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1.
Elife ; 122024 May 13.
Artigo em Inglês | MEDLINE | ID: mdl-38739437

RESUMO

In several large-scale replication projects, statistically non-significant results in both the original and the replication study have been interpreted as a 'replication success.' Here, we discuss the logical problems with this approach: Non-significance in both studies does not ensure that the studies provide evidence for the absence of an effect and 'replication success' can virtually always be achieved if the sample sizes are small enough. In addition, the relevant error rates are not controlled. We show how methods, such as equivalence testing and Bayes factors, can be used to adequately quantify the evidence for the absence of an effect and how they can be applied in the replication setting. Using data from the Reproducibility Project: Cancer Biology, the Experimental Philosophy Replicability Project, and the Reproducibility Project: Psychology we illustrate that many original and replication studies with 'null results' are in fact inconclusive. We conclude that it is important to also replicate studies with statistically non-significant results, but that they should be designed, analyzed, and interpreted appropriately.


Assuntos
Teorema de Bayes , Reprodutibilidade dos Testes , Humanos , Projetos de Pesquisa , Tamanho da Amostra , Interpretação Estatística de Dados
2.
Trials ; 25(1): 317, 2024 May 14.
Artigo em Inglês | MEDLINE | ID: mdl-38741218

RESUMO

BACKGROUND: Surgical left atrial appendage (LAA) closure concomitant to open-heart surgery prevents thromboembolism in high-risk patients. Nevertheless, high-level evidence does not exist for LAA closure performed in patients with any CHA2DS2-VASc score and preoperative atrial fibrillation or flutter (AF) status-the current trial attempts to provide such evidence. METHODS: The study is designed as a randomized, open-label, blinded outcome assessor, multicenter trial of adult patients undergoing first-time elective open-heart surgery. Patients with and without AF and any CHA2DS2-VASc score will be enrolled. The primary exclusion criteria are planned LAA closure, planned AF ablation, or ongoing endocarditis. Before randomization, a three-step stratification process will sort patients by site, surgery type, and preoperative or expected oral anticoagulation treatment. Patients will undergo balanced randomization (1:1) to LAA closure on top of the planned cardiac surgery or standard care. Block sizes vary from 8 to 16. Neurologists blinded to randomization will adjudicate the primary outcome of stroke, including transient ischemic attack (TIA). The secondary outcomes include a composite outcome of stroke, including TIA, and silent cerebral infarcts, an outcome of ischemic stroke, including TIA, and a composite outcome of stroke and all-cause mortality. LAA closure is expected to provide a 60% relative risk reduction. In total, 1500 patients will be randomized and followed for 2 years. DISCUSSION: The trial is expected to help form future guidelines within surgical LAA closure. This statistical analysis plan ensures transparency of analyses and limits potential reporting biases. TRIAL REGISTRATION: Clinicaltrials.gov, NCT03724318. Registered 26 October 2018, https://clinicaltrials.gov/study/NCT03724318 . PROTOCOL VERSION: https://doi.org/10.1016/j.ahj.2023.06.003 .


Assuntos
Apêndice Atrial , Fibrilação Atrial , Procedimentos Cirúrgicos Cardíacos , Estudos Multicêntricos como Assunto , Ensaios Clínicos Controlados Aleatórios como Assunto , Acidente Vascular Cerebral , Humanos , Apêndice Atrial/cirurgia , Fibrilação Atrial/cirurgia , Fibrilação Atrial/complicações , Acidente Vascular Cerebral/prevenção & controle , Acidente Vascular Cerebral/etiologia , Procedimentos Cirúrgicos Cardíacos/efeitos adversos , Fatores de Risco , Resultado do Tratamento , Medição de Risco , Interpretação Estatística de Dados , Ataque Isquêmico Transitório/prevenção & controle , Ataque Isquêmico Transitório/etiologia , Masculino , Feminino , Oclusão do Apêndice Atrial Esquerdo
4.
Rev. Ciênc. Plur ; 10 (1) 2024;10(1): 34483, 2024 abr. 30. tab
Artigo em Português | LILACS, BBO | ID: biblio-1553344

RESUMO

Objetivo:Avaliar a correlação entre a ansiedade com qualidade do sono e sonolência diurna dos estudantes universitários em curso de graduação em enfermagem, assim como caracterizar o perfil sociodemográfico dos estudantes. Metodologia:Estudodescritivo transversalcom abordagem quantitativa envolvendo 255 acadêmicos do curso de Enfermagem de uma instituição privada. A coleta dos dados ocorreu em Outubro de 2022 em salas de aula. Os instrumentos utilizados na coleta dos dados foram o Inventário de Ansiedade de Beck,Índice de Qualidade do Sono de Pittsburgh, Escala de Sonolência de Epwort-th e um questionário sociodemográfico. Utilizou-se o software estatístico R versão 4.0.2 e foram feitas suas correlações. Resultados: Foram encontrados padrões de ansiedade grave, com níveis de sonolência diurna anormal. A correlação entre o Inventário de Ansiedade de Beckcom a Escala de Sonolência de Epworth e o Índice de Qualidade de sono de Pittsburgh, que embora tenha sido considerada fraca a moderada, por sua vez mostrou-se significativa (r=0,29; p=0,01). Ademais, temos que, a relação entre oinventário de Becke o Índice de qualidade de sono de Pittsburgh mostrou-se também com valor de significância (r=0,35; p=0,01). Conclusões:Foi possível compreender que a ansiedade pode interferir na qualidade do sono e sonolência diurna do público de estudantes de Enfermagem (AU).


Objective: To evaluate the correlation between anxiety and sleep quality and daytime sleepiness among undergraduate nursing students, as well as to characterize the students' sociodemographic profile. Methodology: A cross-sectional descriptive study with a quantitative approach involving 255 nursing students from a private institution. Data collection took place in October 2022 in classrooms. The instruments used to collect the data were the Beck Anxiety Inventory, the Pittsburgh Sleep Quality Index, the Epwort-Th Sleepiness Scale and a sociodemographic questionnaire. Statistical software R version 4.0.2 was used and correlations were made. Results:Patterns of severe anxiety were found, with abnormal levels of daytime sleepiness. The correlation between the Beck Anxiety Inventory, the Epworth Sleepiness Scale and the Pittsburgh Sleep Quality Index, although considered weak to moderate, was significant (r=0.29; p=0.01). Furthermore, the relationship between the Beck Inventory and the Pittsburgh Sleep Quality Index was also found to be significant (r=0.35; p=0.01). Conclusions:It was possible to understand that anxiety can interfere with the quality of sleep and daytime sleepiness among nursing students (AU).


Objetivo:Evaluar la correlación entre la ansiedad y la calidad del sueño y la somnolencia diurna en estudiantes universitarios de enfermería, así como caracterizar el perfil sociodemográfico de los estudiantes. Metodología:Estudio descriptivo transversal con enfoque cuantitativo en el que participaron 255 estudiantes universitarios de enfermería de una institución privada. La recogida de datos tuvo lugar en octubre de 2022 en las aulas. Los instrumentos utilizados para la recogida de datos fueron el Inventario de Ansiedad de Beck, el Índice de Calidad del Sueño de Pittsburgh, la Escala de Somnolencia de Epwort-Th y un cuestionario sociodemográfico. Se utilizó el software estadístico R versión 4.0.2 y se realizaron correlaciones. Resultados:Se encontraron patrones de ansiedad severa, con niveles anormales de somnolencia diurna. La correlación entre el Inventario de Ansiedad de Beck con la Escala de Somnolencia de Epworth y el Índice de Calidad del Sueño de Pittsburgh, aunque se consideró de débil a moderada, fue significativa (r=0,29; p=0,01). Además, la relación entre el Inventario de Beck y el Índice de Calidad del Sueño de Pittsburgh también resultó significativa (r=0,35; p=0,01). Conclusiones: Se pudo comprender que la ansiedad puede interferir en la calidad del sueño y en la somnolencia diurna de los estudiantes de enfermería (AU).


Assuntos
Humanos , Masculino , Feminino , Adulto , Pessoa de Meia-Idade , Transtornos de Ansiedade/psicologia , Estudantes de Enfermagem , Qualidade do Sono , Distúrbios do Sono por Sonolência Excessiva/psicologia , Transtornos do Sono-Vigília , Estudos Transversais/métodos , Inquéritos e Questionários , Interpretação Estatística de Dados , Determinantes Sociais da Saúde , Fatores Sociodemográficos
5.
Rev. Ciênc. Plur ; 10 (1) 2024;10(1): 33471, 2024 abr. 30. tab, ilus
Artigo em Português | LILACS, BBO | ID: biblio-1553341

RESUMO

Introdução: O componente hospitalar da Rede de Atenção Psicossocial preconiza o fechamento progressivo de hospitais psiquiátricos e a implementação de leitos de saúde mental em hospital geral, capazes de fornecer atendimento para os casos agudos que necessitem de internação de forma articulada com os demais pontos de atenção da rede. Objetivo: Diante disso, o objetivo do presente artigo foi analisar a distribuição do número de leitos de atenção hospitalar em saúde mental no Rio Grande do Norte entre 2012 e 2022 e apresentar uma proposta de planejamento e avaliação para fortalecer a Rede de Atenção Psicossocial do estado. Metodologia: Trata-se de um estudo ecológico realizado no estado do Rio Grande do Norte, Brasil, no período de 2012 a 2022, usando dados secundários sobre as internações, seguido de um estudo propositivo com base em referências de planejamento e avaliação em saúde. Resultados: Observa-se uma redução do número de leitos psiquiátricos ao longo do tempo, mas que não se mostra suficiente e não se traduz em um crescimento satisfatório de leitos de saúde mental em hospital geral. Foram propostas cinco ações com o intuito de fortalecer a Rede de Atenção Psicossocial através da implantação e qualificação de leitos de saúde mental em hospitais gerais. Conclusão: Conclui-se que o movimento de constituição do componente hospitalar da Rede de Atenção Psicossocial do Rio Grande do Norte tem se apresentado em movimento irregular e o número de leitos de saúde mental em hospital geral é insuficiente. Espera-se que as intervenções e avaliações sugeridas possam contribuir para subsidiar importantes encaminhamentos no âmbito das políticas públicas de saúde mental do Rio Grande do Norte, Brasil (AU).


Introduction: The hospital component of the Psychosocial Care Network (PCN) advocates the progressive closure of psychiatric hospitals and the implementation of mental health beds in general hospitals, capable of providing care for acute cases that require hospitalization in conjunction with other network attention points. Objective: In view of this, the objective of this article was to analyze the quantitative distribution of hospital mental health care beds in Rio Grande do Norte between 2012 and 2022 and present a planning and evaluation proposal to strengthen the state's PCN. Methodology: This is an ecological study carried out in the state of Rio Grande do Norte, Brazil, from 2012 to 2022 using secondary data on hospitalizations, followed by a proactive study based on health planning and evaluation references. Results:There has been a reduction in the number of psychiatric beds over time, but not sufficient andnot translated into a satisfactory growth in mental health beds in general hospitals. Five actions were proposed with the aim of strengthening the PCN through the implementation and qualification of mental health beds in general hospitals.Conclusion: It is concluded that the movement to establish the hospital component of PCN in Rio Grande do Norte has been irregular and the number of mental health beds in general hospitals is insufficient. It is expected that the suggested interventions and evaluations may contribute to supporting important developments within the scope of public mental health policies in Rio Grande do Norte, Brazil (AU).


Introducción: El componente hospitalario de la Red de Atención Psicosocial (RAPS) aboga por el cierre progresivo de los hospitales psiquiátricos y la implementación de camas de salud mental en los hospitales generales, capaces de brindar atención a casos agudos que requieran hospitalización en conjunto con otros puntos de atención de la red. Objetivo:Ante esto, el objetivo de este artículo fue analizar la distribución del número de camas hospitalarias de atención a la salud mental en Rio Grande do Norte entre 2012 y 2022 y presentar una propuesta de planificación y evaluación para fortalecer el RAPS del Estado. Metodología:Se trata de un estudio ecológico realizado en el Estado de Rio Grande do Norte, Brasil, de 2012 a 2022, utilizando datos secundarios sobre hospitalizaciones, seguido de un estudio propositivo basado en referencias de planificación y evaluación de la salud. Resultados:Hay una reducción en el número de camas psiquiátricas a lo largo del tiempo, pero esto no es suficiente y no se traduce en un crecimiento satisfactorio de camas de salud mental en los hospitales generales. Se propusieron cinco acciones con el objetivo de fortalecer el RAPS a través de la implementación y habilitación de camas de salud mental en hospitales generales. Conclusión:Se concluye que el movimiento para establecer el componente hospitalario del RAPS en Rio Grande do Norte ha sido irregular y el número de camas de salud mental en un hospital general es insuficiente. Se espera que las intervenciones y evaluaciones sugeridas puedan contribuir a apoyar derivaciones importantes dentro del alcance de las políticas públicas de salud mental en Rio Grande do Norte, Brasil (AU).


Assuntos
Avaliação em Saúde , Saúde Mental , Planejamento em Saúde , Serviços de Saúde Mental , Atenção Terciária à Saúde , Brasil/epidemiologia , Interpretação Estatística de Dados , Estudos Ecológicos , Análise de Dados Secundários , Hospitais Psiquiátricos
6.
Stat Methods Med Res ; 33(4): 557-573, 2024 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-38426821

RESUMO

We compared methods to project absolute risk, the probability of experiencing the outcome of interest in a given projection interval accommodating competing risks, for a person from the target population with missing predictors. Without missing data, a perfectly calibrated model gives unbiased absolute risk estimates in a new target population, even if the predictor distribution differs from the training data. However, if predictors are missing in target population members, a reference dataset with complete data is needed to impute them and to estimate absolute risk, conditional only on the observed predictors. If the predictor distributions of the reference data and the target population differ, this approach yields biased estimates. We compared the bias and mean squared error of absolute risk predictions for seven methods that assume predictors are missing at random (MAR). Some methods imputed individual missing predictors, others imputed linear predictor combinations (risk scores). Simulations were based on real breast cancer predictor distributions and outcome data. We also analyzed a real breast cancer dataset. The largest bias for all methods resulted from different predictor distributions of the reference and target populations. No method was unbiased in this situation. Surprisingly, violating the MAR assumption did not induce severe biases. Most multiple imputation methods performed similarly and were less biased (but more variable) than a method that used a single expected risk score. Our work shows the importance of selecting predictor reference datasets similar to the target population to reduce bias of absolute risk predictions with missing risk factors.


Assuntos
Neoplasias da Mama , Projetos de Pesquisa , Humanos , Feminino , Fatores de Risco , Viés , Interpretação Estatística de Dados
8.
Toxicol Pathol ; 52(1): 13-20, 2024 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-38445634

RESUMO

The Tumor Combination Guide was created at the request of the U. S. Food and Drug Administration (FDA) by a Working Group of biopharmaceutical experts from international societies of toxicologic pathology, the Food and Drug Administration (FDA), and members of the Standard for Exchange of Nonclinical Data (SEND) initiative, to assist pharmacology/toxicology reviewers and biostatisticians in statistical analysis of nonclinical tumor data. The guide will also be useful to study and peer review pathologists in interpreting the tumor data. This guide provides a higher-level hierarchy of tumor types or categories correlating the tumor names from the International Harmonization of Nomenclature and Diagnostic Criteria (INHAND) publications with those available in the NEOPLASM controlled terminology (CT) code list in SEND. The version of CT used in a study should be referenced in the nonclinical study data reviewer's guide (SDRG) (section 3.1) of electronic submissions to the FDA. The tumor combination guide instructions and examples are in a tabular format to make informed decisions for combining tumor data for statistical analysis. The strategy for combining tumor types for statistical analysis is based on scientific criteria gleaned from the current scientific literature; as SEND and INHAND terminology and information evolve, this guide will be updated.


Assuntos
Testes de Carcinogenicidade , Animais , Testes de Carcinogenicidade/métodos , Testes de Carcinogenicidade/normas , Neoplasias/induzido quimicamente , Neoplasias/patologia , Estados Unidos , Ratos , United States Food and Drug Administration , Roedores , Camundongos , Guias como Assunto , Interpretação Estatística de Dados
10.
Ann Surg ; 279(6): 907-912, 2024 Jun 01.
Artigo em Inglês | MEDLINE | ID: mdl-38390761

RESUMO

OBJECTIVE: To determine the prevalence of clinical significance reporting in contemporary comparative effectiveness research (CER). BACKGROUND: In CER, a statistically significant difference between study groups may or may not be clinically significant. Misinterpreting statistically significant results could lead to inappropriate recommendations that increase health care costs and treatment toxicity. METHODS: CER studies from 2022 issues of the Annals of Surgery , Journal of the American Medical Association , Journal of Clinical Oncology , Journal of Surgical Research , and Journal of the American College of Surgeons were systematically reviewed by 2 different investigators. The primary outcome of interest was whether the authors specified what they considered to be a clinically significant difference in the "Methods." RESULTS: Of 307 reviewed studies, 162 were clinical trials and 145 were observational studies. Authors specified what they considered to be a clinically significant difference in 26 studies (8.5%). Clinical significance was defined using clinically validated standards in 25 studies and subjectively in 1 study. Seven studies (2.3%) recommended a change in clinical decision-making, all with primary outcomes achieving statistical significance. Five (71.4%) of these studies did not have clinical significance defined in their methods. In randomized controlled trials with statistically significant results, sample size was inversely correlated with effect size ( r = -0.30, P = 0.038). CONCLUSIONS: In contemporary CER, most authors do not specify what they consider to be a clinically significant difference in study outcome. Most studies recommending a change in clinical decision-making did so based on statistical significance alone, and clinical significance was usually defined with clinically validated standards.


Assuntos
Pesquisa Comparativa da Efetividade , Humanos , Interpretação Estatística de Dados , Projetos de Pesquisa , Ensaios Clínicos como Assunto
11.
Int. j. morphol ; 42(1): 185-196, feb. 2024. ilus, tab, graf
Artigo em Inglês | LILACS | ID: biblio-1528838

RESUMO

SUMMARY: The new paradigm in Forensic Sciences initiated by the entry of genetics (the current standard of legal evidence) and accentuated by recognized wrongful convictions derived from experts today in the eye of criticism, has highlighted the potential for bias and error in forensic disciplines when they depend on human interpretation and subjectivity, which has not been avoided by Forensic Odontology (FO). However, a subjective judgment is not necessarily wrong, so the refinement of processes, the development of standards, and robust research can contribute to the validity of interpretation to increase objectivity. Latin America (LATAM) has its own realities and needs, which have conditioned the priorities and objectives of FO research. A scoping review is presented to systematically map the investigation of LATAM researchers and identify the objective or subjective nature of their assessments. LATAM shows interesting productivity and intentions to adhere to international standards, with Brazil leading this research significantly, followed by Chile and Colombia, among others. However, there is a disproportionate approach in certain lines of research (dental age estimation), and needs to address other quantitative studies, and to improve the visibility of the LATAM FO research.


El nuevo paradigma en ciencias forenses iniciado por la entrada de la genética (el actual estándar de la evidencia jurídica), y acentuado por reconocidas condenas injustas derivadas de pericias hoy en el ojo de la crítica, ha destacado el potencial de sesgo y error que poseen algunas disciplinas forenses cuando dependen de la interpretación humana y la subjetividad, lo cual no ha sido ajeno a la odontología forense (OF). Sin embargo, un juicio subjetivo no necesariamente es erróneo, con lo que el refinamiento de procesos, el desarrollo de estándares y la investigación robusta pueden contribuir a validar esa interpretación para aumentar su objetividad. Latinoamérica (LATAM) posee realidades y necesidades propias que han condicionado las prioridades y objetivos de la investigación en OF. Se presenta una revisión con búsqueda sistemática para mapear sistemáticamente la investigación en OF realizada por investigadores latinoamericanos, así como identificar la naturaleza objetiva o subjetiva de sus evaluaciones. LATAM demuestra una productividad interesante e intenciones de adherirse a estándares internacionales, con Brasil liderando significativamente esta investigación, seguido por Chile y Colombia entre otros. Sin embargo, se observa un enfoque desproporcionado en ciertas líneas de investigación (estimación de edad dental particularmente), y necesidad tanto de abordar otros estudios cuantitativos como de mejorar la visibilidad de la investigación latinoamericana en OF.


Assuntos
Humanos , Pesquisa , Odontologia Legal , Interpretação Estatística de Dados , Pesquisa Qualitativa , América Latina
12.
Int J Mol Sci ; 25(2)2024 Jan 06.
Artigo em Inglês | MEDLINE | ID: mdl-38255820

RESUMO

The dopamine D4 receptor (D4R) is a promising therapeutic target in widespread diseases, and the search for novel agonists and antagonists appears to be clinically relevant. The mechanism of binding to the receptor (R) for antagonists and agonists varies. In the present study, we conducted an in-depth computational study, teasing out key similarities and differences in binding modes, complex dynamics, and binding energies for D4R agonists and antagonists. The dynamic network method was applied to investigate the communication paths between the ligand (L) and G-protein binding site (GBS) of human D4R. Finally, the fragment molecular orbitals with pair interaction energy decomposition analysis (FMO/PIEDA) scheme was used to estimate the binding energies of L-R complexes. We found that a strong salt bridge with D3.32 initiates the inhibition of the dopamine D4 receptor. This interaction also occurs in the binding of agonists, but the change in the receptor conformation to the active state starts with interaction with cysteine C3.36. Such a mechanism may arise in the case of agonists unable to form a hydrogen bond with the serine S5.46, considered, so far, to be crucial in the activation of GPCRs. The energy calculations using the FMO/PIEDA method indicate that antagonists show higher residue occupancy of the receptor binding site than agonists, suggesting they could form relatively more stable complexes. Additionally, antagonists were characterized by repulsive interactions with S5.46 distinguishing them from agonists.


Assuntos
Comunicação , Receptores de Dopamina D4 , Humanos , Sítios de Ligação , Cisteína , Interpretação Estatística de Dados
13.
Stat Methods Med Res ; 33(2): 344-356, 2024 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-38262434

RESUMO

Identifying and distinguishing risk factors for heterogeneous disease subtypes has been of great interest. However, missingness in disease subtypes is a common problem in those data analyses. Several methods have been proposed to deal with the missing data, including complete-case analysis, inverse-probability weighting, and multiple imputation. Although extant literature has compared these methods in missing problems, none has focused on the competing risk setting. In this paper, we discuss the assumptions required when complete-case analysis, inverse-probability weighting, and multiple imputation are used to deal with the missing failure subtype problem, focusing on how to implement these methods under various realistic scenarios in competing risk settings. Besides, we compare these three methods regarding their biases, efficiency, and robustness to model misspecifications using simulation studies. Our results show that complete-case analysis can be seriously biased when the missing completely at random assumption does not hold. Inverse-probability weighting and multiple imputation estimators are valid when we correctly specify the corresponding models for missingness and for imputation, and multiple imputation typically shows higher efficiency than inverse-probability weighting. However, in real-world studies, building imputation models for the missing subtypes can be more challenging than building missingness models. In that case, inverse-probability weighting could be preferred for its easy usage. We also propose two automated model selection procedures and demonstrate their usage in a study of the association between smoking and colorectal cancer subtypes in the Nurses' Health Study and Health Professional Follow-Up Study.


Assuntos
Modelos Estatísticos , Humanos , Modelos de Riscos Proporcionais , Seguimentos , Interpretação Estatística de Dados , Probabilidade , Simulação por Computador , Fatores de Risco
15.
Lifetime Data Anal ; 30(1): 34-58, 2024 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-36821062

RESUMO

Survival causal effect estimation based on right-censored data is of key interest in both survival analysis and causal inference. Propensity score weighting is one of the most popular methods in the literature. However, since it involves the inverse of propensity score estimates, its practical performance may be very unstable, especially when the covariate overlap is limited between treatment and control groups. To address this problem, a covariate balancing method is developed in this paper to estimate the counterfactual survival function. The proposed method is nonparametric and balances covariates in a reproducing kernel Hilbert space (RKHS) via weights that are counterparts of inverse propensity scores. The uniform rate of convergence for the proposed estimator is shown to be the same as that for the classical Kaplan-Meier estimator. The appealing practical performance of the proposed method is demonstrated by a simulation study as well as two real data applications to study the causal effect of smoking on survival time of stroke patients and that of endotoxin on survival time for female patients with lung cancer respectively.


Assuntos
Modelos Estatísticos , Fumar , Humanos , Feminino , Interpretação Estatística de Dados , Simulação por Computador , Pontuação de Propensão
16.
Pharm Stat ; 23(1): 91-106, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-37786317

RESUMO

Duration of response (DOR) and time to response (TTR) are typically evaluated as secondary endpoints in early-stage clinical studies in oncology when efficacy is assessed by the best overall response and presented as the overall response rate. Despite common use of DOR and TTR in particular in single-arm studies, the definition of these endpoints and the questions they are intended to answer remain unclear. Motivated by the estimand framework, we present relevant scientific questions of interest for DOR and TTR and propose corresponding estimand definitions. We elaborate on how to deal with relevant intercurrent events which should follow the same considerations as implemented for the primary response estimand. A case study in mantle cell lymphoma illustrates the implementation of relevant estimands of DOR and TTR. We close the paper with practical recommendations to implement DOR and TTR in clinical study protocols.


Assuntos
Neoplasias , Projetos de Pesquisa , Adulto , Humanos , Interpretação Estatística de Dados , Oncologia , Ensaios Clínicos como Assunto
18.
Pharm Stat ; 23(3): 385-398, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-38124266

RESUMO

Multiregional clinical trials (MRCTs) have become increasingly common during the development of new drugs to obtain simultaneous drug approvals worldwide. When planning MRCTs, a major statistical challenge is determination of the regional sample size. In general, the regional sample size must be determined as the sample size such that the regional consistency probability, defined as the probability of meeting the regional consistency criterion, is greater than a prespecified value. The Japanese Ministry of Health, Labour and Welfare proposed two criteria for regional consistency. Moreover, many researchers have proposed corresponding closed-form formulas for calculating regional consistency probabilities when the primary outcome is continuous. Although some researchers have argued that those formulas are also applicable to cases with binary outcomes, it remains questionable whether such an argument can be true. Based on simulation results, we demonstrate that the existing formulas are inappropriate for binary cases, even when the regional sample size is sufficiently large. To address this issue, we develop alternative formulas and use simulation to show that they provide accurate regional consistency probabilities. Furthermore, we present an application of our proposed formulas for an MRCT of advanced or metastatic clear-cell renal cell carcinoma.


Assuntos
Simulação por Computador , Humanos , Tamanho da Amostra , Estudos Multicêntricos como Assunto/métodos , Probabilidade , Modelos Estatísticos , Projetos de Pesquisa/estatística & dados numéricos , Ensaios Clínicos como Assunto/métodos , Ensaios Clínicos como Assunto/estatística & dados numéricos , Neoplasias Renais/tratamento farmacológico , Carcinoma de Células Renais/tratamento farmacológico , Aprovação de Drogas/métodos , Interpretação Estatística de Dados , Japão
19.
Rev. saúde pública (Online) ; 58: 01, 2024. graf
Artigo em Inglês | LILACS | ID: biblio-1536768

RESUMO

ABSTRACT OBJECTIVE This study aims to propose a comprehensive alternative to the Bland-Altman plot method, addressing its limitations and providing a statistical framework for evaluating the equivalences of measurement techniques. This involves introducing an innovative three-step approach for assessing accuracy, precision, and agreement between techniques, which enhances objectivity in equivalence assessment. Additionally, the development of an R package that is easy to use enables researchers to efficiently analyze and interpret technique equivalences. METHODS Inferential statistics support for equivalence between measurement techniques was proposed in three nested tests. These were based on structural regressions with the goal to assess the equivalence of structural means (accuracy), the equivalence of structural variances (precision), and concordance with the structural bisector line (agreement in measurements obtained from the same subject), using analytical methods and robust approach by bootstrapping. To promote better understanding, graphical outputs following Bland and Altman's principles were also implemented. RESULTS The performance of this method was shown and confronted by five data sets from previously published articles that used Bland and Altman's method. One case demonstrated strict equivalence, three cases showed partial equivalence, and one showed poor equivalence. The developed R package containing open codes and data are available for free and with installation instructions at Harvard Dataverse at https://doi.org/10.7910/DVN/AGJPZH. CONCLUSION Although easy to communicate, the widely cited and applied Bland and Altman plot method is often misinterpreted, since it lacks suitable inferential statistical support. Common alternatives, such as Pearson's correlation or ordinal least-square linear regression, also fail to locate the weakness of each measurement technique. It may be possible to test whether two techniques have full equivalence by preserving graphical communication, in accordance with Bland and Altman's principles, but also adding robust and suitable inferential statistics. Decomposing equivalence into three features (accuracy, precision, and agreement) helps to locate the sources of the problem when fixing a new technique.


Assuntos
Intervalos de Confiança , Análise de Regressão , Interpretação Estatística de Dados , Inferência Estatística , Confiabilidade dos Dados
20.
Pesqui. bras. odontopediatria clín. integr ; 24: e220153, 2024. tab, graf
Artigo em Inglês | LILACS, BBO | ID: biblio-1529138

RESUMO

Abstract Objective: To analyze the existing state legislation, including not only the ordinary laws, but also their infralegal regulation and the state legislation on Hospital Dentistry. Material and Methods: A survey was carried out in the databases of the Legislative Assemblies of the Brazilian States and the Federal District, as well as the Regional and Federal Councils of Dentistry in Brazil. Subsequently, a survey was carried out in the databases of the Ministry of Health, State Dental Councils and Federal Dental Council in Brazil. Results: Only 8 Brazilian states have legislation in force regarding hospital dentistry, which represents 29.63% of the federative units. Among the Brazilian regions, the Midwest presented the highest prevalence of the laws found (37.50%), followed by the North (25%) and the other regions with the same coverage (12.50%). Also, an orientation and an ordinance from the Ministry of Health, six resolutions from the Federal Council of Dentistry, and a technical note from the National Health Surveillance Agency were found. Conclusion: Several States do not have rules on the subject, making it imperative to create a federal rule that not only imposes the presence of the dentist, but also regulates the proportion of the team, workload, and availability.


Assuntos
Saúde Bucal/legislação & jurisprudência , Unidade Hospitalar de Odontologia/legislação & jurisprudência , Equipe Hospitalar de Odontologia/legislação & jurisprudência , Unidades de Terapia Intensiva/legislação & jurisprudência , Inquéritos e Questionários , Interpretação Estatística de Dados , Carga de Trabalho , Odontólogos , Legislação Odontológica
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