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INTRODUCTION: Ideally, evaluating NGS performance requires a gold standard; in its absence, concordance between replicates is often used as substitute standard. However, the appropriateness of the concordance-discordance criterion has been rarely evaluated. This study analyzes the relationship between the probability of discordance and the probability of error under different conditions. METHODS: This study used a conditional probability approach under conditional dependence then conditional independence between two sequencing results and compares the probabilities of discordance and error in different theoretical conditions of sensitivity, specificity, and correlation between replicates, then on real results of sequencing genome NA12878. The study examines also covariate effects on discordance and error using generalized additive models with smooth functions. RESULTS: With 99% sensitivity and 99.9% specificity under conditional independence, the probability of error for a positive concordant pair of calls is 0.1%. With additional hypotheses of 0.1% prevalence and 0.9 correlation between replicates, the probability of error for a positive concordant pair is 47.4%. With real data, the estimated sensitivity, specificity, and correlation between tests for variants are around 98.98%, 99.996%, and 93%, respectively, and the error rate for positive concordant calls approximates 2.5%. In covariate effect analyses, the effects' functional form are close between discordance and error models, though the parts of deviance explained by the covariates differ between discordance and error models. CONCLUSION: With conditional independence of two sequencing results, the concordance-discordance criterion seems acceptable as substitute standard. However, with high correlation, the criterion becomes questionable because a high percentage of false concordant results appears among concordant results.
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BACKGROUND: Togo's National Malaria Control Programme has initiated an active home-based malaria management model for all age groups in rural areas of Bassar Health District. This report describes the model, reports its main results, and determines the factors associated with positive rapid diagnostic test results. METHODS: From 2014 to 2017, in three peripheral care units of Bassar Health District (Binaparba, Nangbani, and Baghan), community health workers visited residents' homes weekly to identify patients with malaria symptoms, perform rapid diagnostic tests in symptomatic patients, and give medication to positive cases. Univariate and multivariate logistic regression models were used to determine the factors associated with positive tests. RESULTS: The study covered 11,337 people (817 in 2014, 1804 in 2015, 2638 in 2016, and 6078 in 2017). The overall mean age was 18 years (95% CI 5-29; min-max: 0-112 years). The median age was 10 years (SD: 16.9). The proportions of people tested positive were 75.3% in Binaparba, 77.4% in Nangbani, and 56.6% in Baghan. The 5-10 age group was the most affected category (24.2% positive tests). Positive tests were more frequent during the rainy than during the dry season (62 vs. 38%) and the probability of positive test was 1.76 times higher during the rainy than during the dry season (adjusted OR = 1.74; 95% CI 1.60-1.90). A fever (37.5 °C or higher) increased significantly the probability of positive test (adjusted OR = 2.19; 95% CI 1.89-2.54). The risk of positive test was 1.89 times higher in passive than in active malaria detection (adjusted OR = 1.89; 95% CI 1.73-2.0). CONCLUSIONS: This novel experimental community and home-based malaria management in Togo suggested that active detection of malaria cases is feasible within 24 h, which allows rapid treatments before progression to often-fatal complications. This PECADOM + program will help Togo's National Malaria Control Programme reduce malaria morbidity and mortality in remote and hard-to-reach communities.
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Malária , População Rural , Humanos , Togo/epidemiologia , Adolescente , Criança , Adulto , População Rural/estatística & dados numéricos , Pré-Escolar , Adulto Jovem , Projetos Piloto , Masculino , Feminino , Pessoa de Meia-Idade , Idoso , Lactente , Malária/prevenção & controle , Malária/diagnóstico , Recém-Nascido , Idoso de 80 Anos ou mais , Testes Diagnósticos de Rotina/estatística & dados numéricosRESUMO
BACKGROUND: In 2012, the World Health Organization (WHO) recommended seasonal malaria chemoprevention (SMC) in areas of high seasonal transmission. Though implemented since 2013, the effectiveness of SMC in Togo was never evaluated. METHODS: This study concerned routine data from 2013 to 2020 mass SMC campaigns for children under five in all health facilities of three Regions of Togo. Treatment coverage, reasons for non-treatment, and SMC-attributable adverse reactions were analysed per year and treatment round. Random effect logistic models estimated SMC effectiveness per health district, year, and treatment round. RESULTS: The overall coverage was 98% (7,971,877 doses for 8,129,668 children). Contraindication was the main reason for non-administration. Over the study period, confirmed malaria cases decreased from 11,269 (1st round of 2016) to 1395 (4th round of 2020). Only 2,398 adverse reactions were reported (prevalence: 3/10,000), but no severe Lyell syndrome or Stevens-Johnson-type skin reaction. Compared to 2016, malaria prevalence decrease was estimated at 22.6% in 2017 (p < 0.001) and 75% in 2020 (p < 0.001). SMC effectiveness ranged from 76.6% (2nd round) to 96.2% (4th round) comparison with the 1st round. CONCLUSIONS: SMC reduced significantly malaria cases among children under five. The results reassure all actors and call for effort intensification to reach the WHO goals for 2030.
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Antimaláricos , Malária , Criança , Humanos , Lactente , Antimaláricos/uso terapêutico , Estações do Ano , Togo/epidemiologia , Estudos Longitudinais , Malária/epidemiologia , Malária/prevenção & controle , Malária/tratamento farmacológico , Quimioprevenção/métodosRESUMO
In epidemiology, left-truncated data may bias exposure effect estimates. We analyzed the bias induced by left truncation in estimating breast cancer risk associated with exposure to airborne dioxins. Simulations were run with exposure estimates from a Geographic Information System (GIS)-based metric and considered two hypotheses for historical exposure, three scenarios for intra-individual correlation of annual exposures, and three exposure-effect models. For each correlation/model combination, 500 nested matched case-control studies were simulated and data fitted using a conditional logistic regression model. Bias magnitude was assessed by estimated odds-ratios (ORs) versus theoretical relative risks (TRRs) comparisons. With strong intra-individual correlation and continuous exposure, left truncation overestimated the Beta parameter associated with cumulative dioxin exposure. Versus a theoretical Beta of 4.17, the estimated mean Beta (5%; 95%) was 73.2 (67.7; 78.8) with left-truncated exposure and 4.37 (4.05; 4.66) with lifetime exposure. With exposure categorized in quintiles, the TRR was 2.0, the estimated ORQ5 vs. Q1 2.19 (2.04; 2.33) with truncated exposure versus 2.17 (2.02; 2.32) with lifetime exposure. However, the difference in exposure between Q5 and Q1 was 18× smaller with truncated data, indicating an important overestimation of the dose effect. No intra-individual correlation resulted in effect dilution and statistical power loss. Left truncation induced substantial bias in estimating breast cancer risk associated with exposure with continuous and categorical models. With strong intra-individual exposure correlation, both models detected associations, but categorical models provided better estimates of effect trends. This calls for careful consideration of left truncation-induced bias in interpreting environmental epidemiological data.
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Neoplasias da Mama , Dioxinas , Neoplasias da Mama/induzido quimicamente , Neoplasias da Mama/epidemiologia , Estudos de Casos e Controles , Dioxinas/toxicidade , Feminino , Humanos , Razão de Chances , RiscoRESUMO
OBJECTIVE: To assess the interrater reliability of the SOFMER Activity Score (SAS) (version 2 [v2], an 8-item [4 motor and 4 cognitive] and 5-level scale) and improve its scoring system before conducting further validation steps. DESIGN: Cross-sectional, prospective, observational, noninterventional, and multicentric study. SETTING: The study was conducted between November 2018 and September 2019 in 4 French rehabilitation centers (2 public university hospitals for adults and 2 private not-for-profit rehabilitation centers for children). PARTICIPANTS: The study included 101 participants (N=101; mean age, 44.5±25.4 years; 28.7% younger than 18 and 18.8% older than 65 years). The female/male sex ratio was 0.6. The causes for admission to the center were mainly neurologic (65%) or orthopedic (24%). INTERVENTIONS: None. MAIN OUTCOME MEASURES: Activity limitation was rated with the SAS the same day by 2 independent multidisciplinary teams. The interrater reliabilities of the score items were assessed using weighted kappa coefficients. RESULTS: All weighted kappa coefficients ranged between 0.83 and 0.92, indicating "good" to "excellent" interrater reliability. Interteam score disagreements occurred in 227 of 808 scores (28%). The reason for most disagreements was unnoticed human or material aid during the observation period. CONCLUSIONS: The results demonstrate the high interrater reliability of the SASv2 and allow carrying out further validation steps after minor changes to item scoring instructions and clearer definitions of some items that help improving scoring standardization. The SASv2 may then become a consistent measure of activity level for clinical research or burden of care investigations.
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Avaliação da Deficiência , Centros de Reabilitação , Adulto , Idoso , Criança , Estudos Transversais , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Variações Dependentes do Observador , Estudos Prospectivos , Reprodutibilidade dos Testes , Adulto JovemRESUMO
Background and Objectives: To test the long-term ability of human ovarian cortex cells to develop in unconventional culture conditions. Materials and Methods. Ovarian cortex cells from fetuses aged 23 to 39 weeks gestation were cultured for 90 days in hollow chitosan hydrogel micro-bioreactors and concurrently in traditional wells. Various cell-type counts were considered. Results: With intact follicles as a denominator, the percentage of growing intact follicles at Day 0 varied widely between ovaries (0 to 31.7%). This percentage tended to increase or stay relatively constant in bioreactor as in control cultures; it tended more toward an increase over time in bioreactor vs. control cultures. Modeled percentages showed differences (though not significant) in favor of bioreactor cultures (16.12% difference at D50 but only 0.12% difference at D90). With all follicles present as a denominator, the percentage of growing primary and secondary follicles at D0 varied widely between ovaries (0 to 29.3%). This percentage tended to increase over time in bioreactor cultures but to decrease in control cultures. Modeled percentages showed significant differences in favor of bioreactor cultures (8.9% difference at D50 and 11.1% difference at D90). At D50 and D90, there were only few and sparse apoptotic cells in bioreactor cultures vs. no apoptotic cells in control cultures. Conclusions: Over three months, bioreactor folliculogenesis outperformed slightly traditional culture. This is an interesting perspective for follicle preservation and long-term toxicological studies.
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Quitosana , Ovário , Feminino , Humanos , Hidrogéis , Técnicas de Cultura de Tecidos/métodos , Reatores BiológicosRESUMO
OBJECTIVES: The one-compartment iohexol plasma clearance has been proposed as a reliable alternative to renal inulin clearance. However, this method's performance depends on the formula used to calculate glomerular filtration rate (GFR). This study reports on performance comparisons between various mathematical formulas proposed for iohexol plasma clearance vs. inulin urinary clearance. METHODS: GFR was simultaneously determined by inulin and iohexol clearance in 144 participants (age: 10-84 years; glomerular filtration rate: 15-169 mL/min/1.73 m2). A retrospective cross-sectional study evaluated the performance of four formulas proposed to calculate plasma iohexol clearance (Brøchner-Mortensen, Fleming et al., Jødal-Brøchner-Mortensen, and Ng-Schwartz-Munoz). The performance of each formula was assessed using bias, precision (standard deviation of the bias), accuracy (percentage iohexol within 5, 10, and 15%), root mean square error, and concordance correlation coefficient vs. renal inulin clearance as reference. RESULTS: Regarding accuracy, there was no difference in root mean square error (RMSE), P5, P10, or P15 between the four formulas. The four concordance correlation coefficients (CCC) between the value from each formula and in-GFR were high and not significantly different. At in-GFR ≥90 mL/min/1.73 m2, Ng-Schwartz-Munoz formula performed slightly better than other formulas regarding median bias (-0.5; 95% CI [-3.0 to 2.0] and accuracy P15 (95.0; 95% CI [88.0-100.0]). CONCLUSIONS: The studied formulas were found equivalent in terms of precision and accuracy, but the Ng-Schwartz-Munoz formula improved the accuracy at higher levels of in-GFR.
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Taxa de Filtração Glomerular , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Criança , Creatinina , Humanos , Inulina , Iohexol , Pessoa de Meia-Idade , Estudos Retrospectivos , Adulto JovemRESUMO
Objective and Background: Men's experiences of first-fatherhood discovery differ widely with various circumstances; they were examined according to the intended/unintended occurrence of partner's pregnancy.Methods: An Associative Network study targeted 44 first-time fathers and analysed their reactions to a stimulus-sentence 'In the first days after pregnancy announcement 'Results: Word-associations formed 19 themes and 5 metathemes. The main metathemes were Emotional (30%), Cognitive (27%) and Relational (23%). Negative emotions were more frequent in unintended vs. intended pregnancies. Subthemes Chock, Feeling of unreality, Emotion control, Guilt, Denial, or Regret prevailed in unintended pregnancies. The major theme in all pregnancies was Personal advancement but subthemes Physical and psychological preparation and Responsibility prevailed in intended pregnancies vs. Acceptation and Personal progress in unintended pregnancies. Themes Relationships (with partner and others) were more frequently mentioned in unintended pregnancies vs. theme Logistics in intended pregnancies. Overall, the participants expressed opposed conflicting emotions but a desire for real involvement in pregnancy, birth, and child-raising.Conclusion: The participants proved interested and concerned. Fathers's presence and support should be solicited, accounted for, and valued by mothers-to-be and health professionals in all pregnancies. Involving fathers since pregnancy is essential for mental equilibrium, child-parent attachment, child and parent development, and family relationships.
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Pai , Homens , Emoções , Feminino , Humanos , Masculino , Mães , Parto , GravidezRESUMO
There is a growing interest in using health care (HC) data to produce epidemiological surveillance indicators such as incidence. Typically, in the field of cancer, incidence is provided by local cancer registries which, in many countries, do not cover the whole territory; using proxy measures from available nationwide HC databases would appear to be a suitable approach to fill this gap. However, in most cases, direct counts from these databases do not provide reliable measures of incidence. To obtain accurate incidence estimations and prediction intervals, these databases need to be calibrated using a registry-based gold standard measure of incidence. This article presents a calibration model for count data developed to predict cancer incidence from HC data in geographical areas without cancer registries. First, the ratio between the proxy measure and incidence is modeled in areas with registries using a Poisson mixed model that allows for heterogeneity between areas (calibration stage). This ratio is then inverted to predict incidence from the proxy measure in areas without registries. Prediction error admits closed-form expression which accounts for heterogeneity in the ratio between areas. A simulation study shows the accuracy of our method in terms of prediction and coverage probability. The method is further applied to predict the incidence of two cancers in France using hospital data as the proxy measure. We hope this approach will encourage sound use of the usually imperfect information extracted from HC data.
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Monitoramento Epidemiológico , Modelos Biológicos , Modelos Estatísticos , Neoplasias/epidemiologia , Calibragem , Simulação por Computador , Hospitais/estatística & dados numéricos , Humanos , IncidênciaRESUMO
OBJECTIVE: To examine whether a Rasch analysis is sufficient to establish the construct validity of the Motor Function Measure (MFM) and discuss whether weighting the MFM item scores would improve the MFM construct validity. DESIGN: Observational cross-sectional multicenter study. SETTING: Twenty-three physical medicine departments, neurology departments, or reference centers for neuromuscular diseases. PARTICIPANTS: Patients (N=911) aged 6 to 60 years with Charcot-Marie-Tooth disease (CMT), facioscapulohumeral dystrophy (FSHD), or myotonic dystrophy type 1 (DM1). INTERVENTIONS: None. MAIN OUTCOME MEASURE(S): Comparison of the goodness-of-fit of the confirmatory factor analysis (CFA) model vs that of a modified multidimensional Rasch model on MFM item scores in each considered disease. RESULTS: The CFA model showed good fit to the data and significantly better goodness of fit than the modified multidimensional Rasch model regardless of the disease (P<.001). Statistically significant differences in item standardized factor loadings were found between DM1, CMT, and FSHD in only 6 of 32 items (items 6, 27, 2, 7, 9 and 17). CONCLUSIONS: For multidimensional scales designed to measure patient abilities in various diseases, a Rasch analysis might not be the most convenient, whereas a CFA is able to establish the scale construct validity and provide weights to adapt the item scores to a specific disease.
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Doença de Charcot-Marie-Tooth/diagnóstico , Avaliação da Deficiência , Distrofia Muscular Facioescapuloumeral/diagnóstico , Distrofia Miotônica/diagnóstico , Inquéritos e Questionários/normas , Adolescente , Adulto , Doença de Charcot-Marie-Tooth/fisiopatologia , Criança , Estudos Transversais , Análise Fatorial , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Destreza Motora , Distrofia Muscular Facioescapuloumeral/fisiopatologia , Distrofia Miotônica/fisiopatologia , Psicometria , Reprodutibilidade dos Testes , Adulto JovemRESUMO
BACKGROUND: The survival rates of renal transplant children are indeed on the rise, but it is still important to ensure that there is optimal renal function in these children in all their future growing years. The number of functioning nephrons and the graft ability to adapt to an increasing demand during body growth seem to be the most important factors for long-term allograft function. This study examined the long-term change in the glomerular filtration rate in a pediatric kidney transplant cohort and the importance of the recipient and donor ages in predicting transplant outcome. METHODS: Data on 67 renal transplant children who underwent 278 inulin-clearance measurements between 2000 and 2010 were examined. A longitudinal latent class model was used to identify renal function trajectories and classify the children. RESULTS: This model identified 3 trajectories of renal allograft function after pediatric kidney transplantation: 'low and decreasing', 'moderate and stable', and 'high and sharply decreasing'. The probability of belonging to the low and decreasing trajectory - that is, the poorer outcome - was lower in recipients of grafts from living versus deceased donor (adjusted OR (aOR) 0.02; p = 0.03). This probability increased with recipient age (aOR 1.20 per year of recipient ageing; p = 0.07) and donor-recipient age-difference (aOR 1.13 per additional year; p = 0.07). CONCLUSION: This study suggests that donation from living donors and from younger donors are favorable factors for long-term allograft function.
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Taxa de Filtração Glomerular , Falência Renal Crônica/cirurgia , Transplante de Rim , Complicações Pós-Operatórias/metabolismo , Insuficiência Renal/metabolismo , Anormalidades Urogenitais/cirurgia , Adolescente , Fatores Etários , Criança , Pré-Escolar , Progressão da Doença , Feminino , França/epidemiologia , Rejeição de Enxerto/prevenção & controle , Humanos , Imunossupressores/uso terapêutico , Lactente , Recém-Nascido , Testes de Função Renal , Doadores Vivos , Masculino , Complicações Pós-Operatórias/epidemiologia , Insuficiência Renal/epidemiologia , Fatores de Risco , Doadores de TecidosRESUMO
BACKGROUND: Estimating kidney glomerular filtration rate (GFR) is of utmost importance in many clinical conditions. However, very few studies have evaluated the performance of GFR estimating equations over all ages and degrees of kidney impairment. We evaluated the reliability of two major equations for GFR estimation, the CKD-EPI and Schwartz equations, with urinary clearance of inulin as gold standard. METHODS AND FINDINGS: The study included 10,610 participants referred to the Renal and Metabolic Function Exploration Unit of Edouard Herriot Hospital (Lyon, France). GFR was measured by urinary inulin clearance (only first measurement kept for analysis) then estimated with isotope dilution mass spectrometry (IDMS)-traceable CKD-EPI and Schwartz equations. The participants' ages ranged from 3 to 90 y, and the measured GFRs from 3 to 160 ml/min/1.73 m2. A linear mixed-effects model was used to model the bias (mean ratio of estimated GFR to measured GFR). Equation reliability was also assessed using precision (interquartile range [IQR] of the ratio) and accuracy (percentage of estimated GFRs within the 10% [P10] and 30% [P30] limits above and below the measured GFR). In the whole sample, the mean ratio with the CKD-EPI equation was significantly higher than that with the Schwartz equation (1.17 [95% CI 1.16; 1.18] versus 1.08 [95% CI 1.07; 1.09], p < 0.001, t-test). At GFR values of 60-89 ml/min/1.73 m2, the mean ratios with the Schwartz equation were closer to 1 than the mean ratios with the CKD-EPI equation whatever the age class (1.02 [95% CI 1.01; 1.03] versus 1.15 [95% CI 1.13; 1.16], p < 0.001, t-test). In young adults (18-40 y), the Schwartz equation had a better precision and was also more accurate than the CKD-EPI equation at GFR values under 60 ml/min/1.73 m2 (IQR: 0.32 [95% CI 0.28; 0.33] versus 0.40 [95% CI 0.36; 0.44]; P30: 81.4 [95% CI 78.1; 84.7] versus 63.8 [95% CI 59.7; 68.0]) and also at GFR values of 60-89 ml/min/1.73 m2. In all patients aged ≥65 y, the CKD-EPI equation performed better than the Schwartz equation (IQR: 0.33 [95% CI 0.31; 0.34] versus 0.40 [95% CI 0.38; 0.41]; P30: 77.6 [95% CI 75.7; 79.5] versus 67.5 [95% CI 65.4; 69.7], respectively). In children and adolescents (2-17 y), the Schwartz equation was superior to the CKD-EPI equation (IQR: 0.23 [95% CI 0.21; 0.24] versus 0.33 [95% CI 0.31; 0.34]; P30: 88.6 [95% CI 86.7; 90.4] versus 29.4 [95% CI 26.8; 32.0]). This study is limited by its retrospective design, single-center setting with few non-white patients, and small number of patients with severe chronic kidney disease. CONCLUSIONS: The results from this study suggest that the Schwartz equation may be more reliable than the CKD-EPI equation for estimating GFR in children and adolescents and in adults with mild to moderate kidney impairment up to age 40 y.
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Taxa de Filtração Glomerular , Rim/metabolismo , Insuficiência Renal Crônica/diagnóstico , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Criança , Pré-Escolar , Creatinina/metabolismo , Estudos Transversais , Feminino , Frutanos/metabolismo , Humanos , Inulina/urina , Masculino , Espectrometria de Massas , Pessoa de Meia-Idade , Modelos Teóricos , Insuficiência Renal Crônica/metabolismo , Reprodutibilidade dos Testes , Estudos Retrospectivos , Adulto JovemRESUMO
OBJECTIVE: Attention-deficit hyperactivity disorder is one of the most frequent neurodevelopmental disorders. Its diagnosis requires reference questionnaires such as the Conners' Parent Rating Scale (CPRS). Presently, in French-speaking countries, a few translations of the revised short CPRS have been put to use without previous formal validation. We sought here for the validation of a French version (Lausanne, Switzerland) of the revised short CPRS regarding construct validity, internal consistency, and item reliability in a sample of French schoolchildren. METHOD: The study involved 795 children and adolescents aged 9 to 19 years from a single school. The factorial structure and item reliability were assessed with a confirmatory factor analysis for ordered categorical variables. The dimension internal consistency was assessed with Guttman's lambda 6 coefficient. RESULTS: The results confirmed the original and strong 3-dimensional factorial structure (Oppositional, Cognitive Problems/Inattention, and Hyperactivity), showed satisfactory item reliability, and indicated a good dimension internal consistency (Guttman's lambda 6 coefficient: 0.87, 0.90, and 0.82, respectively, to the 3 dimensions). CONCLUSIONS: Thus, the Lausanne French version of the revised short CPRS may be considered validated regarding construct validity and item and dimension reliability; it can be now more confidently used in clinical practice.
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Transtorno do Deficit de Atenção com Hiperatividade/diagnóstico , Escalas de Graduação Psiquiátrica/normas , Psicometria/instrumentação , Adolescente , Adulto , Criança , Feminino , França , Humanos , Masculino , Reprodutibilidade dos Testes , Adulto JovemRESUMO
UNLABELLED: Renal dysfunction is frequent in liver cirrhosis and is a strong prognostic predictor of orthotopic liver transplantation (OLT) outcome. Therefore, an accurate evaluation of the glomerular filtration rate (GFR) is crucial in pre-OLT patients. However, in these patients plasma creatinine (Pcr) is inaccurate and the place of serum cystatine C (CystC) is still debated. New GFR-predicting equations, based on standardized assays of Pcr and/or CystC, have been recently recommended in the general population but their performance in cirrhosis patients has been rarely studied. We evaluated the performance of the recently published Chronic Kidney Disease Epidemiology Collaboration equations (CKD-EPI-Pcr, CKD-EPI-CystC, and CKD-EPI-Pcr-CystC) and the more classical ones (4- and 6-variable MDRD and Hoek formulas) in cirrhosis patients referred for renal evaluation before OLT. Inulin clearance was performed in 202 consecutive patients together with the determination of Pcr and CystC with assays traceable to primary reference materials. The performance of the GFR-predicting equations was evaluated according to ascites severity (no, moderate, or refractory) and to hepatic and renal dysfunctions (MELD score ≤ or >15 and KDOQI stages, respectively). In the whole population, CystC-based equations showed a better performance than Pcr-based ones (lower bias and higher 10% and 30% accuracies). CKD-EPI-CystC equation showed the best performance whatever the ascites severity and in presence of a significant renal dysfunction (GFR <60 mL/min/1.73 m(2)). CONCLUSION: Pcr-based GFR predicting equations are not reliable in pre-OLT patients even when an IDMS-traceable enzymatic Pcr assay is used. Whenever a CystC-assay traceable to primary reference materials is performed and when a true measurement of GFR is not possible, CystC-based equations, especially CKD-EPI-CystC, may be recommended to evaluate renal function and for KDOQI staging.
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Creatinina/sangue , Cistatina C/sangue , Taxa de Filtração Glomerular/fisiologia , Rim/fisiologia , Cirrose Hepática/cirurgia , Transplante de Fígado , Adolescente , Adulto , Idoso , Biomarcadores/sangue , Feminino , Humanos , Inulina/metabolismo , Cirrose Hepática/sangue , Masculino , Pessoa de Meia-Idade , Modelos Biológicos , Valor Preditivo dos Testes , Índice de Gravidade de Doença , Adulto JovemRESUMO
BACKGROUND: Since the 1990s and since the development of humanised monoclonal antibodies in 1998, the treatment of non-Hodgkin lymphoma has undergone profound changes. Follicular lymphoma (FL) was the first to benefit from this treatment, and several clinical trials have shown a significant improvement in overall survival, but little information is available at a population level. OBJECTIVE: Our objective was to estimate changes in FL-specific mortality at a population level, with an appropriate methodology. METHODS: Two French retrospective population-based studies on FL were conducted, one from 1995 to 2004, in 1477 patients, and one from 1995 to 2010, in 451 patients. Trends in excess mortality rates (EMRs) according to age, sex, Ann Arbor stage and year of diagnosis were evaluated using the flexible model of Remontet et al. RESULTS: Trends in the EMR differed according to age at diagnosis and was higher in advanced stage (III, IV) in patients older than 65 yr. The EMR decreased linearly from 1995 to 2010. This decrease was more marked for advanced stages. CONCLUSION: FL-specific mortality decreased over the years of diagnosis, and the difference according to the lymphoma stage diminished in more recent years. However, progress in the management of FL was not able to erase age-related differences.
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Linfoma Folicular/mortalidade , Vigilância da População , Adolescente , Adulto , Idade de Início , Idoso , Idoso de 80 Anos ou mais , Feminino , França/epidemiologia , História do Século XX , História do Século XXI , Humanos , Linfoma Folicular/diagnóstico , Linfoma Folicular/epidemiologia , Linfoma Folicular/história , Masculino , Pessoa de Meia-Idade , Estadiamento de Neoplasias , Sistema de Registros , Estudos Retrospectivos , Adulto JovemRESUMO
BACKGROUND: Several previous studies have shown relationships between adherence to HIV antiretroviral therapy (ART) and the viral load, the CD4 cell count, or mortality. However, the impact of variability in adherence to ART on the immunovirological response does not seem to have been investigated yet. METHODS: Monthly adherence data (November 1999 to April 2009) from 317 HIV-1 infected patients enrolled in the Senegalese ART initiative were analyzed. Latent-class trajectory models were used to build typical trajectories for the average adherence and the standardized variance of adherence. The relationship between the standardized variance of adherence and each of the change in CD4 cell count, the change in viral load, and mortality were investigated using, respectively, a mixed linear regression, a mixed logistic regression, and a Cox model with time-dependent covariates. All the models were adjusted on the average adherence. RESULTS: Three latent trajectories for the average adherence and three for the standardized variance of adherence were identified. The increase in CD4 cell count and the increase in the percentage of undetectable viral loads were negatively associated with the standardized variance of adherence but positively associated with the average adherence. The risk of death decreased significantly with the increase in the average adherence but increased significantly with the increase of the standardized variance of adherence. CONCLUSIONS: The impacts of the level and the variability of adherence on the immunovirological response and survival justify the inclusion of these aspects into the process of patient education: adherence should be both high and constant.
Assuntos
Fármacos Anti-HIV/provisão & distribuição , Terapia Antirretroviral de Alta Atividade , Infecções por HIV/tratamento farmacológico , Adesão à Medicação/estatística & dados numéricos , Adulto , Fármacos Anti-HIV/uso terapêutico , Contagem de Linfócito CD4 , Feminino , Programas Governamentais/métodos , Programas Governamentais/estatística & dados numéricos , Infecções por HIV/imunologia , Infecções por HIV/virologia , Humanos , Modelos Lineares , Modelos Logísticos , Masculino , Avaliação de Resultados em Cuidados de Saúde/métodos , Avaliação de Resultados em Cuidados de Saúde/estatística & dados numéricos , Modelos de Riscos Proporcionais , Senegal , Análise de Sobrevida , Fatores de Tempo , Carga Viral/efeitos dos fármacosRESUMO
PURPOSE: Inflammatory back pain, the main symptom in spondyloarthritis, is defined by the presence of two or more positive responses among the four items of Berlin Criteria. This study assesses the value of these criteria in detecting previously undiagnosed spondyloarthritis after an episode of acute anterior uveitis. METHODS: Records of patients addressed for etiological diagnosis after an acute anterior uveitis (April 2006 to November 2013) were retrospectively analysed. Patient characteristics, the presence of back pain, Berlin Criteria, the final diagnosis, and the ASAS classification criteria for spondyloarthritis were collected and analysed. RESULTS: The study included 102 patients (59.8% women). The mean age was 44.5 years. Uveitis cases were mainly unilateral (73.5%) and recurrent (58.8%). Twenty-one patients had some type of spondyloarthritis, 20 fulfilling retrospectively the ASAS criteria. Back pain with at least two positive items from Berlin Criteria was 61.9% sensitive and 97.5% specific in diagnosing spondyloarthritis. Considering only one positive item increased the sensitivity (90.5%) but decreased the specificity (91.4%). CONCLUSIONS: Acute anterior uveitis may be the key symptom that reveals a formerly undiagnosed spondyloarthritis. A referral to a rheumatologist should be considered in presence of back pain, even without fulfilment of the classical definition with Berlin Criteria.
Assuntos
Dor nas Costas/diagnóstico , Sociedades Médicas/normas , Espondilartrite/diagnóstico , Uveíte Anterior/diagnóstico , Doença Aguda , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Algoritmos , Berlim , Árvores de Decisões , Reações Falso-Positivas , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Valor Preditivo dos Testes , Estudos Retrospectivos , Sensibilidade e Especificidade , Espondilartrite/classificaçãoRESUMO
OBJECTIVE: To develop and validate an English version of the Neuromuscular (NM)-Score, a classification for patients with NM diseases in each of the 3 motor function domains: D1, standing and transfers; D2, axial and proximal motor function; and D3, distal motor function. DESIGN: Validation survey. SETTING: Patients seen at a medical research center between June and September 2013. PARTICIPANTS: Consecutive patients (N=42) aged 5 to 19 years with a confirmed or suspected diagnosis of congenital muscular dystrophy. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: An English version of the NM-Score was developed by a 9-person expert panel that assessed its content validity and semantic equivalence. Its concurrent validity was tested against criterion standards (Brooke Scale, Motor Function Measure [MFM], activity limitations for patients with upper and/or lower limb impairments [ACTIVLIM], Jebsen Test, and myometry measurements). Informant agreement between patient/caregiver (P/C)-reported and medical doctor (MD)-reported NM scores was measured by weighted kappa. RESULTS: Significant correlation coefficients were found between NM scores and criterion standards. The highest correlations were found between NM-score D1 and MFM score D1 (ρ=-.944, P<.0001), ACTIVLIM (ρ=-.895, P<.0001), and hip abduction strength by myometry (ρ=-.811, P<.0001). Informant agreement between P/C-reported and MD-reported NM scores was high for D1 (κ=.801; 95% confidence interval [CI], .701-.914) but moderate for D2 (κ=.592; 95% CI, .412-.773) and D3 (κ=.485; 95% CI, .290-.680). Correlation coefficients between the NM scores and the criterion standards did not significantly differ between P/C-reported and MD-reported NM scores. CONCLUSIONS: Patients and physicians completed the English NM-Score easily and accurately. The English version is a reliable and valid instrument that can be used in clinical practice and research to describe the functional abilities of patients with NM diseases.
Assuntos
Destreza Motora/classificação , Distrofias Musculares/fisiopatologia , Inquéritos e Questionários , Traduções , Atividades Cotidianas , Adolescente , Criança , Pré-Escolar , Competência Cultural , Inglaterra , Feminino , Humanos , Masculino , Distrofias Musculares/congênito , Variações Dependentes do Observador , Reprodutibilidade dos Testes , Índice de Gravidade de Doença , Adulto JovemRESUMO
OBJECTIVES: To monitor treatment effects in patients with congenital myopathies and congenital muscular dystrophies, valid outcome measures are necessary. The Motor Function Measure (MFM) was examined for robustness, and changes are proposed for better adequacy. DESIGN: Observational study based on data previously collected from several cohorts. SETTING: Nineteen departments of physical medicine or neuromuscular consultation in France, Belgium, and the United States. PARTICIPANTS: Patients (N=289) aged 5 to 77 years. INTERVENTIONS: None. MAIN OUTCOME MEASURES: A Rasch analysis examined the robustness of the MFM across the disease spectrum. The 3 domains of the scale (standing position and transfers, axial and proximal motor function, and distal motor function) were independently examined with a partial credit model. RESULTS: The original 32-item MFM did not sufficiently fit the Rasch model expectations in either of its domains. Switching from a 4- to a 3-category response scale in 18 items restored response order in 16. Various additional checks suggested the removal of 7 items. The resulting Rasch-scaled Motor Function Measure with 25 items for congenital disorders of the muscle (Rs-MFM25(CDM)) demonstrated a good fit to the Rasch model. Domain 1 was well targeted to the whole severity spectrum-close mean locations for items and persons (0 vs 0.316)-whereas domains 2 and 3 were better targeted to severe cases. The reliability coefficients of the Rs-MFM25(CDM) suggested sufficient ability for each summed score to distinguish between patient groups (0.9, 0.8, and 0.7 for domains 1, 2, and 3, respectively). A sufficient agreement was found between results of the Rasch analysis and physical therapists' opinions. CONCLUSIONS: The Rs-MFM25(CDM) can be considered a clinically relevant linear scale in each of its 3 domains and may be soon reliably used for assessment in congenital disorders of the muscle.
Assuntos
Atividade Motora/fisiologia , Destreza Motora/fisiologia , Distrofias Musculares/fisiopatologia , Postura , Adolescente , Adulto , Idoso , Criança , Pré-Escolar , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Distrofias Musculares/congênito , Distrofias Musculares/reabilitação , Psicometria , Reprodutibilidade dos Testes , Resultado do Tratamento , Adulto JovemRESUMO
Net survival, the survival which might occur if cancer was the only cause of death, is a major epidemiological indicator required for international or temporal comparisons. Recent findings have shown that all classical methods used for routine estimation of net survival from cancer-registry data, sometimes called "relative-survival methods," provide biased estimates. Meanwhile, an unbiased estimator, the Pohar-Perme estimator (PPE), was recently proposed. Using real data, we investigated the magnitude of the errors made by four "relative-survival" methods (Ederer I, Hakulinen, Ederer II and a univariable regression model) vs. PPE as reference and examined the influence of time of follow-up, cancer prognosis, and age on the errors made. The data concerned seven cancer sites (2,51,316 cases) collected by FRANCIM cancer registries. Net survivals were estimated at 5, 10 and 15 years postdiagnosis. At 5 years, the errors were generally small. At 10 years, in good-prognosis cancers, the errors made in nonstandardized estimates with all classical methods were generally great (+2.7 to +9% points in prostate cancer) and increased in age-class estimations (vs. 5-year ones). At 15 years, in bad- or average-prognosis cancers, the errors were often substantial whatever the nature of the estimation. In good-prognosis cancers, the errors in nonstandardized estimates of all classical methods were great and sometimes very important. With all classical methods, great errors occurred in age-class estimates resulting in errors in age-standardized estimates (+0.4 to +3.2% points in breast cancer). In estimating net survival, cancer registries should abandon all classical methods and adopt the new Pohar-Perme estimator.