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1.
Pediatr Crit Care Med ; 24(2): 84-92, 2023 02 01.
Artigo em Inglês | MEDLINE | ID: mdl-36661416

RESUMO

OBJECTIVES: Guidelines recommend against RBC transfusion in hemodynamically stable (HDS) children without cardiac disease, if hemoglobin is greater than or equal to 7 g/dL. We sought to assess the clinical and economic impact of compliance with RBC transfusion guidelines. DESIGN: A nonprespecified secondary analysis of noncardiac, HDS patients in the randomized trial Age of Blood in Children (NCT01977547) in PICUs. Costs analyzed included ICU stay and physician fees. Stabilized inverse propensity for treatment weighting was used to create a cohort balanced with respect to potential confounding variables. Weighted regression models were fit to evaluate outcomes based on guideline compliance. SETTING: Fifty international tertiary care centers. PATIENTS: Critically ill children 3 days to 16 years old transfused RBCs at less than or equal to 7 days of ICU admission. Six-hundred eighty-seven subjects who met eligibility criteria were included in the analysis. INTERVENTIONS: Initial RBC transfusions administered when hemoglobin was less than 7 g/dL were considered "compliant" or "non-compliant" if hemoglobin was greater than or equal to 7 g/dL. MEASUREMENTS AND MAIN RESULTS: Frequency of new or progressive multiple organ system dysfunction (NPMODS), ICU survival, and associated costs. The hypothesis was formulated after data collection but exposure groups were masked until completion of planned analyses. Forty-nine percent of patients (338/687) received a noncompliant initial transfusion. Weighted cohorts were balanced with respect to confounding variables (absolute standardized differences < 0.1). No differences were noted in NPMODS frequency (relative risk, 0.86; 95% CI, 0.61-1.22; p = 0.4). Patients receiving compliant transfusions had more ICU-free days (mean difference, 1.73; 95% CI, 0.57-2.88; p = 0.003). Compliance reduced mean costs in ICU by $38,845 U.S. dollars per patient (95% CI, $65,048-$12,641). CONCLUSIONS: Deferring transfusion until hemoglobin is less than 7 g/dL is not associated with increased organ dysfunction in this population but is independently associated with increased likelihood of live ICU discharge and lower ICU costs.


Assuntos
Estado Terminal , Cardiopatias , Humanos , Criança , Estado Terminal/terapia , Transfusão de Eritrócitos/métodos , Hemoglobinas/análise , Unidades de Terapia Intensiva Pediátrica
2.
Stroke ; 53(7): 2299-2306, 2022 07.
Artigo em Inglês | MEDLINE | ID: mdl-35360927

RESUMO

BACKGROUND: There have been multiple efforts toward individual prediction of recurrent strokes based on structured clinical and imaging data using machine learning algorithms. Some of these efforts resulted in relatively accurate prediction models. However, acquiring clinical and imaging data is typically possible at provider sites only and is associated with additional costs. Therefore, we developed recurrent stroke prediction models based solely on data easily obtained from the patient at home. METHODS: Data from 384 patients with ischemic stroke were obtained from the Erlangen Stroke Registry. Patients were followed at 3 and 12 months after first stroke and then annually, for about 2 years on average. Multiple machine learning algorithms were applied to train predictive models for estimating individual risk of recurrent stroke within 1 year. Double nested cross-validation was utilized for conservative performance estimation and models' learning capabilities were assessed by learning curves. Predicted probabilities were calibrated, and relative variable importance was assessed using explainable artificial intelligence techniques. RESULTS: The best model achieved the area under the curve of 0.70 (95% CI, 0.64-0.76) and relatively good probability calibration. The most predictive factors included patient's family and housing circumstances, rehabilitative measures, age, high calorie diet, systolic and diastolic blood pressures, percutaneous endoscopic gastrotomy, number of family doctor's home visits, and patient's mental state. CONCLUSIONS: Developing fairly accurate models for individual risk prediction of recurrent ischemic stroke within 1 year solely based on registry data is feasible. Such models could be applied in a home setting to provide an initial risk assessment and identify high-risk patients early.


Assuntos
AVC Isquêmico , Acidente Vascular Cerebral , Inteligência Artificial , Humanos , Aprendizado de Máquina , Sistema de Registros , Acidente Vascular Cerebral/diagnóstico , Acidente Vascular Cerebral/epidemiologia
3.
Value Health ; 24(11): 1570-1577, 2021 11.
Artigo em Inglês | MEDLINE | ID: mdl-34711356

RESUMO

OBJECTIVES: To assist with planning hospital resources, including critical care (CC) beds, for managing patients with COVID-19. METHODS: An individual simulation was implemented in Microsoft Excel using a discretely integrated condition event simulation. Expected daily cases presented to the emergency department were modeled in terms of transitions to and from ward and CC and to discharge or death. The duration of stay in each location was selected from trajectory-specific distributions. Daily ward and CC bed occupancy and the number of discharges according to care needs were forecast for the period of interest. Face validity was ascertained by local experts and, for the case study, by comparing forecasts with actual data. RESULTS: To illustrate the use of the model, a case study was developed for Guy's and St Thomas' Trust. They provided inputs for January 2020 to early April 2020, and local observed case numbers were fit to provide estimates of emergency department arrivals. A peak demand of 467 ward and 135 CC beds was forecast, with diminishing numbers through July. The model tended to predict higher occupancy in Level 1 than what was eventually observed, but the timing of peaks was quite close, especially for CC, where the model predicted at least 120 beds would be occupied from April 9, 2020, to April 17, 2020, compared with April 7, 2020, to April 19, 2020, in reality. The care needs on discharge varied greatly from day to day. CONCLUSIONS: The DICE simulation of hospital trajectories of patients with COVID-19 provides forecasts of resources needed with only a few local inputs. This should help planners understand their expected resource needs.


Assuntos
COVID-19/economia , Simulação por Computador/normas , Alocação de Recursos/métodos , Capacidade de Resposta ante Emergências/economia , COVID-19/prevenção & controle , COVID-19/terapia , Humanos , Alocação de Recursos/normas , Capacidade de Resposta ante Emergências/tendências
4.
Value Health ; 23(4): 461-470, 2020 04.
Artigo em Inglês | MEDLINE | ID: mdl-32327163

RESUMO

OBJECTIVES: Biologic disease-modifying antirheumatic drugs (bDMARDs) are prescribed sequentially in the treatment of rheumatoid arthritis (RA). Healthcare decision makers continue to debate their use, mainly because of their high costs. Our aim was to perform an economic evaluation for France of bDMARD sequences for treatment of moderate-to-severe RA after inadequate response or intolerance to conventional DMARDs (eg, methotrexate). METHODS: A discretely integrated condition event simulation was developed to track the course of patients from first bDMARD through switches to further lines in a sequence. The model included 11 events, 91 conditions, and 21 controlling equations. Inputs were obtained from a meta-analysis of clinical trials, a French registry, national drug lists, and databases. Survival, time with minimal activity, quality-adjusted life-years (QALYs), and total costs were output. Structural and probabilistic sensitivity analyses were conducted. RESULTS: Sequences starting with etanercept biosimilars (ETB) cost less, with ETB-abatacept-infliximab the least expensive: the mean lifetime discounted total cost was €116 912 per patient, with a mean of 11.166 QALYs. Most other strategies were dominated or led to small QALY gains (0.0008-0.0329). Only ETB-tocilizumab-abatacept made it onto the efficiency frontier, but at €955 778 per QALY gained. These results were confirmed in several scenarios and uncertainty analyses. CONCLUSION: Given minor differences in QALYs gained between bDMARD sequences with large cost differences, starting with biosimilars was more efficient than starting with branded products. Our model and findings should provide French and other decision makers with useful tools to address the challenges of comparing sequences of treatments for RA.


Assuntos
Antirreumáticos/administração & dosagem , Artrite Reumatoide/tratamento farmacológico , Metotrexato/administração & dosagem , Modelos Econômicos , Antirreumáticos/economia , Artrite Reumatoide/economia , Artrite Reumatoide/fisiopatologia , Medicamentos Biossimilares/administração & dosagem , Medicamentos Biossimilares/economia , Análise Custo-Benefício , França , Humanos , Metotrexato/efeitos adversos , Metotrexato/economia , Anos de Vida Ajustados por Qualidade de Vida , Índice de Gravidade de Doença
5.
Value Health ; 23(8): 1049-1055, 2020 08.
Artigo em Inglês | MEDLINE | ID: mdl-32828217

RESUMO

OBJECTIVES: Using an example of an existing model constructed by the National Institute for Health and Care Excellence (NICE) to inform a real health technology assessment, this study seeks to demonstrate how a discretely integrated condition event (DICE) simulation can improve the implementation of Markov models. METHODS: Using the technical report and spreadsheet, the original model was translated to a standard DICE simulation without making any changes to the design. All original analyses were repeated and the results were compared. Aspects that could have improved the original design were then considered. RESULTS: The original model consisted of 32 copies (8 risk strata × 4 treatments) of the Markov structure, containing more than 6000 Microsoft Excel® formulas (18 MB files). Three aspects (nonadherence, scheduled treatment stop, and end of fracture risk) were handled by incorporating weighted averages into the cycle-specific calculations. The DICE implementation used 3 conditions to represent the states and a single transition event to apply the probabilities; 3 additional events processed the special aspects, and profiles handled the 8 strata (0.12 MB file). One replication took 16 seconds. The original results were reproduced but extensive additional sensitivity analyses, including structural analyses, were enabled. CONCLUSION: Implementing a real Markov model using DICE simulation both preserves the advantages of the approach and expands the available tools, improving transparency and ease of use and review.


Assuntos
Simulação por Computador , Cadeias de Markov , Modelos Estatísticos , Avaliação da Tecnologia Biomédica/organização & administração , Técnicas de Apoio para a Decisão , Humanos , Probabilidade
6.
JAMA ; 322(22): 2179-2190, 2019 12 10.
Artigo em Inglês | MEDLINE | ID: mdl-31821429

RESUMO

Importance: The clinical consequences of red blood cell storage age for critically ill pediatric patients have not been examined in a large, randomized clinical trial. Objective: To determine if the transfusion of fresh red blood cells (stored ≤7 days) reduced new or progressive multiple organ dysfunction syndrome compared with the use of standard-issue red blood cells in critically ill children. Design, Setting, and Participants: The Age of Transfused Blood in Critically-Ill Children trial was an international, multicenter, blinded, randomized clinical trial, performed between February 2014 and November 2018 in 50 tertiary care centers. Pediatric patients between the ages of 3 days and 16 years were eligible if the first red blood cell transfusion was administered within 7 days of intensive care unit admission. A total of 15 568 patients were screened, and 13 308 were excluded. Interventions: Patients were randomized to receive either fresh or standard-issue red blood cells. A total of 1538 patients were randomized with 768 patients in the fresh red blood cell group and 770 in the standard-issue group. Main Outcomes and Measures: The primary outcome measure was new or progressive multiple organ dysfunction syndrome, measured for 28 days or to discharge or death. Results: Among 1538 patients who were randomized, 1461 patients (95%) were included in the primary analysis (median age, 1.8 years; 47.3% girls), in which there were 728 patients randomized to the fresh red blood cell group and 733 to the standard-issue group. The median storage duration was 5 days (interquartile range [IQR], 4-6 days) in the fresh group vs 18 days (IQR, 12-25 days) in the standard-issue group (P < .001). There were no significant differences in new or progressive multiple organ dysfunction syndrome between fresh (147 of 728 [20.2%]) and standard-issue red blood cell groups (133 of 732 [18.2%]), with an unadjusted absolute risk difference of 2.0% (95% CI, -2.0% to 6.1%; P = .33). The prevalence of sepsis was 25.8% (160 of 619) in the fresh group and 25.3% (154 of 608) in the standard-issue group. The prevalence of acute respiratory distress syndrome was 6.6% (41 of 619) in the fresh group and 4.8% (29 of 608) in the standard-issue group. Intensive care unit mortality was 4.5% (33 of 728) in the fresh group vs 3.5 % (26 of 732) in the standard-issue group (P = .34). Conclusions and Relevance: Among critically ill pediatric patients, the use of fresh red blood cells did not reduce the incidence of new or progressive multiple organ dysfunction syndrome (including mortality) compared with standard-issue red blood cells. Trial Registration: ClinicalTrials.gov Identifier: NCT01977547.


Assuntos
Preservação de Sangue , Estado Terminal/terapia , Transfusão de Eritrócitos , Insuficiência de Múltiplos Órgãos/prevenção & controle , Adolescente , Criança , Pré-Escolar , Estado Terminal/mortalidade , Progressão da Doença , Transfusão de Eritrócitos/efeitos adversos , Feminino , Mortalidade Hospitalar , Humanos , Lactente , Recém-Nascido , Unidades de Terapia Intensiva Pediátrica , Estimativa de Kaplan-Meier , Masculino , Insuficiência de Múltiplos Órgãos/mortalidade , Gravidade do Paciente , Síndrome do Desconforto Respiratório do Recém-Nascido/terapia , Sepse/etiologia
7.
HNO ; 67(7): 515-518, 2019 Jul.
Artigo em Alemão | MEDLINE | ID: mdl-31197423

RESUMO

Nowadays, social projects are usually oriented in such a way that after a given period of time, they can either support themselves independently or even allow a pecuniary reimbursement. In the latter case, experts speak of a profit-oriented reimbursement. On the other hand, there is so-called social reimbursement, which in contrast to the abovementioned form is not profit oriented, but, for example, considers its task fulfilled by the fact of successful knowledge transfer. The Spanish-German Society for ENT Medicine and Head and Neck Surgery (SDGHNO) launched the Latin America project in 2001 under the patronage of the then President Prof. Dr. Wolfgang Draf (Fulda). The goal of the SDGHNO was and is to create a professional as well as cultural platform for Spanish- and German-speaking ENT doctors. This platform can and should be used for professional purposes, e.g., for knowledge transfer. Since the beginning of its existence, the Latin America project has thus brought numerous scientific events into being and created specific contacts which have lasted until today or have even been continued and further developed. Particularly successful examples are Chile, Colombia, and Peru. This is a vivid example of social reimbursement, because the participating German-speaking members/speakers carried out their tasks on an entirely voluntary basis. Thus, the SDGHNO did not bear any travel, catering, or accommodation costs. The activities of the SDGHNO within the framework of the Latin America project are explained.


Assuntos
Otolaringologia , América Latina , Condições Sociais
8.
Value Health ; 21(4): 394-397, 2018 04.
Artigo em Inglês | MEDLINE | ID: mdl-29680094

RESUMO

The potential for multi-criteria decision analysis (MCDA) to support health technology assessment (HTA) has been much discussed, and various HTA agencies are piloting or applying MCDA. Alongside these developments, good practice guidelines for the application of MCDA in health care have been developed. An assessment of current applications of MCDA to HTA in light of good practice guidelines reveals, however, that many have methodologic flaws that undermine their usefulness. Three challenges are considered: the use of additive models, a lack of connection between criteria scales and weights, and the use of MCDA in economic evaluation. More attention needs to be paid to MCDA good practice by researchers, journal editors, and decision makers and further methodologic developments are required if MCDA is to achieve its potential to support HTA.


Assuntos
Técnicas de Apoio para a Decisão , Avaliação da Tecnologia Biomédica/métodos , Análise Custo-Benefício , Fidelidade a Diretrizes , Custos de Cuidados de Saúde , Humanos , Modelos Econômicos , Medidas de Resultados Relatados pelo Paciente , Guias de Prática Clínica como Assunto , Participação dos Interessados , Avaliação da Tecnologia Biomédica/economia , Avaliação da Tecnologia Biomédica/normas
9.
Int J Technol Assess Health Care ; 34(1): 105-110, 2018 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-29277175

RESUMO

OBJECTIVES: Patient preferences should be a central consideration in healthcare decision making. However, stories of patients challenging regulatory and reimbursement decisions has led to questions on whether patient voices are being considered sufficiently during those decision making processes. This has led some to argue that it is necessary to quantify patient preferences before they can be adequately considered. METHODS: This study considers the lessons from the use of multi-criteria decision analysis (MCDA) for efforts to quantify patient preferences. It defines MCDA and summarizes the benefits it can provide to decision makers, identifies examples of MCDAs that have involved patients, and summarizes good practice guidelines as they relate to quantifying patient preferences. RESULTS: The guidance developed to support the use of MCDA in healthcare provide some useful considerations for the quantification of patient preferences, namely that researchers should give appropriate consideration to: the heterogeneity of patient preferences, and its relevance to decision makers; the cognitive challenges posed by different elicitation methods; and validity of the results they produce. Furthermore, it is important to consider how the relevance of these considerations varies with the decision being supported. CONCLUSIONS: The MCDA literature holds important lessons for how patient preferences should be quantified to support healthcare decision making.


Assuntos
Técnicas de Apoio para a Decisão , Preferência do Paciente , Assistência Centrada no Paciente/organização & administração , Avaliação da Tecnologia Biomédica/organização & administração , Tomada de Decisões , Humanos
10.
Chem Soc Rev ; 44(20): 7128-54, 2015 Oct 21.
Artigo em Inglês | MEDLINE | ID: mdl-26155855

RESUMO

The latest developments in zeolite membranes are reviewed, with an emphasis on the synthesis techniques, including seed assembly and secondary growth methods. This review also discusses the current industrial applications of zeolite membranes, the feasibility of their use in membrane reactors and their hydrothermal stability. Finally, zeolite membranes are compared with metal-organic framework (MOF) membranes and the latest advancements in MOF and mixed matrix membranes are highlighted.

12.
Eur Heart J ; 35(30): 2001-9, 2014 Aug 07.
Artigo em Inglês | MEDLINE | ID: mdl-24904027

RESUMO

Patient-reported outcomes (PROs), such as symptoms, health-related quality of life (HRQOL), or patient perceived health status, are reported directly by the patient and are powerful tools to inform patients, clinicians, and policy-makers about morbidity and 'patient suffering', especially in chronic diseases. Patient-reported outcomes provide information on the patient experience and can be the target of therapeutic intervention. Patient-reported outcomes can improve the quality of patient care by creating a holistic approach to clinical decision-making; however, PROs are not routinely used as key outcome measures in major cardiovascular clinical trials. Thus, limited information is available on the impact of cardiovascular therapeutics on PROs to guide patient-level clinical decision-making or policy-level decision-making. Cardiovascular clinical research should shift its focus to include PROs when evaluating the efficacy of therapeutic interventions, and PRO assessments should be scientifically rigorous. The European Society of Cardiology and other professional societies can take action to influence the uptake of PRO data in the research and clinical communities. This process of integrating PRO data into comprehensive efficacy evaluations will ultimately improve the quality of care for patients across the spectrum of cardiovascular disease.


Assuntos
Doenças Cardiovasculares/terapia , Ensaios Clínicos como Assunto/métodos , Avaliação de Resultados da Assistência ao Paciente , Cardiologia , Coleta de Dados , Interpretação Estatística de Dados , Europa (Continente) , Humanos , Disseminação de Informação , Reembolso de Seguro de Saúde , Política Organizacional , Qualidade de Vida , Sociedades Médicas , Terminologia como Assunto
14.
Value Health ; 17(2): 174-82, 2014 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-24636375

RESUMO

The evaluation of the cost and health implications of agreeing to cover a new health technology is best accomplished using a model that mathematically combines inputs from various sources, together with assumptions about how these fit together and what might happen in reality. This need to make assumptions, the complexity of the resulting framework, the technical knowledge required, as well as funding by interested parties have led many decision makers to distrust the results of models. To assist stakeholders reviewing a model's report, questions pertaining to the credibility of a model were developed. Because credibility is insufficient, questions regarding relevance of the model results were also created. The questions are formulated such that they are readily answered and they are supplemented by helper questions that provide additional detail. Some responses indicate strongly that a model should not be used for decision making: these trigger a "fatal flaw" indicator. It is hoped that the use of this questionnaire, along with the three others in the series, will help disseminate what to look for in comparative effectiveness evidence, improve practices by researchers supplying these data, and ultimately facilitate their use by health care decision makers.


Assuntos
Pesquisa Comparativa da Efetividade/normas , Tomada de Decisões , Modelos Teóricos , Inquéritos e Questionários , Comitês Consultivos , Tecnologia Biomédica/economia , Atenção à Saúde/métodos , Humanos , Internacionalidade , Projetos de Pesquisa/normas , Avaliação da Tecnologia Biomédica/métodos
15.
Value Health ; 17(1): 5-14, 2014.
Artigo em Inglês | MEDLINE | ID: mdl-24438712

RESUMO

BACKGROUND: Budget impact analyses (BIAs) are an essential part of a comprehensive economic assessment of a health care intervention and are increasingly required by reimbursement authorities as part of a listing or reimbursement submission. OBJECTIVES: The objective of this report was to present updated guidance on methods for those undertaking such analyses or for those reviewing the results of such analyses. This update was needed, in part, because of developments in BIA methods as well as a growing interest, particularly in emerging markets, in matters related to affordability and population health impacts of health care interventions. METHODS: The Task Force was approved by the International Society for Pharmacoeconomics and Outcomes Research Health Sciences Policy Council and appointed by its Board of Directors. Members were experienced developers or users of BIAs; worked in academia and industry and as advisors to governments; and came from several countries in North America and South America, Oceania, Asia, and Europe. The Task Force solicited comments on the drafts from a core group of external reviewers and, more broadly, from the membership of the International Society for Pharmacoeconomics and Outcomes Research. RESULTS: The Task Force recommends that the design of a BIA for a new health care intervention should take into account relevant features of the health care system, possible access restrictions, the anticipated uptake of the new intervention, and the use and effects of the current and new interventions. The key elements of a BIA include estimating the size of the eligible population, the current mix of treatments and the expected mix after the introduction of the new intervention, the cost of the treatment mixes, and any changes expected in condition-related costs. Where possible, the BIA calculations should be performed by using a simple cost calculator approach because of its ease of use for budget holders. In instances, however, in which the changes in eligible population size, disease severity mix, or treatment patterns cannot be credibly captured by using the cost calculator approach, a cohort or patient-level condition-specific model may be used to estimate the budget impact of the new intervention, accounting appropriately for those entering and leaving the eligible population over time. In either case, the BIA should use data that reflect values specific to a particular decision maker's population. Sensitivity analysis should be of alternative scenarios chosen from the perspective of the decision maker. The validation of the model should include at least face validity with decision makers and verification of the calculations. Data sources for the BIA should include published clinical trial estimates and comparator studies for the efficacy and safety of the current and new interventions as well as the decision maker's own population for the other parameter estimates, where possible. Other data sources include the use of published data, well-recognized local or national statistical information, and, in special circumstances, expert opinion. Reporting of the BIA should provide detailed information about the input parameter values and calculations at a level of detail that would allow another modeler to replicate the analysis. The outcomes of the BIA should be presented in the format of interest to health care decision makers. In a computer program, options should be provided for different categories of costs to be included or excluded from the analysis. CONCLUSIONS: We recommend a framework for the BIA, provide guidance on the acquisition and use of data, and offer a common reporting format that will promote standardization and transparency. Adherence to these good research practice principles would not necessarily supersede jurisdiction-specific BIA guidelines but may support and enhance local recommendations or serve as a starting point for payers wishing to promulgate methodology guidelines.


Assuntos
Tecnologia Biomédica/economia , Orçamentos , Análise Custo-Benefício/métodos , Modelos Econômicos , Comitês Consultivos , Ensaios Clínicos como Assunto , Medicina Baseada em Evidências , Custos de Cuidados de Saúde , Política de Saúde , Humanos , Formulação de Políticas
16.
Orphanet J Rare Dis ; 19(1): 14, 2024 Jan 12.
Artigo em Inglês | MEDLINE | ID: mdl-38216959

RESUMO

BACKGROUND: Clinical trials for rare diseases often include multiple endpoints that capture the effects of treatment on different disease domains. In many rare diseases, the primary endpoint is not standardized across trials. The win ratio approach was designed to analyze multiple endpoints of interest in clinical trials and has mostly been applied in cardiovascular trials. Here, we applied the win ratio approach to data from COMET, a phase 3 trial in late-onset Pompe disease, to illustrate how this approach can be used to analyze multiple endpoints in the orphan drug context. METHODS: All possible participant pairings from both arms of COMET were compared sequentially on changes at week 49 in upright forced vital capacity (FVC) % predicted and six-minute walk test (6MWT). Each participant's response for the two endpoints was first classified as a meaningful improvement, no meaningful change, or a meaningful decline using thresholds based on published minimal clinically important differences (FVC ± 4% predicted, 6MWT ± 39 m). Each comparison assessed whether the outcome with avalglucosidase alfa (AVA) was better than (win), worse than (loss), or equivalent to (tie) the outcome with alglucosidase alfa (ALG). If tied on FVC, 6MWT was compared. In this approach, the treatment effect is the ratio of wins to losses ("win ratio"), with ties excluded. RESULTS: In the 2499 possible pairings (51 receiving AVA × 49 receiving ALG), the win ratio was 2.37 (95% confidence interval [CI], 1.30-4.29, p = 0.005) when FVC was compared before 6MWT. When the order was reversed, the win ratio was 2.02 (95% CI, 1.13-3.62, p = 0.018). CONCLUSION: The win ratio approach can be used in clinical trials of rare diseases to provide meaningful insight on treatment benefits from multiple endpoints and across disease domains.


Assuntos
Doença de Depósito de Glicogênio Tipo II , Humanos , Doença de Depósito de Glicogênio Tipo II/tratamento farmacológico , Produção de Droga sem Interesse Comercial , Doenças Raras/tratamento farmacológico , Resultado do Tratamento , Terapia de Reposição de Enzimas/métodos , alfa-Glucosidases/uso terapêutico
17.
Artigo em Inglês | MEDLINE | ID: mdl-39025229

RESUMO

AIM: To evaluate the efficacy, safety, structural and functional progression following the insertion of iStent inject ® implants in patients with open-angle glaucoma or ocular hypertension at a tertiary-level hospital. MATERIALS AND METHODS: A retrospective study included 98 eyes (57 males and 41 females) with open-angle glaucoma or ocular hypertension, which underwent iStent inject W® implantation (Glaukos, Corporation, CA) between December 2018 and December 2022. Differences in intraocular pressure (IOP), the number of hypotensive eye drops used, and structural and functional tests were assessed between preoperative values and subsequent reviews during a follow-up period of one (n = 98), two (n = 55), and three years (n = 15) after surgery. RESULTS: Among the 98 eyes studied, 85% were diagnosed with open-angle glaucoma (50% mild, 32% moderate, and 18% severe) and 15% with ocular hypertension. There was a statistically significant reduction in IOP compared to preoperative values for all visits except the 1-month (p = 0.36) and 3-year (p = 0.39) visits. Visual acuity increased from 0.39 ±â€¯0.25 to 0.72 ±â€¯0.24 (p < 0.01), considering that a significant portion of the interventions included cataract surgery. Before surgery, 66% of the sample used 2 or more hypotensive medications. Post-surgery, the number of hypotensive medications decreased (from 1.88 ±â€¯0.84 to 0.21 ±â€¯0.59 at 3 years) (p < 0.01), with an 88.9% reduction in the number of medications over three years. After surgery, 75% of cases did not require any medication. Regarding structural and functional tests, thickness of retinal nerve fiber layers (RNFL (p = 0.35), excavation / papilla ratio E/P (p = 0.31), visual function index (VFI (p = 0.06), and deviation mean (MD (p = 0.06) showed no statistically significant differences post-intervention. However, standard deviation of the pattern (DSM) did exhibit differences, decreasing from 5.46 ±â€¯4.03 dB to 5.34 ±â€¯3.48 dB (p = 0.02). CONCLUSION: The results of this study suggest that the iStent inject W® technique constitutes an effective and safe option for tension control and glaucoma treatment.

18.
Semergen ; 50(6): 102263, 2024 Jun 04.
Artigo em Inglês | MEDLINE | ID: mdl-38838584

RESUMO

OBJECTIVES: To determine the prevalence of suspected abuse of non-institutionalised elderly people and the associated variables. PATIENTS AND METHOD: Observational, descriptive, cross-sectional, multicentre study in patients aged 65 years or older, non-institutionalised, consecutively selected in primary care (PC). The EASI questionnaires (Suspected Elderly Abuse Index), the EAI questionnaire (Suspected Abuse Index in patients with cognitive impairment), the Barthel index, and the EUROQOL-5D questionnaire were used with patients, and the CASE questionnaire and the Zarit test were used with caregivers. Socio-demographic, health, and quality of life variables were analysed in all patients. RESULTS: Eight hundred four patients were included, mean age 78.9±7.9 years, 58.3% women. The prevalence of suspected abuse was 11.3% (95% CI: 9.1%-13.9%). Suspected abuse was more frequent in women than in men (14.4% vs. 7.1%; odds ratio (OR)=1.97; 95% CI=1.1-3.4; p=0.016) and in those who lived with two or more people compared to those who lived alone (18.4% vs. 7.3%; OR=2.42; 95% CI=1.1-5.0; p=0.017). Among older patients, the lower their dependency, the lower the prevalence of suspected abuse (30.0% in highly dependent vs. 8.7% in non-dependent: p-trend=0.006); and the better the perceived health status, the lower the prevalence of suspected abuse (29.6% in poor health status vs. 6.9% in optimal health status; p-trend=<0.001). Among caregivers, the prevalence of suspected abuse was 20.4% (95% CI=12.8%-28.0%). A trend of higher prevalence of suspected abuse could be observed with higher scores on the CASE questionnaire (56.3% at high risk and 9.6% with no risk of abuse; p-trend=0.007). In the case of the ZARIT questionnaire with scores below 47, the prevalence of suspected abuse was 9.1%, and for scores above 55, it was 52.6% (p-trend<0.001). CONCLUSIONS: The results of the PRESENCIA study show that approximately 1 in 10 patients aged ≥65 meet the criteria for suspected abuse. The probability of abuse increases in women, in patients with greater dependency and in patients with poorer perceived health status. Caregivers with greater overload and greater risk presented a greater suspicion of elder abuse.

19.
Artigo em Inglês | MEDLINE | ID: mdl-23653967

RESUMO

BACKGROUND: Hereditary angioedema due to C1 inhibitor deficiency (HAE-C1-INH) has considerable implications for dental health care providers, since dental procedures may trigger severe and even life-threatening episodes. The aim of the present study was to analyze the efficacy and safety of premedication with attenuated androgens (AAs), plasma-derived human C1 esterase inhibitor concentrate (pdhC1INH), or both to prevent the development of upper airway angioedema after dental-oral procedures in patients with HAE-C1-INH. MATERIAL AND METHODS: All dental-oral procedures performed on patients with HAE-C1-INH who were followed up at La Paz University Hospital, Madrid, Spain were reviewed. Demographic data, maintenance treatment, preprocedure prophylaxis, disease severity, and occurrence of upper airway angioedema were recorded. RESULTS: Twenty-four patients (14 male/10 female; mean age, 42.6 years) underwent 66 procedures. Most procedures were performed on patients with severe HAE-C1-INH (20 procedures) or moderate HAE-C1-INH (26 procedures). Only 9 procedures were performed without short-term prophylaxis. Mild upper airway angioedema developed after 3 procedures performed without short-term prophylaxis in patients with minimal or asymptomatic HAE-C1-INH. A statistically significant association was found between development of mild postprocedure upper airway angioedema and lack of maintenance treatment with AA, lack of increased dose of preprocedure AA, and failure to administer preprocedure pdhC1INH (P = .002, Fisher exact test). CONCLUSIONS: Increased doses of prophylactic AA, administration of pdhC1INH, or both were good options for ambulatory management of dental-oral procedures in patients with HAE-C1-INH. Prophylaxis with pdC1INH or increased doses of AA is advisable before dental-oral procedures, even in patients with low disease severity.


Assuntos
Androgênios/uso terapêutico , Proteína Inibidora do Complemento C1/uso terapêutico , Angioedema Hereditário Tipos I e II/tratamento farmacológico , Angioedema Hereditário Tipos I e II/etiologia , Complicações Pós-Operatórias , Pré-Medicação , Doenças Estomatognáticas/complicações , Adulto , Androgênios/efeitos adversos , Proteína Inibidora do Complemento C1/efeitos adversos , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Doenças Estomatognáticas/cirurgia , Adulto Jovem
20.
Cancers (Basel) ; 15(18)2023 Sep 21.
Artigo em Inglês | MEDLINE | ID: mdl-37760642

RESUMO

The authors wish to revise two words in Table 1 row 3, and the first paragraph of Section 2 [...].

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