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Mesenchymal stem cells (MSCs) have desirable characteristics for use in therapy in animal models and veterinary medicine, due to their capacity of inducing tissue regeneration and immunomodulation. The objective of this study was to evaluate the differences between canine adipose tissue-derived MSCs (AD-MSCs) extracted from subcutaneous (Sc) and visceral (Vs) sites. Surface antigenic markers, in vitro differentiation, and mineralized matrix quantification of AD-MSCs at different passages (P4, P6, and P8) were studied. Immunophenotypic analysis showed that AD-MSCs from both sites were CD44+, CD90+, and CD45-. Moreover, they were able, in vitro, to differentiate into fat, cartilage, and bone. Sc-AD-MSCs preserve in vitro multipotentiality up to P8, but Vs-AD-MSCs only tri-differentiated up to P4. In addition, compared to Vs-AD-MSCs, Sc-AD-MSCs had greater capacity for in vitro mineralized matrix synthesis. In conclusion, Sc-AD-MSCs have advantages over Vs-AD-MSCs, as Sc AD-MSCs preserve multipotentiality during a greater number of passages, have more osteogenic potential, and require less invasive extraction.

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User satisfaction is known to be related to quality of healthcare. The aim of this study was to evaluate the influence of contextual and individual factors associated with user dissatisfaction with the Brazilian Unified National Health System (SUS). This was a cross-sectional multilevel study. Data were collected via telephone by the ombudsman's office of the SUS. Telephone numbers were randomly selected from a telephone company database. Health services, socioeconomic, and individual demographic variables were evaluated, in addition to information on the municipalities. The outcome variable was dissatisfaction with the SUS. Hierarchical multilevel logistic regression was used, and 18,673 individuals were contacted. Prevalence of dissatisfaction was 63.4% (95%CI: 62.7-64.1). Unmet demand (OR = 3.66), waiting time > 4 hours (OR = 2.82), and number of Primary Healthcare Units (OR = 0.89) were associated statistically with dissatisfaction. Characteristics of the health teams' work process showed a strong association with dissatisfaction.

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Abstract: User satisfaction is known to be related to quality of healthcare. The aim of this study was to evaluate the influence of contextual and individual factors associated with user dissatisfaction with the Brazilian Unified National Health System (SUS). This was a cross-sectional multilevel study. Data were collected via telephone by the ombudsman's office of the SUS. Telephone numbers were randomly selected from a telephone company database. Health services, socioeconomic, and individual demographic variables were evaluated, in addition to information on the municipalities. The outcome variable was dissatisfaction with the SUS. Hierarchical multilevel logistic regression was used, and 18,673 individuals were contacted. Prevalence of dissatisfaction was 63.4% (95%CI: 62.7-64.1). Unmet demand (OR = 3.66), waiting time > 4 hours (OR = 2.82), and number of Primary Healthcare Units (OR = 0.89) were associated statistically with dissatisfaction. Characteristics of the health teams' work process showed a strong association with dissatisfaction.

Resumo: Sabe-se que a satisfação do usuário relaciona-se com a qualidade em saúde. O objetivo foi avaliar a influência de fatores contextuais e individuais associados a insatisfação do usuário com o Sistema Único de Saúde (SUS). Este é um estudo transversal multinível. Os dados foram coletados pela ouvidoria através de contato telefônico. Números de telefone foram selecionados aleatoriamente de um banco de dados de empresas de telefonia. Foram avaliadas variáveis de serviço de saúde, socioeconômicas e demográficas individuais, bem como informações dos municípios. O desfecho foi insatisfação com o SUS. Regressão logística multinível foi utilizada, com uma abordagem hierárquica. 18.673 indivíduos foram contatados. A prevalência de insatisfação foi 63,4% (IC95%: 62,7-64,1). Demanda não resolvida (OR = 3,66), espera > 4 horas (OR = 2,82) e número de unidades básicas de saúde (OR = 0,89) estiveram associados à insatisfação. Características do processo de trabalho das equipes de saúde foram fortemente associadas à insatisfação.

Resumen: Se sabe que la satisfacción del usuario se relaciona con la calidad en salud. El objetivo fue evaluar la influencia de factores contextuales e individuales asociados a la insatisfacción del usuario con el Sistema Único de Salud brasileño (SUS). Este es un estudio transversal multinivel. Los datos fueron recogidos por una auditoría a través de contacto telefónico. Se seleccionaron los números de teléfono aleatoriamente de un banco de datos de empresas de telefonía. Se evaluaron variables de servicio de salud, socioeconómicas y demográficas individuales, así como información de los municipios. El resultado fue insatisfactorio en relación con el SUS. Se utilizó la regresión logística multinivel, con un enfoque jerárquico. 18.673 individuos fueron contactados. La prevalencia de insatisfacción fue de un 63,4% (IC95%: 62,7-64,1). La demanda no resuelta (OR = 3,66), espera > 4 horas (OR = 2,82) y número de unidades básicas de salud (OR = 0,89) estuvieron asociados a la insatisfacción. Características del proceso de trabajo de los equipos de salud estuvieron fuertemente asociadas a la insatisfacción.

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Background. Changes in proprioception may contribute to postural instability in individuals with neurological disorders. Objectives. Evaluate proprioception in the lower limbs of patients with Parkinson's disease (PD) and the association between proprioception and cognitive ability, motor symptoms, postural instability, and disease severity. Methods. This is a cross-sectional, controlled study that evaluated proprioception in PD patients and healthy age- and sex-matched individuals. Kinetic postural proprioception of the knee was evaluated using an isokinetic dynamometer (Biodex® Multi-Joint System 4 Pro). Participants were evaluated using the Montreal Cognitive Assessment (MoCA), the Hoehn and Yahr rating scale and postural instability (pull test and stabilometric analysis), and motor function (UPDRS-III) tests. Results. A total of 40 individuals were enrolled in the study: 20 PD patients and 20 healthy controls (CG). The PD patients had higher angular errors on the proprioceptive ratings than the CG participants (p = 0.002). Oscillations of the center of pressure (p = 0.002) were higher in individuals with PD than in the controls. Proprioceptive errors in the PD patients were associated with the presence of tremors as the dominant symptom and more impaired motor performance. Conclusion. These findings show that individuals with PD have proprioceptive deficits, which are related to decreased cognitive ability and impaired motor symptoms.

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Various methods are available for estimating usual dietary intake distributions. Hence, there is a need for simulation studies to compare them. The methods Iowa State University (ISU), National Cancer Institute (NCI), Multiple Source Method (MSM) and Statistical Program to Assess Dietary Exposure (SPADE) were previously compared in another study, but some results were inconclusive due to the small number of replications used in the simulation. Seeking to overcome this limitation, the present study used 1000 simulated samples for 12 different scenarios to compare the accuracy of estimates yielded by the aforementioned methods. The focus is on scenarios that exhibited the most uncertainty in the conclusions of the mentioned study above, i.e., scenarios with small sample sizes, skewed intake distributions, and large ratios of the between- and within-person variances. Bias was used as a measure of accuracy. For scenarios with small sample sizes (n = 150), the ISU, MSM and SPADE methods generally achieved more accurate estimates than the NCI method, particularly for the 10th and 90th percentiles. The differences between methods became smaller with larger sample sizes (n = 300 and n = 500). With few exceptions, the methods were found to perform similarly.

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Onset of Machado-Joseph disease (SCA3/MJD) before adolescence has been rarely reported. This study aims to describe a cohort of SCA3/MJD with onset before 12 years of age, comparing their disease progression with the progression observed in patients with usual disease onset. We identified all cases from our cohort whose onset was before adolescence. After consent, patients were examined with clinical scales Scale for the Assessment and Rating of Ataxia (SARA) and Neurological Examination Score for Spinocerebellar Ataxia (NESSCA). Gender, age, age at onset, disease duration, CAG expanded repeats, transmitting parent, and anticipation of cases with infantile and adult onset were studied. Progression of NESSCA and SARA scores was estimated through a mixed model, and was compared with a historical group with onset after adolescence. Between 2000 and 2014, 461 symptomatic individuals from our region were diagnosed as SCA3/MJD. Onset of eight cases (2.2%), all heterozygotes, was before adolescence: seven were females (p = 0.054). CAG expanded repeats--75 ± 3 versus 84 ± 4--and anticipations--7 ± 9.7 versus 14.4 ± 7.2 years--were different between early childhood and adult onset groups (p < 0.03). The median survival of early childhood onset group was 23 years of age. The annual progression of SARA--2.3 and 0.6 points/year (p = 0.001)--and NESSCA--2.04 and 0.88 points/year (p = 0.043)--was faster in childhood than in adult onset group. Onset of SCA3/MJD before adolescence was related to larger expanded CAG repeats in heterozygosis; females seemed to be at higher risk. Disease progression was faster than in SCA3/MJD starting after 12 years.

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The aim of the present study is to describe the serum concentrations of a broad spectrum of cytokines in symptomatic and asymptomatic carriers of Machado Joseph disease (SCA3/MJD) CAG expansions. Molecularly confirmed carriers and controls were studied. Age at onset, disease duration, and clinical scales Scale for the Assessment and Rating of Ataxia (SARA), Neurological Examination Score for Spinocerebellar Ataxias (NESSCA), SCA Functional Index (SCAFI), and Composite Cerebellar Functional Score (CCFS) were obtained from the symptomatic carriers. Serum was obtained from all individuals and a cytokine panel "consisted of" eotaxin, granulocyte-macrophage colony-stimulating factor (GM-CSF), interferon (IFN)-α, IFN-γ, interleukin (IL)-1ß, IL-1RA, IL-2, IL-2R, IL-4, IL-5, IL-6, IL-7, IL-8, IL-10, IL-12, IL-13, IL-15, IL-17, interferon gamma-induced protein (IP)-10, monocyte chemoattractant protein (MCP)-1, monokine induced by gamma interferon (MIG), macrophage inflammatory protein (MIP)-a, MIP-b, regulated on activation, normal T cell expressed and secreted (RANTES) and tumor necrosis factor (TNF)-α was analyzed. In a subgroup of symptomatic carriers, the cytokine panel was repeated after 360 days. Cytokine distribution among groups was studied by discriminant analysis; changes in serum levels after 360 days were studied by generalized estimation equation. Sixty-six symptomatic carriers, 13 asymptomatic carriers, and 43 controls were studied. No differences in cytokine patterns were found between controls and carriers of the CAG expansions or between controls and symptomatic carriers only. In contrast, eotaxin concentrations were significantly higher in asymptomatic than in symptomatic carriers or in controls (p = 0.001, ANCOVA). Eotaxin did not correlate with age, disease duration, CAG expansion, NESSCA score, and SARA score. Among symptomatic carriers, eotaxin dropped after 360 days (p = 0.039, GEE). SCA3/MJD patients presented a benign pattern of serum cytokines. In contrast, levels of eotaxin, a peptide secreted by astrocytes, were elevated in the asymptomatic carriers, suggesting that a specific response of these cells can be related to symptom progression, in SCA3/MJD.

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BACKGROUND: In a recent phase 2 clinical trial in spinocerebellar ataxia type 3/Machado Joseph disease (SCA3/MJD), a neurogenetic disorder without specific therapy, benefits of lithium carbonate were found only on secondary efficacy outcomes, all related to ataxic features. In order to help designing future studies, we further analyzed the trial data searching for treatment response modifiers and metric properties of spinocerebellar ataxia (SCA) scales. METHODS: Efficacy analysis was performed with the Neurological Examination Score for the Assessment of Spinocerebellar Ataxia (NESSCA) and the Scale for the Assessment and Rating of Ataxia (SARA) subscores and with the subgroup of patients with independent gait according to the 8-meter walking-time (8MW). Interactions of clinical/molecular findings with treatment response, minimally important differences (MIDs), and sample size estimations for NESSCA, SARA, Spinocerebellar Ataxia Functional Index (SCAFI) and Composite Cerebellar Functional Score (CCFS) were evaluated. RESULTS: 62 SCA3/MJD patients had been randomly assigned (1:1) for the double-blind, placebo-controlled trial. While cerebellar NESSCA (range: 0-7 points) differed between groups 0.64 points (95% CI 0.23 to 1.05, p<0.001) over the whole 48weeks of study, favoring lithium, no effect was found on non-ataxia subscores. Among patients able to perform the 8MW on baseline, NESSCA (p=0.010) and SCAFI (p=0.015) differed between groups favoring lithium. Finally, estimated sample sizes for the scales were provided. CONCLUSION: Lithium efficacy on cerebellar NESSCA, and on SCAFI and CCFS in the primary analysis, together with the lack of effect on non-ataxia features suggests that lithium should be tested in phase 3 trials in SCA3/MJD and that ataxia scales should be preferred to multisystem neurological instruments as the primary outcome. The inclusion of early stage patients is advisable in future clinical trials in SCA3/MJD. TRIAL REGISTRATION: clinicaltrials.gov identifier: NCT01096082.

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BACKGROUND: Disadvantages have already been pointed out on the use of odds ratio (OR) as a measure of association for designs such as cohort and cross sectional studies, for which relative risk (RR) or prevalence ratio (PR) are preferable. The model that directly estimates RR or PR and correctly specifies the distribution of the outcome as binomial is the log-binomial model, however, convergence problems occur very often. Robust Poisson regression also estimates these measures but it can produce probabilities greater than 1. RESULTS: In this paper, the use of Bayesian approach to solve the problem of convergence of the log-binomial model is illustrated. Furthermore, the method is extended to incorporate dependent data, as in cluster clinical trials and studies with multilevel design, and also to analyse polytomous outcomes. Comparisons between methods are made by analysing four data sets. CONCLUSIONS: In all cases analysed, it was observed that Bayesian methods are capable of estimating the measures of interest, always within the correct parametric space of probabilities.

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X-linked adrenoleukodystrophy heterozygote women can present adult onset myeloneuropathy and little is known about its natural history. We aimed to describe the progression rate of the neurological impairment in the prospective follow-up of our cohort and to look for prognostic factors. The neurological scales Japanese Orthopaedic Association (JOA) and Severity Score System for Progressive Myelopathy (SSPROM) were applied at baseline in 29 symptomatic carriers and in follow-up visits. Age at onset, disease duration, X inactivation pattern, determination of the allele expressed, plasma levels of the very long chain fatty acids and of the neuron-specific enolase, and somato-sensory evoked potentials, were taken at baseline. The slope of the linear regression of both JOA and SSPROM versus disease duration since the first symptom was estimated using mixed modeling. JOA and SSPROM decreased 0.42 and 1.87 points per year, respectively (p < 0.001). None of the parameters under study influenced these rates. We estimated that the number of carriers per arm needed in a future 12 month trial with 80% power and a 50% reduction in disease progression would be 225 women for JOA and 750 for SSPROM. The progression rates of the studied neurological scales were small, did not depend on any modifier factor known, and reflected the characteristically slow worsening of symptoms in X-ALD heterozygotes. Better biomarkers are still necessary for future studies.

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INTRODUÇÃO: Diversos estudos mostram o Funcionamento Diferencial do Item (DIF) em itens do Inventário de Depressão Beck (BDI), ao compararem homens e mulheres. A presença de um grande número de itens com DIF no BDI é uma severa ameaça à validade da medida da intensidade de sintomas depressivos obtida pela Teoria da Resposta ao Item (TRI) e às conclusões baseadas nos escores derivados dos itens com e sem DIF. OBJETIVO: Os objetivos deste estudo foram identificar esses itens do BDI, ajustar o modelo de TRI para itens constrangedores (modelo 2), o qual acomoda itens com a presença de DIF, e comparar esses resultados com os do ajuste do modelo logístico de dois parâmetros tradicional da TRI (modelo 1). MÉTODOS: Os resultados obtidos com ambos os modelos foram comparados. RESULTADOS: Os itens que apresentaram DIF foram: tristeza, sentimento de fracasso, insatisfações, culpa, punição, choro, fatigabilidade e perda da libido. Os resultados do ajuste dos dois modelos são similares quanto à discriminação, gravidade (à exceção dos itens com DIF) e no cálculo de escores para os indivíduos. Apesar disso, o modelo 2 é vantajoso, pois mostra as diferenças em gravidade do sintoma depressivo para os grupos avaliados, trazendo, dessa forma, mais informação ao pesquisador sobre a população estudada. CONCLUSÃO: Esse modelo, que tem um alcance mais amplo em termos de população-alvo, pode ser uma ótima alternativa na identificação e acompanhamento de indivíduos com potencial depressivo. .

INTRODUCTION: There are several studies showing the presence of Differential Item Functioning (DIF) in some items of the Beck Depression Inventory (BDI), when comparing men and women. The presence of a large number of items with DIF in BDI is a severe threat to the validity of measurement of the intensity of depressive symptoms obtained by Item Response Theory (IRT) and to the conclusions based on the scores derived from the items with or without DIF. OBJECTIVE: The objectives of this study were to identify these items from the BDI, adjust the IRT model for embarrassing items (model 2), which accommodates items with the presence of DIF, and compare these results with the fit of the traditional two-parameter logistic IRT model (model 1). METHODS: The results obtained with the both models were compared. RESULTS: Items with DIF were: sadness, feeling of failure, dissatisfaction, guilty, punishment, crying, fatigability and loss of libido. The results of the adjustment of the two models are similar in discrimination, gravity (except for items with DIF), and in the calculation of scores for individuals. Nevertheless, model 2 is beneficial because it shows the differences in gravity of depressive symptoms for groups evaluated, thus providing more information to the researcher on the study population. CONCLUSION: This model, which has a broader scope in terms of target population, may be a good alternative to the identification and follow-up of individuals with potential depression. .

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INTRODUCTION: There are several studies showing the presence of Differential Item Functioning (DIF) in some items of the Beck Depression Inventory (BDI), when comparing men and women. The presence of a large number of items with DIF in BDI is a severe threat to the validity of measurement of the intensity of depressive symptoms obtained by Item Response Theory (IRT) and to the conclusions based on the scores derived from the items with or without DIF. OBJECTIVE: The objectives of this study were to identify these items from the BDI, adjust the IRT model for embarrassing items (model 2), which accommodates items with the presence of DIF, and compare these results with the fit of the traditional two-parameter logistic IRT model (model 1). METHODS: The results obtained with the both models were compared. RESULTS: Items with DIF were: sadness, feeling of failure, dissatisfaction, guilty, punishment, crying, fatigability and loss of libido. The results of the adjustment of the two models are similar in discrimination, gravity (except for items with DIF), and in the calculation of scores for individuals. Nevertheless, model 2 is beneficial because it shows the differences in gravity of depressive symptoms for groups evaluated, thus providing more information to the researcher on the study population. CONCLUSION: This model, which has a broader scope in terms of target population, may be a good alternative to the identification and follow-up of individuals with potential depression.

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BACKGROUND: Although aspiration is one of the main causes of death in SCA, such as SCA3/Machado Joseph disease (SCA3/MJD), clinical studies on dysphagia are lacking for these diseases. The aims of this study were to characterize dysphagia in SCA3/MJD through videofluoroscopy (VF) of swallowing, correlate VF with disease severity criteria and weight loss, and determine the clinical criteria cutoffs for performing VF in the clinical routine, in order to detect aspiration. METHODS: A cross-sectional study on 34 SCA3/MJD patients was performed. Clinical and molecular data, as well as body mass index (BMI), were obtained. Neurological scales, such as the Scale for the Assessment and Rating of Ataxia (SARA), and the Swallowing Quality of Life (SWAL-QOL) questionnaire were applied. The VF scores, Dysphagia Outcome and Severity Scale (DOSS) and penetration/aspiration scale (PAS), were obtained: Moderate-to-severe scores were grouped as "significant dysphagia." RESULTS: Overall, 31 of 34 individuals showed abnormal scores at VF. SARA, BMI, and the domain "eating duration" of SWAL-QOL correlated with VF: Their relation to significant dysphagia (DOSS <4 points or PAS >3) was evaluated through receiver operating characteristic curves. A sensitivity of 100% was equivalent to a cutoff of 15 points on SARA score, 23.72 kg/m2 on BMI, and 60% on eating duration-SWAL-QOL (P < 0.05). CONCLUSION: Significant dysphagia was not related to age at onset, disease duration, or CAG repeat expansion, but with SARA scores, lower BMI, and the domain eating duration of SWAL-QOL. As a guideline for preventing aspiration, we suggest that SARA scores greater than 15 or eating duration-SWAL-QOL lower than 60% should urge VF studies in SCA3/MJD.

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BACKGROUND: Obesity and diabetes mellitus are well-defined risk factors for cardiovascular mortality. The impact of antecedent hyperglycemia and body size on mortality in critical ill patients in intensive care units (ICUs) may vary across their range of values. Therefore, we prospectively analyzed the relationship between in-hospital mortality and preexisting hyperglycemia and body size in critically ill ICU patients to understand how mortality varied among normal, overweight, and obese patients and those with low, intermediate, and high glycated hemoglobin (HbA1c) levels. METHODS: Medical history, weight, height, physiologic variables, and HbA1c were obtained during the first 24 h for patients who were consecutively admitted to the high complexity ICU of Hospital de Clínicas de Porto Alegre, Brazil, from April to August 2011. The relationships between mortality and obesity and antecedent hyperglycemia were prospectively analyzed by cubic spline analysis and a Cox proportional hazards model. RESULTS: The study comprised 199 patients. The overall hospital mortality rate was 43.2% during a median 16 (8-28) days of follow-up. There was a progressive risk of in-hospital mortality with higher HbA1c levels, with the relationship becoming significant at HbA1c >9.3% compared with lower levels (hazard ratio 1.74; 95% confidence interval with Bonferroni correction 1.49-2.80). In contrast, mean body mass index (BMI) was higher in survivors than in nonsurvivors (27.2 kg/m2 ± 7.3 vs. 24.7 kg/m2 ± 5.0 P = 0.031, respectively). Cubic spline analysis showed that these relationships differed nonlinearly through the spectrum of BMI values. In a Cox proportional hazards model adjusted for Acute Physiology and Chronic Health Evaluation II score and HbA1c, the risk of in-hospital mortality progressively decreased with increasing BMI (BMI <20 vs. 20-23.9 kg/m2, P = 0.032; BMI <20 vs. 24-34.9 kg/m2, P = 0.010; BMI <20 vs. ≥35 kg/m2, P = 0.032). CONCLUSIONS: Our findings suggest that significant hyperglycemia prior to ICU admission is a risk factor for in-hospital mortality. Conversely, increasing BMI may confer an advantageous effect against mortality in critical illness independently of previous glycemic control.

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Recent studies have emphasized that there is no justification for using the odds ratio (OR) as an approximation of the relative risk (RR) or prevalence ratio (PR). Erroneous interpretations of the OR as RR or PR must be avoided, as several studies have shown that the OR is not a good approximation for these measures when the outcome is common (> 10%). For multinomial outcomes it is usual to use the multinomial logistic regression. In this context, there are no studies showing the impact of the approximation of the OR in the estimates of RR or PR. This study aimed to present and discuss alternative methods to multinomial logistic regression based upon robust Poisson regression and the log-binomial model. The approaches were compared by simulating various possible scenarios. The results showed that the proposed models have more precise and accurate estimates for the RR or PR than the multinomial logistic regression, as in the case of the binary outcome. Thus also for multinomial outcomes the OR must not be used as an approximation of the RR or PR, since this may lead to incorrect conclusions.

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BACKGROUND: Because lithium exerts neuroprotective effects in preclinical models of polyglutamine disorders, our objective was to assess the safety and efficacy of lithium carbonate (0.5-0.8 milliequivalents per liter) in patients with Machado-Joseph disease (spinocerebellar ataxia type 3 [MJD/SCA3]). METHODS: For this phase 2, single-center, double-blind, parallel, placebo-controlled trial (ClinicalTrials.gov identifier NCT01096082), 62 patients who had MJD/SCA3 with a disease duration ≤10 years and an independent gait were randomly assigned (1:1) to receive either lithium or placebo. RESULTS: After 24 weeks, 169 adverse events were reported, including 50.3% in the lithium group (P = 1.00; primary safety outcome). Sixty patients (31 in the placebo group and 29 in the lithium group) were analyzed for efficacy (intention-to-treat analysis). Mean progression between groups did not differ according to scores on the Neurological Examination Score for the Assessment of Spinocerebellar Ataxia (NESSCA) after 48 weeks (-0.35; 95% confidence interval, -1.7 to 1.0; primary efficacy outcome). The lithium group exhibited minor progression on the PATA speech-rate (P = 0.002), the nondominant Click Test (P = 0.023), the Spinocerebellar Ataxia Functional Index (P = 0.003), and the Composite Cerebellar Functional Score (P = 0.029). CONCLUSIONS: Lithium was safe and well tolerated, but it had no effect on progression when measured using the NESSCA in patients with MJD/SCA3. This slowdown in secondary outcomes deserves further clarification.

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Recent studies have emphasized that there is no justification for using the odds ratio (OR) as an approximation of the relative risk (RR) or prevalence ratio (PR). Erroneous interpretations of the OR as RR or PR must be avoided, as several studies have shown that the OR is not a good approximation for these measures when the outcome is common (> 10%). For multinomial outcomes it is usual to use the multinomial logistic regression. In this context, there are no studies showing the impact of the approximation of the OR in the estimates of RR or PR. This study aimed to present and discuss alternative methods to multinomial logistic regression based upon robust Poisson regression and the log-binomial model. The approaches were compared by simulating various possible scenarios. The results showed that the proposed models have more precise and accurate estimates for the RR or PR than the multinomial logistic regression, as in the case of the binary outcome. Thus also for multinomial outcomes the OR must not be used as an approximation of the RR or PR, since this may lead to incorrect conclusions.

Recentes trabalhos têm enfatizado que já não há justificativa para o uso da razão de chances (RC) como aproximação do risco relativo (RR) ou razão de prevalência (RP). Deve-se evitar a interpretação equivocada da RC como RR ou RP, pois vários estudos demonstraram que a RC não é uma boa aproximação para tais medidas quando o desfecho é comum (> 10%). Para desfechos multinomiais é usual aplicar a regressão logística multinomial. Nesse contexto, não há estudos demonstrando o impacto da aproximação da RC nas estimativas de RR ou RP. O objetivo deste trabalho é apresentar e discutir métodos alternativos à regressão logística multinomial, baseados na regressão de Poisson e no modelo log-binomial. As abordagens foram comparadas por um estudo de simulação com diversos cenários. Assim como no caso do desfecho binário, os modelos propostos apresentaram estimativas mais precisas e acuradas para o RR ou RP do que a regressão logística multinomial. Então, também para os desfechos multinomiais não se deve utilizar a RC como aproximação do RR ou RP, pois conclusões incorretas podem ocorrer.

Los trabajos recientes han enfatizado que ya no se justifica el uso del odds ratio (OR) como una aproximación del riesgo relativo (RR) o razón de prevalencias (RP). El OR no puede ser interpretado como RR o RP, pues varios estudios han demostrado que el OR no es una buena aproximación cuando el suceso es común (> 10%). Para sucesos multinomiales se utiliza comúnmente la regresión logística multinomial. En este contexto, no hay estudios que demuestren el impacto de la aproximación del OR en las estimaciones de RR o RP. Nuestro objetivo es presentar y discutir métodos alternativos a la regresión logística multinomial, en base a la regresión de Poisson y al modelo log-binomial. Los enfoques utilizados fueron comparados en un estudio de simulación con diferentes escenarios. Así como en el caso de suceso binario, los modelos propuestos dieron como resultado estimaciones para RR o RP más precisas y esmeradas que la regresión logística multinomial. Para sucesos multinomiales el OR tampoco debe ser utilizado como aproximación del RR o de la RP, pues se puede llegar a conclusiones incorrectas.

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Introdução: Questiona-se se, em situações em que o indivíduo tem múltiplos contatos ao azar com possíveis portadores de um determinado germe patogênico, o risco de infecção pode ser alto, mesmo quando a prevalência de portadores e a infectividade do germe forem baixas. Objetivos: Objetivou-se estabelecer a probabilidade de um indivíduo tornar-se infectado após um determinado número de contatos com possíveis portadores do germe. Métodos: O trabalho foi desenvolvido de forma totalmente teórica, fazendo uso de análise combinatória, indução e dedução e conceitos da teoria de probabilidades. Resultados: Obteve-se que a probabilidade de um indivíduo infectar-se após c contatos ao azar (com pessoas infectadas ou não) é dada pela expressão, onde é a prevalência da infecção entre os contatantes e é a infectividade do germe. Esta expressão permite inferir que o número de contatos necessários para um indivíduo ser infectado é uma variável aleatória com distribuição Geométrica de parâmetro . Conclusão: Conclui-se, aplicando a expressão deduzida, que a probabilidade de infectar-se pode ser alta, mesmo que a prevalência e a infectividade do germe sejam baixas, desde que ocorra um grande número de exposições à fonte de infecção.

Background: The main question is whether, in situations where the individualhas multiple contacts randomly with possible carriers of a particular pathogenicgerm, the risk of infection can be high even when the prevalence of carriers of thegerm and infectivity are low. Aims: This study aimed to establish the probability of an individual becominginfected after a certain number of contacts with possible carriers of the germ.Methods: The study was conducted in a completely theoretical way, usingcombinatorics, induction and deduction and concepts of probability theory.Results: It was found that the probability of an individual becoming infected after c contactsat random (with infected persons or not) is given by the expression 1 - (1 - πy)c, where πis the prevalence of infection among contacted and y is the infectivity of the germ. Thisexpression allows us to infer that the number of contacts required for an individual to beinfected is a random variable with a Geometric distribution of parameter πy. Conclusion: In conclusion, by applying the expression deduced it was noticed that the probability of becoming infected may be high even though the prevalence and infectivity of the germ are low, provided that there is a large number of exposures to the source of infection.

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Introdução: os principais testes estatísticos têm como suposição a normalidade dos dados, que deve ser verificada antes da realização das análises principais. Objetivo: revisar as técnicas de verificação da normalidade dos dados e comparar alguns testes de aderência à normalidade para diferentes distribuições de origem e tamanho amostral. Metodologia: através da simulação de cinco distribuições (Normal, t-student, Qui-Quadrado, Gama e Exponencial) e seis tamanhos amostrais (10, 30, 50, 100, 500 e 1000) foram simulados 5000 amostras de cada par distribuição-tamanho amostral e realizados os testes Qui-quadrado, Kolmogorov-Smirnov, Lilliefors, Shapiro-Wilk, Shapiro-Francia, Cramer-von Mises, Anderson-Darling e Jarque-Bera. Resultados: os resultados obtidos mostram uma clara superioridade dos testes Shapiro-Francia e Shapiro-Wilk, com percentuais de acerto de 72,41% e 72,15%, respectivamente. Entre os piores resultados encontramos o Kolmogorov-Smirnov e Qui-Quadrado, com percentual de acerto de 44,78% e 61,58%, respectivamente. Conclusões: Para amostras pequenas recomenda-se que sejam utilizados procedimentos não paramétricos diretamente para a análise, em função da baixa performance dos testes de aderência à normalidade, dado o baixo percentual de acertos. Para amostras maiores, recomenda-se o uso dos testes Shapiro-Francia ou Shapiro-Wilk.

Introduction: The main statistical tests have the normality assumption that must be verified before performing the main analyzes. Objective: To review the techniques of testing for normality of data and compare some adherence tests for different true distributions and sample size. Methodology: Through simulation of five distributions (Normal, t-Student, Chi-Square, Gamma and Exponential) and six sample sizes (10, 30, 50, 100, 500 and 1000) were simulated 5000 samples of each pair sample size-distribution and applied the Chi-square, Kolmogorov-Smirnov, Lilliefors, Shapiro-Wilk, Shapiro-Francia, Cramer-von Mises, Anderson-Darling and Jarque-Bera tests. Results: The results show a clear superiority of the Shapiro-Francia and Shapiro-Wilk tests, with percentages of accuracy of 72.41% and 72.15% respectively. Among the worst results we find the Kolmogorov-Smirnov and Chi-Square, with percentage of accuracy of 44.78% and 61.58% respectively. Conclusions: For small samples it is recommended to use non-parametric procedures directly for the analyzes, due to the low performance of the tests of adherence to normality, given the low percentage of accuracy. For larger samples, we recommend the use of the Shapiro-Francia and Shapiro-Wilk tests.

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Introdução: A regressão logística está cada dia mais presente nas pesquisas, porém, sabe-se que seus estimadores só possuem boas propriedades se o tamanho de amostra for grande. Entretanto, nem sempre o tamanho amostral utilizado nos estudos é o ideal, sendo às vezes sendo calculados através de regras de bolso. Objetivo: Mostrar o quão inadequadas são estas regras de bolso. Método: Estudo simulado de três cenários. Resultados: Em nossas simulações, encontramos vícios maiores na estimação da razão de chance do que do respectivo coeficiente do modelo. Conclusões: As regras de bolso amplamente utilizadas não garantem boas propriedades na estimação das razões de chances.

Background: Logistic regression has been increasingly used in research recently. However, only large samples can provide reliable predictors. Nevertheless, sample sizes are not always appropriate because sometimes they are calculated based on a rule of thumb. Aim: To demonstrate that these rules of thumb are inappropriate. Method: Simulation study using three scenarios. Results: Our simulations demonstrated higher bias in the odds ratio than in the model coefficient. Conclusions: We concluded that rules of thumb do not guarantee good proprieties for odds ratio estimation.

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