to predict PEG placement in ALS. Data from a clinical incident cohort of 179 ALS cases attending the multidisciplinary ALS unit of the University of Bari between April 2006 and December 2012 were retrospectively analysed. At baseline, patients underwent detailed neurological, nutritional and respiratory assessments, including measurements of SNIP and forced vital capacity (FVC). Patients were therefore followed up approximately every three to six months until they were able to attend the centre. The censoring date for the survival analysis was 15 April 2014, with PEG placement as the main outcome. Cox proportional hazard regression models were used to examine the association between SNIP and PEG placement, adjusted for possible confounders. During the follow-up period, 75 participants (42%) received PEG implant. PEG placement was more frequent (57% vs. 31%; p = 0.001) and earlier (after 11.6 ± 14.0 months from the first visit, vs. 23.3 ± 15.5 months; p < 0.0001) in the group of patients with baseline SNIP ≤ 40 cm H2O. Baseline SNIP was a predictor of PEG placement even after correction for multiple potential confounders (HR 0.98; 95% CI: 0.96-0.99; p = 0.02). To conclude, the present study showed that SNIP at baseline is an early indicator of disease progression and therefore of the need for enteral nutrition in ALS.