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Objective: This study tested the hypothesis that a neuroprotective combined therapy based on epidermal growth factor (EGF) and growth hormone-releasing hexapeptide (GHRP6) could be safe for acute ischemic stroke patients, admitting up to 30% of serious adverse events (SAE) with proven causality. Methods: A multi-centric, randomized, open-label, controlled, phase I-II clinical trial with parallel groups was conducted (July 2017 to January 2018). Patients aged 18-80 years with a computed tomography-confirmed ischemic stroke and less than 12 h from the onset of symptoms were randomly assigned to the study groups I (75 µg rEGF + 3.5 mg GHRP6 i.v., n=10), II (75 µg rEGF + 5 mg GHRP6 i.v., n=10), or III (standard care control, n=16). Combined therapy was given BID for 7 days. The primary endpoint was safety over 6 months. Secondary endpoints included neurological (NIHSS) and functional [Barthel index and modified Rankin scale (mRS)] outcomes. Results: The study population had a mean age of 66 ± 11 years, with 21 men (58.3%), a baseline median NIHSS score of 9 (95% CI: 8-11), and a mean time to treatment of 7.3 ± 2.8 h. Analyses were conducted on an intention-to-treat basis. SAEs were reported in 9 of 16 (56.2%) patients in the control group, 3 of 10 (30%) patients in Group I (odds ratio (OR): 0.33; 95% CI: 0.06-1.78), and 2 of 10 (20%) patients in Group II (OR: 0.19; 95% CI: 0.03-1.22); only two events in one patient in Group I were attributed to the intervention treatment. Compliance with the study hypothesis was greater than 0.90 in each group. Patients treated with EGF + GHRP6 had a favorable neurological and functional evolution at both 90 and 180 days, as evidenced by the inferential analysis of NIHSS, Barthel, and mRS and by their moderate to strong effect size. At 6 months, proportion analysis evidenced a higher survival rate for patients treated with the combined therapy. Ancillary analysis including merged treated groups and utility-weighted mRS also showed a benefit of this combined therapy. Conclusion: EGF + GHRP6 therapy was safe. The functional benefits of treatment in this study supported a Phase III study. Clinical Trial Registration: RPCEC00000214 of the Cuban Public Registry of Clinical Trials, Unique identifier: IG/CIGB-845I/IC/1601.
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Null hypothesis significant testing (NHST) is the dominant statistical approach in the geriatric and rehabilitation fields. However, NHST is routinely misunderstood or misused. In this case, the findings from clinical trials would be taken as evidence of no effect, when in fact, a clinically relevant question may have a "non-significant" p-value. Conversely, findings are considered clinically relevant when significant differences are observed between groups. To assume that p-value is not an exclusive indicator of an association or the existence of an effect, researchers should be encouraged to report other statistical analysis approaches as Bayesian analysis and complementary statistical tools alongside the p-value (eg, effect size, confidence intervals, minimal clinically important difference, and magnitude-based inference) to improve interpretation of the findings of clinical trials by presenting a more efficient and comprehensive analysis. However, the focus on Bayesian analysis and secondary statistical analyses does not mean that NHST is less important. Only that, to observe a real intervention effect, researchers should use a combination of secondary statistical analyses in conjunction with NHST or Bayesian statistical analysis to reveal what p-values cannot show in the geriatric and rehabilitation studies (eg, the clinical importance of 1kg increase in handgrip strength in the intervention group of long-lived older adults compared to a control group). This paper provides potential insights for improving the interpretation of scientific data in rehabilitation and geriatric fields by utilizing Bayesian and secondary statistical analyses to better scrutinize the results of clinical trials where a p-value alone may not be appropriate to determine the efficacy of an intervention.
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Força da Mão , Projetos de Pesquisa , Humanos , Idoso , Teorema de Bayes , Interpretação Estatística de DadosRESUMO
Among defenses against predation, chemical defenses are possibly the most studied. However, when addressing the effectiveness of those chemical defenses, previous studies did not include properties of the chemical substances themselves. Lipophilicity, for instance, may facilitate crossing membranes, and boiling point may define the duration of the substances in the air. Moreover, other variables may also be relevant: the predator taxon; the prey model chosen to conduct experiments; whether the prey is presented grouped or not in experiments; and whether the chemical defense is a mixture of many substances or only one. To understand how those factors influence chemical defenses' effectiveness, we conducted a multilevel meta-analysis with 43 studies (127 effect sizes), accounting for different types of dependence. We used Akaike Information Criterion (AICc) to select the best model. The model with the lowest AICc value included only the boiling point, which defines how quickly a chemical substance volatilizes. This model indicated that the most effective chemical defenses had lower boiling point values, i.e., higher volatility. Moreover, we did not find chemicals with very low boiling points, suggesting there might be an optimum range of volatility. Other models, including the intercept-only model, were also recovered among the best models, therefore further studies are needed to confirm the relationship between volatility and chemical defenses' effectiveness. Our results highlight the value of incorporating physicochemical properties in the ecological and evolutionary study of chemical defense.
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Artrópodes , Animais , Comportamento Predatório , Evolução Biológica , Modelos BiológicosRESUMO
Abstract Background To illustrate how (standardised) effect sizes (ES) vary based on calculation method and to provide considerations for improved reporting. Methods Data from three trials of tanezumab in subjects with osteoarthritis were analyzed. ES of tanezumab versus comparator for WOMAC Pain (outcome) was defined as least squares difference between means (mixed model for repeated measures analysis) divided by a pooled standard deviation (SD) of outcome scores. Three approaches to computing the SD were evaluated: Baseline (the pooled SD of WOMAC Pain values at baseline [pooled across treatments]); Endpoint (the pooled SD of these values at the time primary endpoints were assessed); and Median (the median pooled SD of these values based on the pooled SDs across available timepoints). Bootstrap analyses were used to compute 95% confidence intervals (CI). Results ES (95% CI) of tanezumab 2.5 mg based on Baseline, Endpoint, and Median SDs in one study were - 0.416 (- 0.796, - 0.060), - 0.195 (- 0.371, - 0.028), and - 0.196 (- 0.373, - 0.028), respectively; negative values indicate pain improvement. This pattern of ES differences (largest with Baseline SD, smallest with Endpoint SD, Median SD similar to Endpoint SD) was consistent across all studies and doses of tanezumab. Conclusion Differences in ES affect interpretation of treatment effect. Therefore, we advocate clearly reporting individual elements of ES in addition to its overall calculation. This is particularly important when ES estimates are used to determine sample sizes for clinical trials, as larger ES will lead to smaller sample sizes and potentially underpowered studies. Trial Registration Clinicaltrials.gov NCT02697773, NCT02709486, and NCT02528188.
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In this work, a quantitative sandwich ELISA was optimized, through a full factorial design of experiments (DOE) in successive steps of a preliminary protocol obtained by the method of one factor at a time (OFAT). The specificity of the optimized ELISA, the lower limit of quantification, the quantification range and the analytical sensitivity of the antigen quantification curve were evaluated, in comparison with the curve obtained from the preliminary protocol. The full factorial DOE was linked to a simple statistical processing, which facilitates the interpretation of the results in those laboratories where there is no trained statistician. The step-by-step optimization of the ELISA and the successive incorporation into the protocol of the best combination of factors and levels, allowed obtaining a specific immunoassay, with an analytical sensitivity 20 times greater and with a lower limit of antigen quantification that decreased from 156.25 at 9.766 ng/mL. As far as we know, there are no reports of optimization of an ELISA following the step-by-step scheme used in this work. The optimized ELISA will be used for the quantification of the TT-P0 protein, the active principle of a vaccine candidate against sea lice.
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Antígenos , Ensaio de Imunoadsorção Enzimática/métodos , ImunoensaioRESUMO
OBJECTIVE: Cohen's d conventional effect size cutoffs [small (0.2), medium (0.5), and large (0.8)] might not be representative of the reported distribution of effect sizes across the different areas of health. Effect size cutoffs might vary not only depending on the area of research, but also on the type of intervention and population. That is, they are context dependent. Therefore, we present strategies to redefine small, medium, and large effect size based on 25, 50, and 75th percentile, respectively. METHODS: We illustrate these techniques applying them to 72 effect sizes, derived from 65 randomized controlled trials described in a recent meta-analysis (10.1016/j.smrv.2021.101556) of improving sleep quality on composite mental health. Such percentiles are equally distanced from the average effect size as suggested by Jacob Cohen and checked for potential attenuation effects (via weight selection model) and outliers (via OutRules). RESULTS: new cutoffs for effect size distribution of -0.177, -0.329, and -0.557, for small, medium, and large effect size were found, respectively. applying Cohen's effect size thresholds (0.2, 0.5, and 0.8) for trials of improving sleep quality on composite mental health might over-estimate effect sizes compared to the real-world context, especially around medium and large effect sizes.
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Saúde Mental , Qualidade do Sono , HumanosRESUMO
Effect sizes of school-based intervention are commonly described as small to moderate when using Cohen's conventional effect size cutoffs (small [0.2], medium [0.5], and large [0.8]). However, Jacob Cohen's rule of thumb might vary across different areas of research, nature of the intervention, and population, because effect sizes are context-dependent. Moreover, when planning research studies, minimum detectable effect sizes are used to calculate the sample size. In the present study, we investigate whether conventional effect size cutoffs (small [0.2], medium [0.5], and large [0.8]) represent the reported distribution of effect sizes in school-based anti-bullying intervention. To determine small, medium, and large effect sizes, we calculated the effect size distribution (ESD) using 50th percentile effect size (medium effect) of the distributions of effect sizes provided by a recent meta-analysis on school-based anti-bullying intervention. Also, the 25th and 75th percentile effects, as they are equidistant from the average effect size, were used redefining small and large effects, respectively. Results showed that 0.07, 0.123, and 0.227 represent small, medium, and large effect sizes in anti-bullying interventions. Our results indicate that Cohen's suggested effect size thresholds (0.2, 0.5, and 0.8) overestimate effect sizes when compared to the real-world context of school based anti-bullying interventions. We also propose sample sizes required to reliably detect small, medium, and large percentile effect sizes in anti-bullying interventions.
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Bullying , Humanos , Bullying/prevenção & controle , Instituições Acadêmicas , Projetos de PesquisaRESUMO
Background: An increased number of breast cancer patients are challenged by acute and persistent treatment side effects. Oncology guidelines have been establishing physical exercise to counteract several treatment-related toxicities throughout cancer care. However, evidence regarding the optimal dose-response, feasibility, and the minimal resistance exercise volume and/or intensity remains unclear. The ABRACE Study will assess the impact of different resistance training volumes (i.e., single or multiple sets) combined with aerobic exercise on physical and psychological outcomes of breast cancer patients undergoing primary treatment. Methods: This study is a randomized, controlled, three-armed parallel trial. A total of 84 participants, aged ≥18 years, with breast cancer stages I-III, initiating adjuvant or neoadjuvant chemotherapy (≤50% of sessions completed) will be randomized to multiple sets resistance training plus aerobic training group, single set resistance training plus aerobic training group or control group. Neuromuscular and cancer-related fatigue (primary outcomes), muscle strength, muscle thickness, muscle quality by echo intensity, body composition, cardiorespiratory capacity, functional performance, upper-body endurance and quality of life will be measured before and after the 12-week intervention. Our analysis will follow the intention-to-treat approach and per-protocol criteria, with additional sub-group analysis. Discussion: Findings support prescribing exercise during chemotherapy for breast cancer and elucidate the potential role of different resistance training volumes as a management strategy for physical and psychological impairments in women with early-stage breast cancer. Our main hypothesis is for superiority in physical and psychological outcomes for both training groups compared to the control group, with no difference between single or multiple sets groups. Trial registration: Clinical trials NCT03314168.
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Metanálise consiste em um conjunto de técnicas estatísticas que visa integrar os resultados de dois ou mais estudos primários. Ela permite produzir estimativas pontuais e intervalares de algum parâmetro populacional, geralmente uma medida de tamanho de efeito. Este artigo tem como objetivo apresentar conceitos fundamentais sobre metanálise e suas aplicações para psicólogos e estudantes de psicologia. O artigo: (1) introduz a lógica da metanálise, seus potenciais e as críticas a ela endereçadas; (2) apresenta dois modelos de metanálise comumente usados por pesquisadores; e (3) aborda dois tópicos importantes para a interpretação correta dos resultados: heterogeneidade e análise de subgrupos. Um exemplo fictício ilustra os conceitos ao longo do artigo. Os Materiais Suplementares contêm equações dos modelos apresentados no texto, resultados comentados de uma síntese metanalítica, código na linguagem R para reproduzir resultados e figuras desse artigo e uma breve lista comentada de fontes adicionais sobre metanálise. (AU)
Meta-analysis consists of a set of statistical techniques that aims to combine the results of two or more primary studies. It enables the calculation of point and interval estimates of some population parameter, usually a measure of effect size. The aim of this article is to introduce fundamental concepts of meta-analysis and its applications for psychologists and psychology students. The article: (1) introduces the logic of meta-analysis, its uses and common criticisms levied against it; (2) presents two computational models of meta-analysis commonly used by researchers; and (3) addresses two issues associated with the correct interpretation of results from meta-analyses: heterogeneity and subgroup analysis. A worked example illustrates the concepts throughout the article. The Supplementary Materials contain a worked example of the models presented in the text, a script in R language that allows the reader to reproduce the results, and a commented list of additional sources. (AU)
El metanálisis consiste en un conjunto de técnicas estadísticas que tiene como objetivo integrar los resultados de dos o más estudios primarios. Permite producir estimaciones puntuales y de intervalo de algún parámetro de población, generalmente una medida del tamaño del efecto. Este artículo presenta conceptos fundamentales sobre el metanálisis y sus aplicaciones para psicólogos y estudiantes de psicología. El artículo: (1) introduce la lógica del metanálisis, sus potencialidades y las críticas que se le dirigen; (2) presenta dos modelos de metanálisis comúnmente utilizados por los investigadores; y (3) aborda dos temas importantes para la correcta interpretación de los resultados: heterogeneidad y análisis de subgrupos. Un ejemplo ficticio ilustra los conceptos a lo largo del artículo. Los Materiales Suplementarios contienen ecuaciones de los modelos presentados en el texto, resultados comentados de una síntesis metanalítica, código en el lenguaje R para reproducir los resultados y las figuras de este artículo, y una breve lista comentada de fuentes adicionales. (AU)
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Revisões Sistemáticas como Assunto , Simulação por Computador , Revisão , Metanálise , Estatística , Estudos de Avaliação como AssuntoRESUMO
O estudo apresenta os tamanhos do efeito, seus intervalos de confiança, classificações e visualizações para a atualização normativa de 2018 do Teste não verbal de Inteligência R-1, visto que as diferenças estatisticamente calculadas com base no valor p oferecerem evidências tênues contra a hipótese nula e, por isso, não servem como uma prova da significância clínica. Utilizou-se a amostra normativa do instrumento, composta por 5.595 adultos, de ambos os sexos, com idades entre 18 e 65 anos, provenientes das cinco regiões geográficas brasileiras e subdivididos pelos níveis de ensino fundamental, médio e superior. Os resultados apontaram tamanhos do efeito entre médios e grandes na comparação entre os níveis de ensino fundamental e médio; grande a muito grande na relação entre o ensino fundamental e o superior e; pequenos a médios entre os níveis médio e superior de ensino. Concluiu-se que as diferenças estatisticamente observadas, por meio das análises de variância e pos-hoc de Tukey refletem um efeito real quando se considera o impacto da variável nível de escolaridade no desempenho do teste. Entendeu-se que os estudos normativos do instrumento refletem um impacto real quando se considera a variável em estudo, devendo incluir o tamanho do efeito e seus respectivos intervalos de confiança.
The study presents the effect sizes, their confidence intervals, ratings, and visualizations for the 2018 normative update of the R-1 Nonverbal Intelligence Test, since the statistically calculated differences based on the p-value offer tenuous evidence against the null hypothesis and do not serve as a proof of clinical significance. We used the normative sample of the instrument, consisting of 5,595 adults of both sexes, aged 18 to 65 years, from the five Brazilian geographic regions and subdivided by elementary, middle, and high school levels. The results showed medium to large effect sizes in the comparison between elementary and high school levels; large to very large in the relation between elementary and high school; and small to medium effect sizes between high school and high school levels. It was concluded that the statistically observed differences, by means of variance and Tukey's post-hoc analyses reflect a real effect when considering the impact of the education level variable on test performance. It was understood that normative studies of the instrument reflect a real impact when considering the variable under study, and should include the effect size and their respective confidence intervals.
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Testes de Inteligência , Benchmarking , Desempenho AcadêmicoRESUMO
Informing in the studies about the effect size of an intervention or the impact of the factor(s) about an outcome, allows better decision-making for the application of the results in clinical practice. This article presents different methods to analyze the effect size, which can be through direct or indirect statistical methods. Within the direct methods, there's the difference in means between groups and the difference of absolute or relative frequencies. Among the indirect methods, Cohen's "d" family (which are based on standard deviation values), the "r and R2" family, measures of association (e.g. OR, RR, HR), and impact measures (e.g. NNT) are shown. The decision to use any of these methods depends on the objectives of the study and the measuring scale that is used to assess the results, as well as the data distribution. In order to enhance the understanding of the methods described in this article, examples are included, and the need to include level of precision (e.g. confidence intervals) is highlighted, along with the clinical decision thresholds for a better interpretation.
Informar en los estudios sobre el tamaño del efecto de una intervención o del impacto de factor(es) sobre un desenlace, permite tomar mejores decisiones para la aplicación de los resultados a la práctica clínica. En este artículo se presenta la manera de analizar el tamaño del efecto, lo cual puede ser mediante métodos estadísticos directos o indirectos. Dentro de los métodos directos, se encuentra la diferencia de promedios entre grupos y la diferencia de frecuencias absolutas o relativas. Dentro de los métodos indirectos se muestran los índices de la familia de "d" de Cohen (que se basan en valores de desviación estándar), la familia de "r y R2", medidas de asociación (RM, RR, HR) e impacto (NNT). La decisión del uso de cualquiera de los métodos descritos, depende de los objetivos del estudio, la escala de medición usada para evaluar los resultados y la distribución de los datos. Para facilitar la comprensión, se incluyen ejemplos y se resalta la necesidad de incluir los diferentes estadísticos con su nivel de precisión (ej. intervalos de confianza), junto con los umbrales clínicos de decisión, a fin de mejorar su interpretación.
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Coleta de Dados , HumanosRESUMO
OBJECTIVE: Studies in rodents associated the deficits of adult hippocampal neurogenesis with behavioural anomalies which may be reversed by antidepressant treatments. A previous systematic review (SR) and meta-analysis (MA) indicated a hierarchy within the proneurogenic effects of different antidepressants in naive rodents. The present review aims to evaluate a more comprehensive sample of studies investigating the links between the effects of different antidepressants and adult hippocampal neurogenesis. SEARCH STRATEGY SCREENING ANNOTATION DATA MANAGEMENT: Protocols were planned following Preferred Reporting Items for Systematic Review and Meta-Analysis Protocols guidelines. Searches in Embase, Medline, Scopus and Web of Science followed by screening with inclusion/exclusion criteria will provide relevant publications. First SR will summarise the effects of antidepressants on adult hippocampal neurogenesis on different laboratory rodents. Second SR will summarise the correlations between neurogenic and behavioural effects of antidepressants while the third will focus on cause-effect relationships between them. If feasible, data will be analysed by pairwise or network random-effect or multivariate MA to determine the direction, magnitude, significance and heterogeneity (I2) of the estimated effect sizes on global or subgroup levels. Funnel plotting, Egger regression, 'trim and fill' will be used to estimate the risk of publication bias. Quality assessment of the included publications will be performed by applying adapted CAMARADES, Syrcles' risk of bias or CINeMA tools. REPORTING: Find a preliminary version of this protocol at the Open Science Framework (https://osf.io/gmsvd/). Data extraction has already started. Results shall be published in a peer-reviewed journal. Due to the continuous production in the field, the implementation of a 'living SR' is intended.
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Spinocerebellar ataxia type 7 (SCA7) is a neurodegenerative disease characterized by progressive ataxia and retinal degeneration. Previous cross-sectional studies show a significant decrease in the gray matter of the cerebral cortex, cerebellum, and brainstem. However, there are no longitudinal studies in SCA7 analyzing whole-brain degeneration and its relation to clinical decline. To perform a 2-year longitudinal characterization of the whole-brain degeneration and clinical decline in SCA7, twenty patients underwent MRI and clinical evaluations at baseline. Fourteen completed the 2-year follow-up study. A healthy-matched control group was also included. Imaging analyses included volumetric and cortical thickness evaluation. We measured the cognitive deterioration in SCA7 patients using MoCA test and the motor deterioration using the SARA score. We found statistically significant differences in the follow-up compared to baseline. Imaging analyses showed that SCA7 patients had severe cerebellar and pontine degeneration compared with the control group. Longitudinal follow-up imaging analyses of SCA7 patients showed the largest atrophy in the medial temporal lobe without signs of a progression of cerebellar and pontine atrophy. Effect size analyses showed that MRI longitudinal analysis has the largest effect size followed by the SARA scale and MoCA test. Here, we report that it is possible to detect significant brain atrophy and motor and cognitive clinical decline in a 2-year follow-up study of SCA7 patients. Our results support the hypothesis that longitudinal analysis of structural MRI and MOCA tests are plausible clinical markers to study the natural history of the disease and to design treatment trials in ecologically valid contexts.
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Substância Cinzenta/diagnóstico por imagem , Doenças Neurodegenerativas/diagnóstico por imagem , Ataxias Espinocerebelares/diagnóstico por imagem , Adolescente , Adulto , Atrofia , Encéfalo/patologia , Encéfalo/fisiopatologia , Cerebelo/diagnóstico por imagem , Córtex Cerebral/diagnóstico por imagem , Progressão da Doença , Feminino , Seguimentos , Substância Cinzenta/fisiopatologia , Humanos , Processamento de Imagem Assistida por Computador , Estudos Longitudinais , Imageamento por Ressonância Magnética , Masculino , Testes de Estado Mental e Demência , Pessoa de Meia-Idade , Doenças Neurodegenerativas/fisiopatologia , Ponte/diagnóstico por imagem , Ataxias Espinocerebelares/fisiopatologia , Aprendizagem Verbal , Adulto JovemRESUMO
Diagnosis of Parkinson's disease (PD) remains a challenge in clinical practice, mostly due to lack of peripheral blood markers. Transcriptomic analysis of blood samples has emerged as a potential means to identify biomarkers and gene signatures of PD. In this context, classification algorithms can assist in detecting data patterns such as phenotypes and transcriptional signatures with potential diagnostic application. In this study, we performed gene expression meta-analysis of blood transcriptome from PD and control patients in order to identify a gene-set capable of predicting PD using classification algorithms. We examined microarray data from public repositories and, after systematic review, 4 independent cohorts (GSE6613, GSE57475, GSE72267 and GSE99039) comprising 711 samples (388 idiopathic PD and 323 healthy individuals) were selected. Initially, analysis of differentially expressed genes resulted in minimal overlap among datasets. To circumvent this, we carried out meta-analysis of 17,712 genes across datasets, and calculated weighted mean Hedges' g effect sizes. From the top-100- positive and negative gene effect sizes, algorithms of collinearity recognition and recursive feature elimination were used to generate a 59-gene signature of idiopathic PD. This signature was evaluated by 9 classification algorithms and 4 sample size-adjusted training groups to create 36 models. Of these, 33 showed accuracy higher than the non-information rate, and 2 models built on Support Vector Machine Regression bestowed best accuracy to predict PD and healthy control samples. In summary, the gene meta-analysis followed by machine learning methodology employed herein identified a gene-set capable of accurately predicting idiopathic PD in blood samples.
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Doença de Parkinson , Transcriptoma , Algoritmos , Perfilação da Expressão Gênica , Humanos , Doença de Parkinson/diagnóstico , Doença de Parkinson/genética , Máquina de Vetores de Suporte , Transcriptoma/genéticaRESUMO
BACKGROUND: The natural history of neurodegeneration in spinocerebellar ataxia type 3/Machado Joseph disease is still unclear. Here, we built a long-term longitudinal clinical and neuroimaging study to address this point. METHODS: Twenty-three patients with spinocerebellar ataxia type 3/Machado Joseph disease and 22 healthy controls underwent 3T MRI twice 5.0 years apart. T1 and diffusion tensor imaging sequences were obtained. We used T1 multiatlas, diffusion tensor imaging multiatlas, SpineSeg, and CERES-SUIT for cerebral gray and white matter, spinal cord and cerebellar analyses, respectively. Clinical severity was assessed with scale for assessment and rating of ataxia. Analysis of covariance evaluated longitudinal between-group changes. Effect sizes were calculated for each significant result. RESULTS: Progressive volumetric abnormalities were most evident in the cerebellum (Lobule X and Crus II; effect size, 2.0), followed by the basal ganglia (effect size, 0.7). The cerebellar peduncles had the largest white-matter diffusivity changes (effect size, 1.29). Scale for assessment and rating of ataxia-related effect size was 0.82. We failed to identify progressive spinal cord abnormalities. CONCLUSIONS: Longitudinal changes in spinocerebellar ataxia type 3/Machado Joseph disease are more evident in the cerebellum and connections, followed by the basal ganglia. © 2020 International Parkinson and Movement Disorder Society.
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Doença de Machado-Joseph , Ataxias Espinocerebelares , Cerebelo , Imagem de Tensor de Difusão , Humanos , Doença de Machado-Joseph/diagnóstico por imagem , Doença de Machado-Joseph/genética , Imageamento por Ressonância Magnética , Ataxias Espinocerebelares/diagnóstico por imagem , Ataxias Espinocerebelares/genéticaRESUMO
El cálculo de tamaño de muestra es un aspecto esencial del diseño de estudios cuantitativos. Un adecuado tamaño de muestra nos permite determinar cuál es la mínima cantidad de participantes necesarios para probar nuestra hipótesis de interés. De esta manera, podemos reducir costos, maximizar el uso de nuestros recursos de investigación y garantizar la factibilidad del estudio. Contradictoriamente, a pesar de su relevancia muy pocos investigadores dominan esta habilidad. Esta revisión tiene por objeto revisar los conceptos básicos para realizar un cálculo de tamaño de muestra y compartir códigos de Stata y R específicamente diseñados para facilitar estos cálculos.
The calculation of sample size is an essential aspect of the design of quantitative studies. An adequate sample size allows us to determine the minimum number of participants necessary to test our hypothesis of interest. Hence, we can reduce costs, maximize the use of our research resources and guarantee the feasibility of the study. Contradictorily, despite its relevance, very few researchers dominate this skill. This review aims to review the basics of sample size calculation and share Stata and R codes specifically designed to facilitate these calculations.
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Este trabajo presenta un programa en MS Excel® para evaluar la magnitud del efecto (ES, por las siglas en inglés) en invarianza de medición de diferentes parámetros como las cargas factoriales, interceptos/thresholds y residuales, con base en estadísticos estandarizados ya conocidos. El funcionamiento del programa y la interpretación de las medidas de ES se ejemplificaron con datos empíricos.
This paper presents a MS Excel® program for estimating effect size (ES) in the measurement invariance of different item parameters, such as the factor loadings, intercepts/thresholds and residuals, based in well-known standardized statistics. Examples with real data are provided for the functioning of the program and the interpretation of the ES measures.
Este artigo apresenta um programa no MS Excel® para avaliar o tamanho do efeito (ES, pela sigla em inglês) na invariância de medida de diferentes parâmetros dos itens, como cargas fatoriais, interceptos/thresholds e resíduos, com base em estatísticas padronizadas bem conhecidas. O funcionamento do programa e interpretação das medidas de ES é exemplificado com dados empíricos.
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Abstract Introduction: Binaurally evoked auditory evoked potentials have good diagnostic values when testing subjects with central auditory deficits. The literature on speech-evoked auditory brainstem response evoked by binaural stimulation is in fact limited. Gender disparities in speech-evoked auditory brainstem response results have been consistently noted but the magnitude of gender difference has not been reported. Objective: The present study aimed to compare the magnitude of gender difference in speech-evoked auditory brainstem response results between monaural and binaural stimulations. Methods: A total of 34 healthy Asian adults aged 19-30 years participated in this comparative study. Eighteen of them were females (mean age = 23.6 ± 2.3 years) and the remaining sixteen were males (mean age = 22.0 ± 2.3 years). For each subject, speech-evoked auditory brainstem response was recorded with the synthesized syllable /da/ presented monaurally and binaurally. Results: While latencies were not affected (p > 0.05), the binaural stimulation produced statistically higher speech-evoked auditory brainstem response amplitudes than the monaural stimulation (p < 0.05). As revealed by large effect sizes (d > 0.80), substantive gender differences were noted in most of speech-evoked auditory brainstem response peaks for both stimulation modes. Conclusion: The magnitude of gender difference between the two stimulation modes revealed some distinct patterns. Based on these clinically significant results, gender-specific normative data are highly recommended when using speech-evoked auditory brainstem response for clinical and future applications. The preliminary normative data provided in the present study can serve as the reference for future studies on this test among Asian adults.
Resumo Introdução: Potenciais auditivos evocados de modo binaural apresentam bons valores diagnósticos ao testar indivíduos com déficits auditivos centrais. A literatura sobre a resposta do potencial evocado do tronco encefálico com estímulo de fala de modo binaural é, de fato, limitada. As diferenças de gênero nos resultados desse exame têm sido consistentemente observadas, mas a magnitude da diferença de gênero ainda não foi relatada. Objetivo: Comparar a magnitude da diferença de gênero nos resultados do potencial evocado do tronco encefálico com estímulo de fala entre estímulos monaural e binaural. Método: Um total de 34 adultos asiáticos saudáveis com idades entre 19 e 30 anos participaram deste estudo comparativo. Dezoito deles eram do sexo feminino (média de idade = 23,6 ± 2,3 anos) e os outros dezesseis do sexo masculino (média de idade = 22,0 ± 2,3 anos). Para cada indivíduo, o potencial evocado do tronco encefálico com estímulo de fala foi registrado com a sílaba sintetizada /da/ apresentada de forma monaural e binaural. Resultados: Embora as latências não tenham sido afetadas (p > 0,05), a estimulação binaural produziu amplitudes de potencial evocado do tronco encefálico com estímulo de fala estatisticamente maiores do que a estimulação monaural (p < 0,05). Como demonstrado pelos grandes tamanhos de efeito (d > 0,80), diferenças substanciais de gênero foram observadas na maioria dos picos de potencial evocado do tronco encefálico com estímulo de fala para ambos os modos de estímulo. Conclusão: A magnitude da diferença de gênero entre os dois modos de estímulo revelou alguns padrões distintos. Com base nesses resultados clinicamente significantes, os dados normativos específicos para o gênero são altamente recomendados quando se usa o potencial evocado do tronco encefálico com estímulo de fala para aplicações clínicas e futuras. Os dados normativos preliminares fornecidos pelo presente estudo podem servir como referência para futuros estudos sobre esse exame em asiáticos adultos.
Assuntos
Humanos , Masculino , Feminino , Adulto Jovem , Fala/fisiologia , Percepção da Fala/fisiologia , Estimulação Acústica/métodos , Fatores Sexuais , Potenciais Evocados Auditivos do Tronco Encefálico/fisiologia , Voluntários SaudáveisRESUMO
INTRODUCTION: Binaurally evoked auditory evoked potentials have good diagnostic values when testing subjects with central auditory deficits. The literature on speech-evoked auditory brainstem response evoked by binaural stimulation is in fact limited. Gender disparities in speech-evoked auditory brainstem response results have been consistently noted but the magnitude of gender difference has not been reported. OBJECTIVE: The present study aimed to compare the magnitude of gender difference in speech-evoked auditory brainstem response results between monaural and binaural stimulations. METHODS: A total of 34 healthy Asian adults aged 19-30 years participated in this comparative study. Eighteen of them were females (mean age=23.6±2.3 years) and the remaining sixteen were males (mean age=22.0±2.3 years). For each subject, speech-evoked auditory brainstem response was recorded with the synthesized syllable /da/ presented monaurally and binaurally. RESULTS: While latencies were not affected (p>0.05), the binaural stimulation produced statistically higher speech-evoked auditory brainstem response amplitudes than the monaural stimulation (p<0.05). As revealed by large effect sizes (d>0.80), substantive gender differences were noted in most of speech-evoked auditory brainstem response peaks for both stimulation modes. CONCLUSION: The magnitude of gender difference between the two stimulation modes revealed some distinct patterns. Based on these clinically significant results, gender-specific normative data are highly recommended when using speech-evoked auditory brainstem response for clinical and future applications. The preliminary normative data provided in the present study can serve as the reference for future studies on this test among Asian adults.
Assuntos
Estimulação Acústica/métodos , Potenciais Evocados Auditivos do Tronco Encefálico/fisiologia , Fatores Sexuais , Percepção da Fala/fisiologia , Fala/fisiologia , Adulto , Feminino , Voluntários Saudáveis , Humanos , Masculino , Adulto JovemRESUMO
Dando continuidade aos artigos da série "Perguntas que você sempre quis fazer, mas nunca teve coragem", que tem como objetivo responder e sugerir referências para o melhor entendimento das principais dúvidas dos pesquisadores do Hospital de Clínicas de Porto Alegre sobre estatística, este segundo artigo se propõe a responder às principais dúvidas levantadas sobre Teste de Hipóteses. São discutidas questões referentes à metodologia de um teste de hipóteses na concepção clássica de Inferência Estatística, bem como tamanho de efeito, tipos de erros, valor de p e poder. Os conceitos são abordados numa linguagem acessível ao público leigo e diversas referências são sugeridas para os curiosos em relação ao tema. (AU)
Continuing the series of articles "Questions you have always wanted to ask, but never had the courage to", which aims to answer the most common questions of researchers at Hospital de Clínicas de Porto Alegre regarding statistics and to suggest references for a better understanding, this second article addresses the topic of hypothesis testing. The hypothesis testing method is discussed from a classical conception of statistical inference, including effect size, type of errors, p-value and power. The concepts are explained in plain language for lay readers and several references are suggested for those curious about the topic. (AU)