A practical guide to estimating treatment effects in patients with rheumatic diseases using real-world data.

OBJECTIVE: Randomized controlled trials are considered the gold standard in study methodology. However, due to their study design and inclusion criteria, these studies may not capture the heterogeneity of real-world patient populations. In contrast, the lack of randomization and the presence of both measured and unmeasured confounding factors could bias the estimated treatment effect when using observational data. While causal inference methods allow for the estimation of treatment effects, their mathematical complexity may hinder their application in clinical research. METHODS: We present a practical, nontechnical guide using a common statistical package (Stata) and a motivational simulated dataset that mirrors real-world observational data from patients with rheumatic diseases. We demonstrate regression analysis, regression adjustment, inverse-probability weighting, propensity score (PS) matching and two robust estimation methods. RESULTS: Although the methods applied to control for confounding factors produced similar results, the commonly used one-to-one PS matching method could yield biased results if not thoroughly assessed. CONCLUSION: The guide we propose aims to facilitate the use of readily available methods in a common statistical package. It may contribute to robust and transparent epidemiological and statistical methods, thereby enhancing effectiveness research using observational data in rheumatology.

'Spin' in urology non-randomised studies comparing therapeutic interventions: a temporal analysis.

OBJECTIVE: To determine the prevalence of 'spin' (i.e., reporting practices that distort the interpretation of results by positively reflecting negative findings or downplaying potential harms) strategies and level of spin in urological observational studies and whether the use of spin has changed over time. MATERIALS AND METHODS: MEDLINE and Embase were searched to identify observational studies comparing therapeutic interventions in the top five urology journals and major urological subspecialty journals, published between 2000 and 2001, 2010 and 2011, and 2020 and 2021. RESULTS: A total of 235 studies were included. Spin was identified in 81% of studies, with a median of two strategies per study. The most commonly used strategies were inadequate implication for clinical practice (30%), causal language or causal claim (29%), and use of linguistic spin (29%). Moderate to high levels of spin were found in 55% of conclusions. From 2000 to 2020, the average number of strategies used has significantly decreased each decade (H = 27.459, P < 0.001), and the median level of spin in conclusions was significantly lower in studies published in the 2020s and 2010s than in the 2000s (H = 11.649, P = 0.003). CONCLUSIONS: Our results suggest that 81% of urological observational studies comparing therapeutic interventions contained spin. Over the past two decades, the use of spin has significantly declined, but this remains an area for improvement, with 70% of included studies published in the 2020s employing spin. Medical writing should scrupulously avoid words or phrases that are not supported by data in the manuscript.

Triptorelin therapy for lower urinary tract symptoms (LUTS) in prostate cancer patients: A systematic meta-analysis.

Objective: This systematic meta-analysis aimed to assess the effectiveness of triptorelin therapy in reducing lower urinary tract symptoms (LUTS) in men with prostate cancer (PCa). Methods: The Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines were followed. PubMed, Web of Science and EMBASE databases were searched for studies conducted between 2013 and 2023. Eligible studies included PCa patients undergoing androgen deprivation therapy (ADT) with triptorelin, with reported baseline and follow-up International Prostate Symptom Scores (IPSS) and quality of life (QoL) data. The Newcastle-Ottawa Scale (NOS) was used to assess the risk of bias, and a random-effects model was applied for the meta-analysis. Results: A total of 29 articles were identified, and three studies met the inclusion criteria. Triptorelin therapy showed a clinically significant reduction in IPSS over 48 weeks in PCa patients with moderate to severe LUTS. The meta-analysis revealed a pooled effect size of 1.05 (95% CI: 0.65; 1.45), indicating a statistically significant improvement in LUTS. QoL also improved in patients receiving triptorelin therapy, although heterogeneity among the studies and a moderate to high risk of bias were noted. Conclusion: Triptorelin therapy demonstrated a positive impact on LUTS in PCa patients. The meta-analysis showed significant reductions in IPSS scores and improved QoL after 48 weeks of triptorelin treatment. However, the results should be interpreted cautiously due to study heterogeneity and potential biases. Further well-designed studies are needed to confirm these findings and determine the optimal use of triptorelin for managing LUTS in men with PCa. Implications for Practice: Triptorelin therapy may offer an effective treatment option for men with PCa experiencing moderate to severe LUTS. Its positive impact on QoL can lead to improved patient well-being and treatment adherence. Clinicians should consider triptorelin as a potential treatment choice, especially in patients who may be reluctant to undergo surgical interventions for their LUTS. However, careful patient selection and close monitoring are essential due to the observed study heterogeneity and risk of bias. Future research should focus on evaluating triptorelin's cost-effectiveness and comparing its efficacy with other LH-RH agonists in managing LUTS in PCa patients.Video Abstract: URL (Reviewers/Editors to select from) Link 1: https://brighton.cloud.panopto.eu/Panopto/Pages/Viewer.aspx?id=071419c8-1ad5-4502-a222-b04300c2ca5e Link 2: https://brighton.cloud.panopto.eu/Panopto/Pages/Viewer.aspx?id=b6305a8a-b977-4fcd-a69e-b04300bed728.

Safety of herbal decoctions: A scoping review of clinical studies in South Korea focusing on liver and kidney functions.

ETHNOPHARMACOLOGICAL RELEVANCE: Approximately 27% of individuals seeking Korean medicine (KM) services in South Korea are prescribed herbal decoctions. The South Korean government has considered the validity of providing National Health Insurance coverage for herbal decoctions. Therefore, it is important to investigate their safety. AIM OF THE STUDY: To investigate the safety of herbal decoctions commonly prescribed by KM doctors and to assess their effects on liver and kidney function by comprehensively analyzing Korean clinical studies in a scoping review. MATERIALS AND METHODS: The Arksey and O'Malley framework and modified methods were applied in this scoping review. A comprehensive search of seven electronic health databases was conducted, and relevant clinical studies published between 2000 and 2022 were identified. Subsequently, only clinical studies reporting the results of liver and/or renal function tests in patient prescribed herbal decoctions by KM doctors were included. The characteristics of the included clinical studies and the reported proportion of each liver and/or renal function indicator were analyzed. Meta-analyses of the effects of herbal decoction on liver and/or renal function reported in prospective cohort studies were also performed. RESULTS: Fifty-nine clinical studies were included in this review. The proportion of prospective cohort studies markedly decreased in the 2010s compared to the 2000s, while there was no noticeable change in the number of relevant clinical studies. Herbal decoctions were prescribed for less than one month in most included studies. Abnormal changes in liver or renal function indicators were identified in a small number of studies (3.70% and 7.69%, respectively). In a meta-analysis of 15 prospective cohort studies, no statistically significant changes in four liver function indices and two renal function indices were observed before and after the prescription of herbal decoctions. CONCLUSIONS: Qualitative and quantitative analyses demonstrated favorable safety profiles for herbal decoctions. This scoping review includes the gaps noted between clinical application and research regarding the safety profiles of herbal decoctions. These findings could be used as evidence to support the inclusion of herbal decoction prescriptions in the National Health Insurance coverage in South Korea.

Sociodemographic and undergraduate-related variables as predictors of dental students' empathy: an e-survey in a south brazilian dental school / Variáveis sociodemográficas e relacionadas à graduação como preditoras da empatia de estudantes de odontologia: um e-survey em uma faculdade de odontologia do Sul do Brasil

Aim: To assess the associations between sociodemographic and undergraduate-related characteristics and empathy scores among dental students in a school in Southern Brazil. Materials and methods: Cross-sectional study developed from self-administered online questionnaires to undergraduate dental students at the Federal University of Pelotas. Outcomes were the total score of empathy obtained through the Interpersonal Reactivity Index (IRI) and the scores obtained in four domains: perspective-taking (PT), fantasy (FA), empathic concern (EC), and personal distress (PD). In RStudio version 4.1.3 simple and adjusted linear regression models with robust residual standard errors were performed. Results: Eighty-seven students were included (response rate 24.4%). Considering the total IRI score, there was a mean score of 3.62 (SD=0.64) by item, and dental students had a mean score of 94.07 (SD=16.62). In adjusted analysis, being a woman increased the IRI (0.505;95%CI 0.187;0.823), EC (0.494;95%CI 0.168;0.819), and PT (0.822; 95%CI 0.329;1.315) scores compared to men. Adjusted associations were found between the EC domain and skin color and between the PT domain and family income. FA scores increased with age and family income and, decreased with dissatisfaction with undergraduate studies, only in the bivariate analysis. Discussion: Dental professionals' empathy is essential in daily practice, improving the patient-professional relationship in a patient-centered care approach. Thus, it is relevant to recognize predictors of empathy among dental students to promote strategies to increase empathy in dental practice. Conclusion: Gender, skin color, family income, age, and satisfaction with undergraduate studies were associated with empathy scores among dental students.

Objetivo: Avaliar associações entre as características sociodemográficas e relacionadas à graduação e os escores de empatia entre estudantes de odontologia de uma escola do Sul do Brasil. Materiais e métodos: Estudo transversal desenvolvido com questionários on-line autoadministrados para estudantes de graduação em odontologia da Universidade Federal de Pelotas. Foram considerados desfechos o escore total de empatia medido pelo Índice de Reatividade Interpessoal (IRI) e os escores obtidos em quatro domínios: tomada de perspectiva (TP), fantasia (FA), consideração empática (CE) e angústia pessoal (AP). No RStudio versão 4.1.3 foram realizadas regressões lineares simples e múltiplas com erros residuais robustos. Resultados: Foram incluídos 87 alunos (taxa de resposta=24,4%). Considerando a pontuação total do IRI, houve média de 3,62 (DP=0,64) por item, e de 94,07 (DP=16,62) por estudante. Na análise multivariável, mulheres tiveram maiores pontuações no IRI (0,505; IC95%0,187;0,823), CE (0,494; IC95%0,168;0,819) e AP (0,822; IC95%0,329;1,315) em comparação aos homens. Foram encontradas associações ajustadas entre o domínio CE e cor da pele e entre o domínio PT e renda familiar. Os escores do domínio FA aumentaram com idade e renda familiar e diminuíram com insatisfação com a graduação, somente em análise bivariada. Discussão: A empatia do profissional de odontologia é essencial na prática diária, melhorando a relação paciente-profissional. Assim, é relevante reconhecer os preditores da empatia entre os estudantes de odontologia, visando estratégias para aumentar a empatia na prática odontológica. Conclusão: Gênero, cor da pele, renda familiar, idade e satisfação com a graduação foram associados aos escores de empatia entre estudantes de odontologia.

The Effect of Sepsis and Septic Shock on the Viscoelastic Properties of Clot Quality and Mass Using Thromboelastometry: A Prospective Observational Study.

Background: Sepsis is associated with wide variable coagulation abnormalities. Thromboelastography (TEG) effectively measures the viscoelastic properties of the clots. This study aims to illustrate the viscoelastic properties of clot quality and mass in sepsis and septic shock patients using TEG, as an effective tool over standard coagulation tests. Materials and methods: A single-center, prospective observational study was conducted. 50 patients each meeting the criteria for sepsis and septic shock, and a healthy group of 30 patients was included in the study. Blood samples were obtained and analyzed for standard coagulation tests, platelet count, fibrinogen, and TEG study. Results: A total of 130 patients were included. Septic shock patients had a higher sequential (sepsis-related) organ failure score. Prothrombin time (PT) and activated partial thromboplastin time (aPTT) were increased significantly as compared to the sepsis and control groups. TEG markers such as alpha angle, and maximum amplitude (MA) were significantly prolonged while reaction time (R time), was significantly shortened in the sepsis group as compared to the healthy group, suggestive of a hypercoagulable state in sepsis patients. While in septic shock patients, MA and Lysis Index 30 (LY 30) were significantly prolonged and, R time was significantly shortened compared to all other groups. Even though LY30 in sepsis patients was found to be within the normal range (p < 0.001), 18% of patients had prolonged LY30 indicating a hypercoagulable state with impaired fibrinolysis. Conclusion: Thromboelastography, as a point-of-care test combined with conventional coagulation tests can provide additional, clinically relevant information on coagulopathy, and outcome, and thus help guide treatment modality in sepsis and septic shock-induced coagulopathy. How to cite this article: Mohapatra P, Kumar A, Singh RK, Gupta R, Hussain M, Singh S, et al. The Effect of Sepsis and Septic Shock on the Viscoelastic Properties of Clot Quality and Mass Using Thromboelastometry: A Prospective Observational Study. Indian J Crit Care Med 2023;27(9):625-634.

Real-world evidence in the use of Bevacizumab in age-related macular degeneration (ArMD): a scoping review.

PURPOSE: Pharmacological treatments for age-related macular degeneration (ArMD) include anti-vascular endothelial growth factor therapies. Bevacizumab is used off-label, as it has no indication for ArMD. This study aims to identify and describe literature on real-world evidence of bevacizumab (originator or biosimilars) use in ArMD. METHODS: A scoping review was conducted in Medline, CINAHL and Embase databases. Studies published in English after September 2017, conducted in USA, including adults (≥ 18 years old) with ArMD who received treatment with bevacizumab for ArMD were included. The review was further limited to peer-reviewed observational studies that quantitatively analyze either clinical or patient-reported outcomes among patients treated with bevacizumab for ArMD. RESULTS: The search strategy retrieved 543 studies. After title and abstract screening, a total of 142 studies were selected for full-text review leading to a total of 12 studies qualifying for data charting. All were retrospective studies. Five (41.6%) of the studies had less than 500 eyes included in the analysis, and the rest had over a thousand eyes. All except one study reported clinical outcomes (visual acuity was the main outcome in 8 (66.6%) studies). There were 3 (25%) studies reporting adverse events of bevacizumab intravitreal injections. None of the studies specified using biosimilars for bevacizumab and none mentioned patient-reported outcomes. CONCLUSION: The lack of studies aiming to study the patient-reported outcomes as well as the use of biosimilars of bevacizumab in ArMD makes this field a potential for future research. The different exposures and times to follow-up make it difficult to compare results among the selected studies.

Estudios de corte transversal: un diseño de investigación a considerar en ciencias morfológicas / Cross section studies: a research design to consider in morphological sciences

Los estudios de corte transversal (ECT) inician en la década de los 40 en países Europeos. Los ECT corresponden a investigaciones de tipo observacional que permiten estudiar prevalencia de enfermedad, determinar asociación entre variables y el desarrollo de un efecto de interés, conocer propiedades de una prueba diagnóstica, y censar poblaciones; describiendo las características de una población en un momento y lugar determinado. Esto implica, que no se requieren periodos de seguimiento, que no son prospectivos ni retrospectivos; características que permiten que sean más baratos y fáciles de realizar en relación a los estudios longitudinales. Por otro lado, al ser de carácter descriptivo sirven como insumo y evidencia preliminar para estudios de mayor complejidad metodológica, como estudios de cohortes. Sin embargo, tienen limitantes y sesgos que deben ser considerados por los investigadores. El objetivo de este manuscrito fue generar un documento de estudio para revisar características esenciales, fortalezas y debilidades; discutir cuestiones metodológicas de los ECT en ciencias de la salud; y proporcionar algunos ejemplos obtenidos de la literatura, para mejor comprensión del diseño.

SUMMARY: Cross-sectional studies (CSS) began European countries in the 1940s in. The CSSs correspond to observational-type investigations that allow studying the prevalence of disease, determining the association between variables and the development of an effect of interest, discovering the properties of a diagnostic test, and censusing populations, describing the characteristics of a population at a given time and place. This implies that follow-up periods are not required, and that they are neither prospective nor retrospective. These characteristics allow them to be cheaper and easier to perform in relation to longitudinal studies. On the other hand, being descriptive in nature, they serve as input and preliminary evidence for studies of greater methodological complexity, such as cohort studies. However, they have limitations and biases that must be considered by researchers. The aim of this manuscript was to generate a study document to review essential characteristics, strengths and weaknesses; discuss methodological issues of ECT in health sciences; and provide some examples obtained from the literature, for a better understanding of the design.

Análisis mediante variables instrumentales para establecer causalidad en investigaciones observacionales / Analysis through instrumental variables to establish causality in observational research

La identificación de relaciones causales es uno de los problemas fundamentales de la investigación científica en medicina y es necesaria para poder ejercerla en forma efectiva. Sin embargo, desde el punto de vista práctico es difícil establecer la existencia de relaciones causales en estudios de carácter observacional, en gran parte por la presencia de factores de confusión. El análisis a través de variables instrumentales es una de las estrategias que permite controlar el efecto confundidor y documentar la presencia de relaciones causa-efecto en estas situaciones. En este artículo, el autor resume los principales supuestos del análisis a través de variables instrumentales, haciendo foco en la aleatorización mendeliana. (AU)

The identification of causal relationships is one of the fundamental challenges in scientific research in medicine and is necessary for its effective practice. However, from a practical standpoint, establishing the existence of causal relationships in observational studies is difficult, largely due to the presence of confounding factors. Analysis through instrumental variables is one of the strategies that allows to control the confounding effect and documenting the presence of cause-and-effect relationships in these situations. In this article, the author summarizes the main assumptions of analysis through instrumental variables, with a focus on Mendelian randomization. (AU)

Post-hoc power analysis: a conceptually valid approach for power based on observed study data.

Power analysis is a key component of planning prospective studies such as clinical trials. However, some journals in biomedical and psychosocial sciences request power analysis for data already collected and analysed before accepting manuscripts for publication. Many have raised concerns about the conceptual basis for such post-hoc power analyses. More recently, Zhang et al showed by using simulation studies that such power analyses do not indicate true power for detecting statistical significance since post-hoc power estimates vary in the range of practical interests and can be very different from the true power. On the other hand, journals' request for information about the reliability of statistical findings in a manuscript due to small sample sizes is justified since the sample size plays an important role in the reproducibility of statistical findings. The problem is the wording of the journals' request, as the current power analysis paradigm is not designed to address journals' concerns about the reliability of the statistical findings. In this paper, we propose an alternate formulation of power analysis to provide a conceptually valid approach to the journals' wrongly worded but practically significant concern.

The importance of full text screening when judging eligibility criteria in a systematic review: a systematic survey / A importância da avaliação do texto completo ao julgar os critérios de elegibilidade em uma revisão sistemática: uma revisão de métodos

Aim: To evaluate if statistically significant results are more likely to be reported in title/abstracts compared to non-significant outcomes. Methods: In this methodological survey, we reanalyzed 59 observational studies from a previous systematic review. The PECO question was: Patient (P): children with primary teeth; Exposure (E): low birth weight and/or preterm; Comparison (C): normal birth weight and/or full-term; Outcome (O): dental caries. We analyzed the presence of key terms in the titles and abstracts, such as gestational age, preterm, full-term, birth weight, low/normal birth weight. Full texts were analyzed for "positive outcomes" (statistically significant association, p < 0.05 or 95% CI not crossing the null effect line) related to the association between the outcome and the exposure; and "negative outcomes" (when the outcome had statistically similar occurrence between the exposure and the comparison group). The odds ratio (OR) was calculated between the presence of key terms in titles/abstracts and the type of outcome (positive or negative). Results: Of 59 studies, 66% cited the key terms in titles/abstracts, and 75% reported negative outcomes. Studies with positive outcomes were more likely to report key terms in the titles/abstracts compared to studies with negative outcomes (OR: 4.5; 95% CI: 0.9-22.4; Chi-square test: p = 0.06). Studies with statistically significant outcomes, favoring the exposure or the comparison, were more likely to report these data in the titles/abstracts. Conclusion: When conducting a systematic review, the final decision related to the inclusion of a study must be based on a full-text level.

Objetivo: Avaliar se os resultados estatisticamente significativos são mais prováveis de serem relatados nos títulos/resumos dos artigos do que os resultados não significativos. Métodos: Neste levantamento metodológico, foram reanalisados 59 estudos observacionais de uma revisão sistemática anterior. A questão PECO foi: Paciente (P): crianças com dentes decíduos; Exposição (E): baixo peso ao nascer e/ou pré-termo; Comparação (C): peso normal ao nascer e/ou a termo; Resultado (O): cárie dentária. Foi analisada a presença de termos-chave nos títulos/resumos, como idade gestacional, pré-termo, a termo, peso ao nascer, baixo/peso normal ao nascer. Textos completos foram analisados para "desfechos positivos" (associação estatisticamente significativa, p < 0,05 ou IC 95% não cruzando a linha de efeito nulo) relacionados à associação entre o desfecho e a exposição; e "desfechos negativos" (quando o desfecho teve ocorrência estatisticamente semelhante entre a exposição e o grupo de comparação). Foi calculada a odds ratio (OR) entre a presença de termos-chave nos títulos/resumos e o tipo de resultado (positivo ou negativo). Resultados: Dos 59 estudos, 66% citaram os termos-chave nos títulos/resumos e 75% relataram resultados negativos. Estudos com resultados positivos foram mais propensos a relatar os termos-chave nos títulos/resumos em comparação com estudos com resultados negativos (OR: 4,5; IC 95%: 0,9-22,4; teste do qui-quadrado: p = 0,06). Estudos com significância estatística os desfechos, favorecendo a exposição ou a comparação, foram mais propensos a relatar esses dados nos títulos/resumos. Conclusão: Ao realizar uma revisão sistemática, a decisão final quanto à inclusão de um estudo deve ser baseada por meio da análise do texto completo.

Discovery® elbow system arthroplasty: results of 10-year follow-up.

BACKGROUND: The semi-constrained Discovery® Elbow System (LimaCorporate, San Daniele del Friuli, Italy) allows varus-valgus laxity of 7° [8]. It has been reported to provide good pain relief and increased range of motion [5, 9] on mid-term follow-up. The aim of the study was to evaluate long-term outcomes of total elbow arthroplasty using the Discovery® Elbow System (LimaCorporate, San Daniele del Friuli, Italy). MATERIALS AND METHODS: The Mayo Elbow Performance Score (MEPS) and elbow range of motion (ROM) were assessed. Plain radiographs were obtained to assess radiolucency in the humerus and ulna. The data were extracted from electronic patient records. RESULTS: During the follow-up period of 105.4 (range 24.6-179.9) months, 132 patients (153 elbows) underwent surgery. The cause of surgery was rheumatoid arthritis in 105 (71%) cases, posttraumatic or primary arthritis in 17 (13%) and fracture in 10 (6%) patients. The total MEPS increased on average by 35.0 points. Elbow extension deteriorated by 5.0°. Respectively, flexion improved by 10.0° and pronation by 5.0°. The difference in supination was 0.0°. Pain severity improved by 2.5 points in motion and by 5.5 points at rest. During follow-up, 24 (16%) patients needed revision surgery. The most common cause for revision was periprosthetic fracture. Radiolucent lines were seen in all zones in both the ulna and the humerus. The Kaplan-Meier survival at 5 years was 88% and 79% at 10-14 years. CONCLUSION: The Discovery® Elbow System provides good results in ROM and pain relief of the elbow. The revision rate was relatively high (16% of patients). LEVEL OF EVIDENCE: IV.

On the use of the E-value for sensitivity analysis in epidemiologic studies / O uso do valor de E para análise de sensibilidade em estudos epidemiológicos / Sobre el uso del E-value para el análisis de sensibilidad en los estudios epidemiológicos

Abstract: This study illustrates the use of a recently developed sensitivity index, the E-value, helpful in strengthening causal inferences in observational epidemiological studies. The E-value aims to determine the minimum required strength of association between an unmeasured confounder and an exposure/outcome to explain the observed association as non-causal. Such parameter is defined as E - v a l u e = R R + R R R R - 1, where RR is the risk ratio between the exposure and the outcome. Our work illustrates the E-value using observational data from a recently published study on the relationship between indicators of prenatal care adequacy and the outcome low birthweight. The E-value ranged between 1.45 and 5.63 according to the category and prenatal care index evaluated, showing the highest value for the "no prenatal care" category of the GINDEX index and the minimum value for "intermediate prenatal care" of the APNCU index. For "inappropriate prenatal care" (all indexes), the E-value ranged between 2.76 (GINDEX) and 4.99 (APNCU). These findings indicate that only strong confounder/low birthweight associations (more than 400% increased risk) would be able to fully explain the prenatal care vs. low birthweight association observed. The E-value is a useful, intuitive sensitivity analysis tool that may help strengthening causal inferences in epidemiological observational studies.

Resumo: O estudo ilustra o uso de um índice recém-desenvolvido para análise de sensibilidade, o valor de E, útil para embasar inferências causais em estudos epidemiológicos observacionais. O valor de E busca identificar a força mínima da associação entre um fator de confusão não medido e uma exposição/desfecho que seria necessária para explicar a associação observada enquanto não causal, definido como valor de E - v a l u e = R R + R R R R - 1, onde RR é a razão de riscos entre a exposição e o desfecho. Aqui, o valor de E é exemplificado através de dados observacionais de um estudo recém-publicado sobre a relação entre indicadores de adequação da assistência pré-natal e o baixo peso ao nascer enquanto desfecho. O valor de E variou entre 1,45 e 5,63, a depender da categoria e do índice de assistência pré-natal avaliado, com o valor máximo para a categoria "sem assistência pré-natal" do índice GINDEX e o mínimo para "assistência pré-natal intermediária" do índice APNCU. Na categoria "assistência pré-natal inadequada" (todos os índices), o valor de E variou entre 2,76 e 4,99 (índices GINDEX e APNCU, respectivamente). Isso indica que apenas associações fortes entre um fator de confusão e baixo peso ao nascer (aumento de risco maior que 400%) seria capaz de explicar plenamente a associação entre assistência pré-natal e baixo peso ao nascer. O valor de E é uma ferramenta útil e intuitiva para análise de sensibilidade, que pode ajudar a fortalecer as inferências causais em estudos epidemiológicos observacionais.

Resumen: El presente estudio ilustra el uso de un índice desarrollado recientemente para el análisis de la sensibilidad, el E-value, útil para el fortalecimiento de las inferencias causales en los estudios epidemiológicos observacionales. El E-value tiene por objetivo identificar la fortaleza de asociación mínima necesaria entre un factor de confusión no calculable y una exposición/resultado que sería necesario para explicar la asociación observada como no-causal, y está definida como E - v a l u e = R R + R R R R - 1, donde RR es la ratio de riesgo entre la exposición y el resultado. Aquí, el E-value es ilustratado con datos observacionales de un estudio recientemente publicado para mostrar la relación entre indicadores de adecuación de cuidado prenatal y el resultado de bajo peso al nacer. El E-value varió entre 1,45 and 5,63, dependiendo de la categoría y el índice cuidado prenatal evaluado, con su valor más alto para la categoría "no cuidado prenatal" del índice GINDEX y el mínimo para el "cuidado prenatal intermedio" del índice APNCU. En la categoría "inapropiado cuidado prenatal" (todos los índices) el E-value varió entre 2,76 y 4,99 (los índices GINDEX y APNCU, respectivamente). Esto indica que solamente un factor de confusión fuerte/asociaciones bajo peso al nacer (incrementaron el riesgo más de un 400%) habría hecho posible explicar completamente la asociación observada cuidado prenatal vs bajo peso al nacer. El E-value es una herramienta de análisis de sensibilidad útil, intuitiva, que puede ayudar a fortalecer las inferencias causales en los estudios epidemiológicos observacionales.

Comparison of an Online-Only Parkinson's Disease Research Cohort to Cohorts Assessed In Person.

BACKGROUND: Online tools for data collection could be of value in patient-oriented research. The Fox Insight (FI) study collects data online from individuals with self-reported Parkinson's disease (PD). Comparing the FI cohort to other cohorts assessed through more traditional (in-person) observational research studies would inform the representativeness and utility of FI data. OBJECTIVE: To compare self-reported demographic characteristics, symptoms, medical history, and PD medication use of the FI PD cohort to other recent observational research study cohorts assessed with in-person visits. METHODS: The FI PD cohort (n = 12,654) was compared to 3 other cohorts, selected based on data accessibility and breadth of assessments: Parkinson's Progression Markers Initiative (PPMI; PD n = 422), Parkinson's Disease Biomarker Program (PDBP; n = 700), and PD participants in the LRRK2 consortium without LRRK2 mutations (n = 508). Demographics, motor and non-motor assessments, and medications were compared across cohorts. Where available, identical items on surveys and assessments were compared; otherwise, expert opinion was used to determine comparable definitions for a given variable. RESULTS: The proportion of females was significantly higher in FI (45.56%) compared to PPMI (34.36%) and PDBP (35.71%). The FI cohort had greater educational attainment as compared to all other cohorts. Overall, prevalence of difficulties with motor experiences of daily living and non-motor symptoms in the FI cohort was similar to other cohorts, with only a few significant differences that were generally small in magnitude. Missing data were rare for the FI cohort, except on a few variables. DISCUSSION: Patterns of responses to patient-reported assessments obtained online on the PD cohort of the FI study were similar to PD cohorts assessed in-person.

Direct costs of glaucoma: Relationship between cost and severity of the disease.

OBJECTIVES: To estimate the direct medical costs associated with the management of patients with primary open-angle glaucoma and to compare the costs of patients according to the degree of severity. METHODS: A longitudinal retrospective study was carried out using all patients with primary open-angle glaucoma that recorded follow-up from May 2010 to June 2013 at the Hospital Privado de Córdoba. We estimated the cost of the disease from the perspectives of the institution, with a bottom-up approach. RESULTS: The three-year follow-up after treatment of 104 patients revealed that the average cost of care for a patient with primary open-angle glaucoma was US$2746 ± 1560. The first year of treatment was significantly more expensive than subsequent ones (US$1100-$810-$827). Cost was related to the degree of severity of glaucoma; patients in "Stage 0" had significantly lower costs than those in other groups (Kruskal-Wallis test, p < 0.01). This was a consequence of lower costs associated with medication and a lower percentage of patients undergoing surgery. DISCUSSION: The direct medical costs of a patient with primary open-angle glaucoma vary according to the severity of their disease and the year of treatment. We found that costs increased with disease severity, but decreased over time.

Caracterización de los pacientes con defectos bucomaxilofaciales atendidos en el Policlínico Universitario Julio Antonio Mella / Characterization of the patients with oral-maxillofacial defects attended in the University Polyclinic Julio Antonio Mella

RESUMEN Fundamento: las lesiones del macizo cráneofacial existen desde la evolución misma del hombre, lo que ha traído por consiguiente la necesidad de crear medios para enmascarar los defectos o restaurar el órgano perdido. Objetivo: caracterizar los pacientes con defectos bocomaxilofacial. Métodos: se realizó un estudio observacional, descriptivo y transversal desde enero del 2015 hasta mayo de 2018 en pacientes con defectos bucomaxilofaciales atendidos en el departamento de prótesis estomatológica del Policlínico Universitario Julio Antonio Mella de la Provincia Camagüey. El universo lo constituyeron 13 pacientes mayores de 20 años de edad de ambos sexos remitidos del Hospital Universitario Manuel Ascunce Domenech y Hospital Provincial Docente Oncológico María Curie. Resultados: respecto a los pacientes con defectos maxilofaciales rehabilitados según edad y sexo, fue más frecuente el sexo masculino y el grupo de 60 a 79 años. Según tipo y causa del defecto, fue superior el nasal y la oncológica con siete pacientes. Según tipo de defecto bucomaxilofacial y experiencia protésica, primó el defecto nasal, así como la experiencia protésica favorable en ocho pacientes. Conclusiones: los pacientes masculinos predominaron, junto con los mayores de 60 años. Los defectos bucomaxilofaciales más rehabilitados fueron los nasales y de origen oncológicos. Hubo un alto índice de experiencia favorable con el uso de las prótesis.

ABSTRACT Background: the injuries of the solid craniofacial exist from evolution of the man, which has brought therefore the need to create means to mask defects or to restore the lost organ. Objective: to characterize the patients with oral-maxillofacial defects. Methods: an observational, descriptive and transverse study was carried out from January, 2015 to May, 2018, in patients with oral-maxillofacial defects attended in the department dentistry prosthesis of the University Polyclinic Julio Antonio Mella of the city of Camagüey. The universe was constituted by 13 patients older than 20 years of age of both sexes cross-referred of the University Hospital Manuel Ascunce Domenech and Provincial University Oncological Hospital María Curie. Results: in relation to the patients with maxillofacial defects rehabilitated according to age and sex, prevailed the group 60 to 79 years, as well as the masculine sex. According to type and etiology of the defect, the nasal and the oncological was a superior with 7 patients respectively. According to type of defect oral-maxillofacial and prosthetic experience, the type of nasal defect had priority as well as the prosthetic favorable experience in 8 patients. Conclusions: the masculine patients predominated along with the older than 60 years old. The oral-maxillofacial defects most rehabilitated were the nasal and those of oncological causes. There was a tall index of favorable experience with the use of the prostheses.

More Research Is Needed on Lifestyle Behaviors That Influence Progression of Parkinson's Disease.

The variability of symptoms in Parkinson's disease (PD) suggests the need for individualized treatment. A key aspect of precision medicine is lifestyle risk factor modification, known to be important in the prevention and management of chronic illness including other neurological diseases. Diet, cognitive training, exercise, and social engagement affect brain health and quality of life, but little is known of the influence of lifestyle on PD progression. Given disease heterogeneity, absence of objective outcome measures, and the confounding effects of medication, investigating lifestyle as a potential therapy in PD is challenging. This article highlights some of these challenges in the design of lifestyle studies in PD, and suggests a more coordinated international effort is required, including ongoing longitudinal observational studies. In combination with pharmaceutical treatments, healthy lifestyle behaviors may slow the progression of PD, empower patients, and reduce disease burden. For optimal care of people with PD, it is important to close this gap in current knowledge and discover whether such associations exist.

The ENCePP Code of Conduct: A best practise for scientific independence and transparency in noninterventional postauthorisation studies.

PURPOSE: The ENCePP Code of Conduct provides a framework for scientifically independent and transparent pharmacoepidemiological research. Despite becoming a landmark reference, practical implementation of key provisions was still limited. The fourth revision defines scientific independence and clarifies uncertainties on the applicability to postauthorisation safety studies requested by regulators. To separate the influence of the funder from the investigator's scientific responsibility, the Code now requires that the lead investigator is not employed by the funding institution. METHOD: To assess how the revised Code fits the ecosystem of noninterventional pharmacoepidemiology research in Europe, we first mapped key recommendations of the revised Code against ISPE Good Pharmacoepidemiology Practices and the ADVANCE Code of Conduct. We surveyed stakeholders to understand perceptions on its value and practical applicability. Representatives from the different stakeholders' groups described their experience and expectations. RESULTS: Unmet needs in pharmacoepidemiological research are fulfilled by providing unique guidance on roles and responsibilities to support scientific independence. The principles of scientific independence and transparency are well understood and reinforce trust in study results; however, around 70% of survey respondents still found some provisions difficult to apply. Representatives from stakeholders' groups found the new version promising, although limitations still exist. CONCLUSION: By clarifying definitions and roles, the latest revision of the Code sets a new standard in the relationship between investigators and funders to support scientific independence of pharmacoepidemiological research. Disseminating and training on the provisions of the Code would help stakeholders to better understand its advantages and promote its adoption in noninterventional research.