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1.
Vestn Oftalmol ; 140(2. Vyp. 2): 34-42, 2024.
Artigo em Russo | MEDLINE | ID: mdl-38739129

RESUMO

PURPOSE: This study analyzes changes in light sensitivity in each test point of the visual field in patients with different stages of glaucoma. MATERIAL AND METHODS: The data of a prospective analytical case-control study were analyzed. All patients underwent assessment of retinal light sensitivity and its variability in 54 points corresponding to the 24-2 program. Mean light sensitivity values were calculated in each point. Intergroup analysis was performed to evaluate changes in light sensitivity in each point. RESULTS: The range of light sensitivity decrease in the early glaucoma group compared to the control group was from 1.5 to 3.6 dB. The range of light sensitivity decrease in the moderate glaucoma group compared to the control group was from 2.1 to 11.5 dB, and compared to the early glaucoma group - from -0.9 to 7.9 dB. The most frequent decrease in light sensitivity was detected in the nasal sector and along the horizontal line in the upper half of the visual field. This trend persisted within the central 10 degrees of the visual field. The range of light sensitivity decrease in the advanced glaucoma group compared to the control group was from 14.1 to 28.0 dB, and compared to the early glaucoma group - from 11.35 to 26.08 dB, compared to the moderate glaucoma group - from 9.1 to 23.5 dB. The most frequent and severe decrease in light sensitivity was detected in the paracentral zone in the lower half of the visual field. CONCLUSION: The study analyzed the trends in the development of glaucoma from the early to the advanced stage. The most frequent and severe defect in light sensitivity in cases of verified advanced glaucoma was found in the lower half of the visual field. Points No. 32, 33 and 40 can be indicated as the area of interest in assessing the progression of glaucoma, as they were found to have the most profound changes in light sensitivity as glaucoma progressed.


Assuntos
Progressão da Doença , Glaucoma , Testes de Campo Visual , Campos Visuais , Humanos , Campos Visuais/fisiologia , Glaucoma/fisiopatologia , Glaucoma/diagnóstico , Masculino , Feminino , Pessoa de Meia-Idade , Testes de Campo Visual/métodos , Estudos Prospectivos , Idoso , Estudos de Casos e Controles , Luz
3.
Braz J Phys Ther ; 28(2): 101050, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-38574558

RESUMO

BACKGROUND: Femoroacetabular impingement syndrome (FAIS) is a hip joint motion-related clinical disorder with a triad of symptoms, clinical signs, and imaging findings. However, scientific evidence is still unclear regarding the best treatment for FAIS. OBJECTIVES: To assess the value of a physical therapy evaluation in predicting the progression of functional status over the subsequent years in patients with FAIS who are candidates for hip arthroscopy surgery. METHODS: In this case-series study, patients with FAIS, candidates for hip arthroscopy surgery, underwent a standard physical therapy evaluation. Baseline data were collected between 2013 and 2019. In 2020/2021, the patients' functional status was assessed through the International Hip Outcome Tool (iHOT-33). Functional status progression was calculated as the difference between the follow-up and baseline iHOT-33 scores. A multivariate forward stepwise regression analysis was conducted to explore the relationship between baseline characteristics and the functional status progression. RESULTS: From 353 patients who completed the baseline assessment, 145 completed the iHOT-33 follow-up. The mean (±SD) follow-up time was 58.7 (27.2) months (minimum 12 and maximum 103 months). The iHOT-33 scores increased 20.7 (21.8) points on average, ranging from -39.8 to 76.9 points. Among the 15 potential predictive factors assessed in this study, only baseline iHOT-33 score (ß -0.44; -0.061, -0.27), femoral version (ß 9.03; 1.36, 16.71), and body mass index (ß -0.99; -1.98, -0.01) had the ability to predict the functional status progression. CONCLUSION: Patients with a lower baseline iHOT-33 score, lower body mass index, and normal femoral version were more likely to increase their functional status after a minimum of one year of follow-up.


Assuntos
Impacto Femoroacetabular , Humanos , Impacto Femoroacetabular/fisiopatologia , Artroscopia , Articulação do Quadril/fisiopatologia , Modalidades de Fisioterapia , Amplitude de Movimento Articular , Progressão da Doença
4.
BMC Pulm Med ; 24(1): 184, 2024 Apr 17.
Artigo em Inglês | MEDLINE | ID: mdl-38632584

RESUMO

Chronic obstructive pulmonary disease (COPD) is a frequent cause of morbidity and mortality in the Philippines and majority of the economic burden lies in hospitalizations during an exacerbation. Despite coverage of hospitalization cost with the national health insurance system (Phil-Health) for COPD exacerbations, patients often pay out-of-pocket. This study aimed to determine the demographic characteristics of COPD admissions at a Philippine tertiary care center, Philippine General Hospital, and assess mean cost of hospitalization, and identify predictors of prolonged hospitalization and cost > 20,000 Philippine pesos (Php). A prospective cross-sectional study was conducted for 6 months by chart review. Patients were categorized as charity service patients, that is, with no charged professional fees and free medications and private service patients who pay for their health care services. A total of 43 COPD admissions were included. The average daily cost of hospitalization (at peso-dollar rate of 56) for service patients was at $ 75.89 compared to private service patients at $ 285.71. Demographic characteristics and type of accommodation were not significant predictors of prolonged hospital stay nor hospitalization cost of ≥ $ 357. Accommodation cost and professional fees accounted for majority or 61.6% of the overall cost for private patients, while medications and diagnostic tests were the major or 76.01% contributor to the overall cost for charity patients. Despite existence of Phil-health, in-patient coverage for COPD remain insufficient. Measures for maximizing COPD control in the out-patient setting could potentially reduce total cost for this disease.


Assuntos
Gastos em Saúde , Doença Pulmonar Obstrutiva Crônica , Humanos , Estudos Prospectivos , Filipinas , Centros de Atenção Terciária , Estudos Transversais , Doença Pulmonar Obstrutiva Crônica/diagnóstico , Hospitalização , Progressão da Doença
5.
BMC Psychiatry ; 24(1): 309, 2024 Apr 24.
Artigo em Inglês | MEDLINE | ID: mdl-38658884

RESUMO

BACKGROUND: Lateral ventricular enlargement represents a canonical morphometric finding in chronic patients with schizophrenia; however, longitudinal studies elucidating complex dynamic trajectories of ventricular volume change during critical early disease stages are sparse. METHODS: We measured lateral ventricular volumes in 113 first-episode schizophrenia patients (FES) at baseline visit (11.7 months after illness onset, SD = 12.3) and 128 age- and sex-matched healthy controls (HC) using 3T MRI. MRI was then repeated in both FES and HC one year later. RESULTS: Compared to controls, ventricular enlargement was identified in 18.6% of patients with FES (14.1% annual ventricular volume (VV) increase; 95%CI: 5.4; 33.1). The ventricular expansion correlated with the severity of PANSS-negative symptoms at one-year follow-up (p = 0.0078). Nevertheless, 16.8% of FES showed an opposite pattern of statistically significant ventricular shrinkage during ≈ one-year follow-up (-9.5% annual VV decrease; 95%CI: -23.7; -2.4). There were no differences in sex, illness duration, age of onset, duration of untreated psychosis, body mass index, the incidence of Schneiderian symptoms, or cumulative antipsychotic dose among the patient groups exhibiting ventricular enlargement, shrinkage, or no change in VV. CONCLUSION: Both enlargement and ventricular shrinkage are equally present in the early stages of schizophrenia. The newly discovered early reduction of VV in a subgroup of patients emphasizes the need for further research to understand its mechanisms.


Assuntos
Imageamento por Ressonância Magnética , Esquizofrenia , Humanos , Esquizofrenia/diagnóstico por imagem , Esquizofrenia/patologia , Esquizofrenia/fisiopatologia , Masculino , Feminino , Estudos Longitudinais , Adulto , Adulto Jovem , Ventrículos Cerebrais/diagnóstico por imagem , Ventrículos Cerebrais/patologia , Ventrículos Laterais/diagnóstico por imagem , Ventrículos Laterais/patologia , Progressão da Doença , Estudos de Casos e Controles , Adolescente
6.
Respir Med ; 226: 107630, 2024 May.
Artigo em Inglês | MEDLINE | ID: mdl-38593886

RESUMO

INTRODUCTION: Definitions of moderate asthma exacerbation have been inconsistent, making their economic burden difficult to assess. An algorithm to accurately identify moderate exacerbations from claims data is needed. METHODS: A retrospective cohort study of Reliant Medical Group patients aged ≥18 years, with ≥1 prescription claim for inhaled corticosteroid/long-acting ß2-agonist, and ≥1 medical claim with a diagnosis code for asthma was conducted. The objective was to refine current algorithms to identify moderate exacerbations in claims data and assess the refined algorithm's performance. Positive and negative predictive values (PPV and NPV) were assessed via chart review of 150 moderate exacerbations events and 50 patients without exacerbations. Sensitivity analyses assessed alternative algorithms and compared healthcare resource utilization (HRU) between algorithm-identified patients (claims group) and those confirmed by chart review (confirmed group) to have experienced a moderate exacerbation. RESULTS: Algorithm-identified moderate exacerbations were: visit of ≤1 day with an asthma exacerbation diagnosis OR visit of ≤1 day with selected asthma diagnoses AND ≥1 respiratory pharmacy claim, excluding systemic corticosteroids, within 14 days after the first claim. The algorithm's PPV was 42%; the NPV was 78%. HRU was similar for both groups. CONCLUSION: This algorithm identified potential moderate exacerbations from claims data; however, the modest PPV underscores its limitations in identifying moderate exacerbations, although performance was partially due to identification of previously unidentified severe exacerbations. Application of this algorithm in future claims-based studies may help quantify the economic burden of moderate and severe exacerbations in asthma when an algorithm identifying severe exacerbations is applied first.


Assuntos
Algoritmos , Asma , Progressão da Doença , Humanos , Asma/tratamento farmacológico , Asma/diagnóstico , Asma/economia , Estudos Retrospectivos , Masculino , Feminino , Pessoa de Meia-Idade , Adulto , Estados Unidos , Corticosteroides/uso terapêutico , Corticosteroides/administração & dosagem , Idoso , Administração por Inalação , Revisão da Utilização de Seguros , Agonistas de Receptores Adrenérgicos beta 2/uso terapêutico , Agonistas de Receptores Adrenérgicos beta 2/administração & dosagem , Estudos de Coortes , Adolescente , Adulto Jovem
7.
Respir Med ; 226: 107629, 2024 May.
Artigo em Inglês | MEDLINE | ID: mdl-38593885

RESUMO

INTRODUCTION: Despite adherence to inhaled corticosteroid/long-acting ß2-agonist (ICS/LABA) therapy, many patients with asthma experience moderate exacerbations. Data on the impact of moderate exacerbations on the healthcare system are limited. This study assessed the frequency and economic burden of moderate exacerbations in patients receiving ICS/LABA. METHODS: Retrospective, longitudinal study analyzed data from Optum's de-identified Clinformatics® Data Mart Database recorded between October 1, 2015, and December 31, 2019. Eligibility criteria included patients ≥18 years of age with ≥1 ICS/LABA claim and ≥1 medical claim for asthma in the 12 months pre-index (first ICS/LABA claim). Primary objectives included describing moderate exacerbation frequency, and associated healthcare resource utilization (HRU) and costs. A secondary objective was assessing the relationship between moderate exacerbations and subsequent risk of severe exacerbations. Patients were stratified by moderate exacerbation frequency in the 12 months post index. Moderate exacerbations were identified using a newly developed algorithm. RESULTS: In the first 12 months post index 61.6% of patients experienced ≥1 moderate exacerbation. Mean number of asthma-related visits was 4.1 per person/year and median total asthma-related costs was $3544. HRU and costs increased with increasing exacerbation frequency. Outpatient and inpatient visits accounted for a similar proportion of these costs. Moderate exacerbations were associated with an increased rate and risk of future severe exacerbations (incidence rate ratio, 1.56; hazard ratio, 1.51 [both p < 0.001]). CONCLUSIONS: This study highlighted that a high proportion of patients continue to experience moderate exacerbations despite ICS/LABA therapy and subsequently experience increased economic burden and risk of future severe exacerbations.


Assuntos
Corticosteroides , Asma , Efeitos Psicossociais da Doença , Progressão da Doença , Humanos , Asma/tratamento farmacológico , Asma/economia , Estudos Retrospectivos , Administração por Inalação , Corticosteroides/administração & dosagem , Corticosteroides/economia , Corticosteroides/uso terapêutico , Masculino , Feminino , Pessoa de Meia-Idade , Adulto , Estudos Longitudinais , Estados Unidos , Agonistas de Receptores Adrenérgicos beta 2/administração & dosagem , Agonistas de Receptores Adrenérgicos beta 2/economia , Agonistas de Receptores Adrenérgicos beta 2/uso terapêutico , Idoso , Custos de Cuidados de Saúde/estatística & dados numéricos , Adulto Jovem , Antiasmáticos/economia , Antiasmáticos/administração & dosagem , Antiasmáticos/uso terapêutico
8.
Mult Scler Relat Disord ; 86: 105595, 2024 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-38598952

RESUMO

INTRODUCTION: Continuously acquired smartphone keyboard interactions may be useful to monitor progression in multiple sclerosis (MS). We aimed to study the correlation between tapping speed (TS), measured as keys/s, and baseline disability scales in patients with MS. METHODS: Single-center prospective study in patients with MS. We passively assessed TS during first week, measured by an "in house" smartphone application. Reliability was assessed by intraclass correlation coefficient (ICC). Correlations between median and maximum keys/s of first week of assessment and baseline disability measures were explored. RESULTS: One-hundred three patients were included: 62.1 % women, with a median (IQR) age of 47 (40.4-54.8) years-old and an EDSS score of 3.0 (2.0-4.0). Distribution by MS subtypes was: 77.7 % relapsing-remitting MS (RRMS), 17.5 % secondary-progressive MS (SPMS) and 4.9 % primary-progressive MS (PPMS). ICC during first week was 0.714 (p < 0.00001). Both median and maximum keys/s showed a negative correlation with Expanded Disability Status Score, 9-hole peg test and timed 25-foot walk and a positive correlation with Processing Speed Test CogEval® raw and Z-score. Median and maximum keys/s were lower in patients diagnosed with SPMS than in RRMS. Both measures of tapping speed were associated with MS phenotype independently of age. CONCLUSION: TS measured through our application is reliable and correlates with baseline disability scales.


Assuntos
Esclerose Múltipla , Smartphone , Humanos , Feminino , Masculino , Pessoa de Meia-Idade , Adulto , Estudos Prospectivos , Esclerose Múltipla/fisiopatologia , Esclerose Múltipla/diagnóstico , Avaliação da Deficiência , Reprodutibilidade dos Testes , Progressão da Doença , Aplicativos Móveis , Esclerose Múltipla Crônica Progressiva/fisiopatologia , Esclerose Múltipla Crônica Progressiva/diagnóstico , Esclerose Múltipla Recidivante-Remitente/fisiopatologia , Esclerose Múltipla Recidivante-Remitente/diagnóstico
10.
Semin Arthritis Rheum ; 66: 152433, 2024 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-38513411

RESUMO

OBJECTIVE: Identifying participants who will progress to advanced stage in knee osteoarthritis (KOA) trials remains a significant challenge. Current tools, relying on total knee replacements (TKR), fall short in reliability due to the extraneous factors influencing TKR decisions. Acknowledging these limitations, our study identifies a critical need for a more robust metric to assess severe KOA. The end-stage KOA (esKOA) measure, which combines symptomatic and radiographic criteria, serves as a solid indicator. To enhance future trials that use esKOA as an endpoint, our study focuses on developing and validating a machine-learning tool to identify individuals likely to develop esKOA within 2 to 5 years. DESIGN: Utilizing the Osteoarthritis Initiative (OAI) data, we trained models on 3,114 participants and validated them with 606 participants for the right knee, and similarly for the left knee, with external validation from the Multicentre Osteoarthritis Study (MOST) involving 1,602 participants. We aimed to predict esKOA onset at 2-to-2.5 years and 4-to-5 years, defining esKOA by severe radiographic KOA with moderate/severe symptoms or mild/moderate radiographic KOA with persistent/intense symptoms. Our analysis considered 51 candidate predictors, including demographics, clinical history, physical examination, and X-ray evaluations. An online tool predicting esKOA progression, based on models with ten and nine predictors for the right and left knees, respectively, was developed. RESULTS: External validation (MOST) for the right knee at 2.5 years yielded an Area Under Curve (AUC) of 0.847 (95 % CI 0.811 to 0.882), and at 5 years, 0.853 (95 % CI 0.823 to 0.881); for the left knee at 2.5 years, AUC was 0.824 (95 % CI 0.782 to 0.857), and at 5 years, 0.807 (95 % CI 0.768 to 0.843). Models with fewer predictors demonstrated comparable performance. The online tool is available at: https://eskoa.shinyapps.io/webapp/. CONCLUSION: Our study unveils a robust, externally validated machine learning tool proficient in predicting the onset of esKOA over the next 2 to 5 years. Our tool can lead to more efficient KOA trials.


Assuntos
Progressão da Doença , Aprendizado de Máquina , Osteoartrite do Joelho , Humanos , Osteoartrite do Joelho/diagnóstico por imagem , Osteoartrite do Joelho/cirurgia , Feminino , Masculino , Pessoa de Meia-Idade , Idoso , Índice de Gravidade de Doença , Reprodutibilidade dos Testes
11.
Lancet Neurol ; 23(4): 344-381, 2024 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-38493795

RESUMO

BACKGROUND: Disorders affecting the nervous system are diverse and include neurodevelopmental disorders, late-life neurodegeneration, and newly emergent conditions, such as cognitive impairment following COVID-19. Previous publications from the Global Burden of Disease, Injuries, and Risk Factor Study estimated the burden of 15 neurological conditions in 2015 and 2016, but these analyses did not include neurodevelopmental disorders, as defined by the International Classification of Diseases (ICD)-11, or a subset of cases of congenital, neonatal, and infectious conditions that cause neurological damage. Here, we estimate nervous system health loss caused by 37 unique conditions and their associated risk factors globally, regionally, and nationally from 1990 to 2021. METHODS: We estimated mortality, prevalence, years lived with disability (YLDs), years of life lost (YLLs), and disability-adjusted life-years (DALYs), with corresponding 95% uncertainty intervals (UIs), by age and sex in 204 countries and territories, from 1990 to 2021. We included morbidity and deaths due to neurological conditions, for which health loss is directly due to damage to the CNS or peripheral nervous system. We also isolated neurological health loss from conditions for which nervous system morbidity is a consequence, but not the primary feature, including a subset of congenital conditions (ie, chromosomal anomalies and congenital birth defects), neonatal conditions (ie, jaundice, preterm birth, and sepsis), infectious diseases (ie, COVID-19, cystic echinococcosis, malaria, syphilis, and Zika virus disease), and diabetic neuropathy. By conducting a sequela-level analysis of the health outcomes for these conditions, only cases where nervous system damage occurred were included, and YLDs were recalculated to isolate the non-fatal burden directly attributable to nervous system health loss. A comorbidity correction was used to calculate total prevalence of all conditions that affect the nervous system combined. FINDINGS: Globally, the 37 conditions affecting the nervous system were collectively ranked as the leading group cause of DALYs in 2021 (443 million, 95% UI 378-521), affecting 3·40 billion (3·20-3·62) individuals (43·1%, 40·5-45·9 of the global population); global DALY counts attributed to these conditions increased by 18·2% (8·7-26·7) between 1990 and 2021. Age-standardised rates of deaths per 100 000 people attributed to these conditions decreased from 1990 to 2021 by 33·6% (27·6-38·8), and age-standardised rates of DALYs attributed to these conditions decreased by 27·0% (21·5-32·4). Age-standardised prevalence was almost stable, with a change of 1·5% (0·7-2·4). The ten conditions with the highest age-standardised DALYs in 2021 were stroke, neonatal encephalopathy, migraine, Alzheimer's disease and other dementias, diabetic neuropathy, meningitis, epilepsy, neurological complications due to preterm birth, autism spectrum disorder, and nervous system cancer. INTERPRETATION: As the leading cause of overall disease burden in the world, with increasing global DALY counts, effective prevention, treatment, and rehabilitation strategies for disorders affecting the nervous system are needed. FUNDING: Bill & Melinda Gates Foundation.


Assuntos
Transtorno do Espectro Autista , COVID-19 , Doenças Transmissíveis , Neuropatias Diabéticas , Nascimento Prematuro , Infecção por Zika virus , Zika virus , Feminino , Humanos , Recém-Nascido , Carga Global da Doença , Doenças Transmissíveis/epidemiologia , Fatores de Risco , Progressão da Doença , Saúde Global , COVID-19/epidemiologia , Anos de Vida Ajustados por Qualidade de Vida , Expectativa de Vida
12.
PLoS Med ; 21(3): e1004361, 2024 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-38512968

RESUMO

BACKGROUND: In Brazil, many individuals with tuberculosis (TB) do not receive appropriate care due to delayed or missed diagnosis, ineffective treatment regimens, or loss-to-follow-up. This study aimed to estimate the health losses and TB program costs attributable to each gap in the care cascade for TB disease in Brazil. METHODS AND FINDINGS: We constructed a Markov model simulating the TB care cascade and lifetime health outcomes (e.g., death, cure, postinfectious sequelae) for individuals developing TB disease in Brazil. We stratified the model by age, human immunodeficiency virus (HIV) status, drug resistance, state of residence, and disease severity, and developed a parallel model for individuals without TB that receive a false-positive TB diagnosis. Models were fit to data (adult and pediatric) from Brazil's Notifiable Diseases Information System (SINAN) and Mortality Information System (SIM) for 2018. Using these models, we assessed current program performance and simulated hypothetical scenarios that eliminated specific gaps in the care cascade, in order to quantify incremental health losses and TB diagnosis and treatment costs along the care cascade. TB-attributable disability-adjusted life years (DALYs) were calculated by comparing changes in survival and nonfatal disability to a no-TB counterfactual scenario. We estimated that 90.0% (95% uncertainty interval [UI]: 85.2 to 93.4) of individuals with TB disease initiated treatment and 10.0% (95% UI: 7.6 to 12.5) died with TB. The average number of TB-attributable DALYs per incident TB case varied across Brazil, ranging from 2.9 (95% UI: 2.3 to 3.6) DALYs in Acre to 4.0 (95% UI: 3.3 to 4.7) DALYs in Rio Grande do Sul (national average 3.5 [95% UI: 2.8 to 4.1]). Delayed diagnosis contributed the largest health losses along the care cascade, followed by post-TB sequelae and loss to follow up from TB treatment, with TB DALYs reduced by 71% (95% UI: 65 to 76), 41% (95% UI: 36 to 49), and 10% (95% UI: 7 to 16), respectively, when these factors were eliminated. Total health system costs were largely unaffected by improvements in the care cascade, with elimination of treatment failure reducing attributable costs by 3.1% (95% UI: 1.5 to 5.4). TB diagnosis and treatment of false-positive individuals accounted for 10.2% (95% UI: 3.9 to 21.7) of total programmatic costs but contributed minimally to health losses. Several assumptions were required to interpret programmatic data for the analysis, and we were unable to estimate the contribution of social factors to care cascade outcomes. CONCLUSIONS: In this study, we observed that delays to diagnosis, post-disease sequelae and treatment loss to follow-up were primary contributors to the TB burden of disease in Brazil. Reducing delays to diagnosis, improving healthcare after TB cure, and reducing treatment loss to follow-up should be prioritized to improve the burden of TB disease in Brazil.


Assuntos
Efeitos Psicossociais da Doença , Tuberculose , Adulto , Criança , Humanos , Anos de Vida Ajustados por Qualidade de Vida , Saúde Global , Brasil/epidemiologia , Tuberculose/diagnóstico , Tuberculose/tratamento farmacológico , Tuberculose/epidemiologia , Progressão da Doença , Carga Global da Doença
13.
J Huntingtons Dis ; 13(1): 91-101, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-38517798

RESUMO

Background: Perivascular spaces (PVS) are fluid-filled cavities surrounding small cerebral blood vessels. There are limited reports of enlarged PVS across the grey matter in manifest Huntington's disease (HD). Little is known about how PVS morphometry in the white matter may contribute to HD. Enlarged PVS have the potential to both contribute to HD pathology and affect the distribution and success of intraparenchymal and intrathecally administered huntingtin-lowering therapies. Objective: To investigate PVS morphometry in the global white matter across the spectrum of HD. Relationships between PVS morphometry and disease burden and severity measures were examined. Methods: White matter PVS were segmented on 3T T2 W MRI brain scans of 33 healthy controls, 30 premanifest HD (pre-HD), and 32 early manifest HD (early-HD) participants from the Vancouver site of the TRACK-HD study. PVS count and total PVS volume were measured. Results: PVS total count slightly increased in pre-HD (p = 0.004), and early-HD groups (p = 0.005), compared to healthy controls. PVS volume, as a percentage of white matter volume, increased subtly in pre-HD compared to healthy controls (p = 0.044), but not in early-HD. No associations between PVS measures and HD disease burden or severity were found. Conclusions: This study reveals relatively preserved PVS morphometry across the global white matter of pre-HD and early-HD. Subtle morphometric abnormalities are implied but require confirmation in a larger cohort. However, in conjunction with previous publications, further investigation of PVS in HD and its potential impact on future treatments, with a focus on subcortical grey matter, is warranted.


Assuntos
Doença de Huntington , Substância Branca , Humanos , Doença de Huntington/complicações , Substância Branca/diagnóstico por imagem , Substância Branca/patologia , Progressão da Doença , Imageamento por Ressonância Magnética , Substância Cinzenta/diagnóstico por imagem , Encéfalo/diagnóstico por imagem , Encéfalo/patologia
14.
Methods Mol Biol ; 2785: 311-320, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-38427202

RESUMO

Cognitive testing is an essential part of clinical diagnostics and clinical trials in Alzheimer's disease. Digital cognitive tests hold a great opportunity to provide more versatile and cost-efficient patient pathways through flexible testing including at home. In this work, we describe a web-based cognitive test, cCOG, that can be used in screening, differential diagnosis, and monitoring the progression of neurodegenerative diseases.


Assuntos
Doença de Alzheimer , Disfunção Cognitiva , Humanos , Disfunção Cognitiva/diagnóstico , Doença de Alzheimer/diagnóstico , Diagnóstico Diferencial , Cognição , Internet , Biomarcadores , Progressão da Doença
15.
CPT Pharmacometrics Syst Pharmacol ; 13(5): 891-903, 2024 May.
Artigo em Inglês | MEDLINE | ID: mdl-38539027

RESUMO

Duchenne muscular dystrophy (DMD) is a rare X-linked recessive disorder characterized by loss-of-function mutations in the gene encoding dystrophin. These mutations lead to progressive functional deterioration including muscle weakness, respiratory insufficiency, and musculoskeletal deformities. Three-dimensional gait analysis (3DGA) has been used as a tool to analyze gait pathology through the quantification of altered joint kinematics, kinetics, and muscle activity patterns. Among 3DGA indices, the Gait Profile Score (GPS), has been used as a sensitive overall measure to detect clinically relevant changes in gait patterns in children with DMD. To enhance our understanding of the clinical translation of 3DGA, we report here the development of a population nonlinear mixed-effect model that jointly describes the disease progression of the 3DGA index, GPS, and the functional endpoint, North Star Ambulatory Assessment (NSAA). The final model consists of a quadratic structure for GPS progression and a linear structure for GPS-NSAA correlation. Our model was able to capture the improvement in function in GPS and NSAA in younger subjects, as well as the decline of function in older subjects. Furthermore, the model predicted NSAA (CFB) at 1 year reasonably well for DMD subjects ≤7 years old at baseline. The model tended to slightly underpredict the decline in NSAA after 1 year for those >7 years old at baseline, but the prediction summary statistics were well maintained within the standard deviation of observed data. Quantitative models such as this may help answer clinically relevant questions to facilitate the development of novel therapies in DMD.


Assuntos
Progressão da Doença , Marcha , Distrofia Muscular de Duchenne , Humanos , Distrofia Muscular de Duchenne/fisiopatologia , Distrofia Muscular de Duchenne/genética , Criança , Estudos Longitudinais , Pré-Escolar , Masculino , Adolescente , Análise da Marcha/métodos
16.
Alzheimers Res Ther ; 16(1): 54, 2024 Mar 09.
Artigo em Inglês | MEDLINE | ID: mdl-38461266

RESUMO

BACKGROUND: Due to the heterogeneity among patients with Mild Cognitive Impairment (MCI), it is critical to predict their risk of converting to Alzheimer's disease (AD) early using routinely collected real-world data such as the electronic health record data or administrative claim data. METHODS: The study used MarketScan Multi-State Medicaid data to construct a cohort of MCI patients. Logistic regression with tree-guided lasso regularization (TGL) was proposed to select important features and predict the risk of converting to AD. A subsampling-based technique was used to extract robust groups of predictive features. Predictive models including logistic regression, generalized random forest, and artificial neural network were trained using the extracted features. RESULTS: The proposed TGL workflow selected feature groups that were robust, highly interpretable, and consistent with existing literature. The predictive models using TGL selected features demonstrated higher prediction accuracy than the models using all features or features selected using other methods. CONCLUSIONS: The identified feature groups provide insights into the progression from MCI to AD and can potentially improve risk prediction in clinical practice and trial recruitment.


Assuntos
Doença de Alzheimer , Disfunção Cognitiva , Humanos , Imageamento por Ressonância Magnética/métodos , Aprendizado de Máquina , Doença de Alzheimer/diagnóstico , Medicaid , Disfunção Cognitiva/diagnóstico , Progressão da Doença
17.
PLoS One ; 19(3): e0299401, 2024.
Artigo em Inglês | MEDLINE | ID: mdl-38478491

RESUMO

OBJECTIVES: Routine screening for chronic kidney disease (CKD) could enable timely interventions to slow down disease progression, but currently there are no clinical guidelines for screening. We aim to evaluate the cost-effectiveness of screening for CKD using a novel analytical tool based on a cumulative sum statistic of estimated glomerular filtration rate (CUSUMGFR). METHODS: We developed a microsimulation model that captured CKD disease progression, major complications, patients' awareness, and treatment adherence for a nationally representative synthetic cohort of age ≥ 30 years in the United States. In addition to the status quo with no screening, we considered four CUSUMGFR-based universal screening policies by frequency (annual or biennial) and starting age (30 or 60 years), and two targeted annual screening policies for patients with hypertension and diabetes, respectively. For each policy, we evaluated the total discounted disability-adjusted life years (DALYs) and direct health costs over a lifetime horizon and estimated the incremental cost-effectiveness ratio (ICER). We further performed one-way and probabilistic sensitivity analyses to assess the impact of parameter uncertainty. RESULTS: Compared with the status quo, all the CUSUMGFR-based screening policies were cost-effective under the willingness-to-pay (WTP) range of $50,000 -$100,000, with the estimated incremental cost-effectiveness ratios (ICERs) ranging from $15,614/DALYs averted to $54,373/DALYs averted. Universal annual screening with starting age of 30 was the non-dominated policy on the cost-effectiveness frontier under the WTP of approximately $25,000. Adding more recent treatment option of sodium-glucose cotransporter-2 (SGLT2) inhibitors to the treatment regimen was found to be cost-saving. Among the most influential model parameters, variation in the CKD progression rate, adherence, and testing cost resulted in the highest variability in model outcomes. CONCLUSIONS: CUSUMGFR-based screening policies for CKD are highly cost-effective in identifying patients at risk of end stage kidney disease in early stages of CKD. Given its simple requirement of a basic blood test, the CUSUMGFR-based screening can be easily incorporated into clinical workflow for disease monitoring and prevention.


Assuntos
Insuficiência Renal Crônica , Humanos , Estados Unidos , Adulto , Pessoa de Meia-Idade , Análise Custo-Benefício , Insuficiência Renal Crônica/complicações , Taxa de Filtração Glomerular , Programas de Rastreamento/métodos , Progressão da Doença , Anos de Vida Ajustados por Qualidade de Vida
18.
Dtsch Med Wochenschr ; 149(7): e38-e46, 2024 Mar.
Artigo em Alemão | MEDLINE | ID: mdl-38479416

RESUMO

BACKGROUND: Patients at increased risk of inadequate immune response to COVID-19 vaccinations due to their underlying disease or therapy are potentially vulnerable to severe COVID-19 courses. The aim is to assess the population size, clinical courses and hospitalization costs of these patients in Germany. METHODS: This retrospective cohort study is based on extrapolations of a representative sample of statutory health insurance (SHI) claims data from 2020. Clinical COVID-19 courses, hospitalization costs and durations are compared between the insured group at increased risk for inadequate immune response to COVID-19 vaccinations (risk group) and the insured group without this risk. RESULTS: There are approximately 1.82 million SHI-insured individuals in the risk group, of whom an estimated 240 000 insured individuals do not develop a humoral immune response after 3 COVID-19 vaccinations. The risk group shows higher proportions with COVID-19 (relative risk [RR] 1.21; 95 % confidence interval [95 % CI] 1.20-1.23), hospitalizations for COVID-19 (RR 3.40; 95 % CI 3.33-3.48), hospitalizations for COVID-19 with intensive care treatment (RR 1.36; 95 % CI 1.30-1.42), and mortality (RR 5.14; 95 % CI 4.97-5.33) compared with the group without risk. In addition, hospitalizations in the risk group are on average 18 % longer (15.36 days vs. 13.00 days) and 19 % more expensive (12 371 € vs. 10 410 €). Expected hospitalization costs in the risk group are four times greater than in the group without risk (4115 € vs. 1017 €). CONCLUSIONS: The risk group is vulnerable to COVID-19 and requires additional resources in the German hospital sector. This results in a need for further protective measures. Further studies are needed to evaluate the impact of different viral variants, active and passive immunizations, and therapies on clinical COVID-19 courses and their costs.


Assuntos
COVID-19 , Humanos , Estudos Retrospectivos , Hospitalização , Fatores de Risco , Progressão da Doença , Alemanha/epidemiologia , Custos de Cuidados de Saúde
19.
NPJ Prim Care Respir Med ; 34(1): 1, 2024 Mar 07.
Artigo em Inglês | MEDLINE | ID: mdl-38453930

RESUMO

Medication adherence is vital for patients suffering from Chronic Obstructive Pulmonary Disease (COPD) to mitigate long-term consequences. The impact of poor medication adherence on inferior outcomes like exacerbations leading to hospital admissions is yet to be studied using real-world data. Using Swiss claims data from 2015-2020, we group patients into five categories according to their medication possession ratio. By employing a logistic regression, we quantify each category's average treatment effect of the medication possession ratio on hospitalized exacerbations. 13,557 COPD patients are included in the analysis. Patients with high medication adherence (daily medication reserve of 80% to 100%) are 51% less likely to incur exacerbation following a hospital stay than patients with the lowest medication adherence (daily medication reserve of 0% to 20%). The study shows that medication adherence varies strongly among Swiss COPD patients. Furthermore, high medication adherence immensely decreases the risk of hospitalized exacerbations.


Assuntos
Doença Pulmonar Obstrutiva Crônica , Humanos , Suíça , Estudos Retrospectivos , Hospitalização , Adesão à Medicação , Seguro Saúde , Progressão da Doença
20.
Gut ; 73(6): 897-909, 2024 May 10.
Artigo em Inglês | MEDLINE | ID: mdl-38553042

RESUMO

Barrett's oesophagus is the only known precursor to oesophageal adenocarcinoma, a cancer with very poor prognosis. The main risk factors for Barrett's oesophagus are a history of gastro-oesophageal acid reflux symptoms and obesity. Men, smokers and those with a family history are also at increased risk. Progression from Barrett's oesophagus to cancer occurs via an intermediate stage, known as dysplasia. However, dysplasia and early cancer usually develop without any clinical signs, often in individuals whose symptoms are well controlled by acid suppressant medications; therefore, endoscopic surveillance is recommended to allow for early diagnosis and timely clinical intervention. Individuals with Barrett's oesophagus need to be fully informed about the implications of this diagnosis and the benefits and risks of monitoring strategies. Pharmacological treatments are recommended for control of symptoms, but not for chemoprevention. Dysplasia and stage 1 oesophageal adenocarcinoma have excellent prognoses, since they can be cured with endoscopic or surgical therapies. Endoscopic resection is the most accurate staging technique for early Barrett's-related oesophageal adenocarcinoma. Endoscopic ablation is effective and indicated to eradicate Barrett's oesophagus in patients with dysplasia. Future research should focus on improved accuracy for dysplasia detection via new technologies and providing more robust evidence to support pathways for follow-up and treatment.


Assuntos
Adenocarcinoma , Esôfago de Barrett , Neoplasias Esofágicas , Esôfago de Barrett/terapia , Esôfago de Barrett/patologia , Esôfago de Barrett/diagnóstico , Humanos , Neoplasias Esofágicas/terapia , Neoplasias Esofágicas/patologia , Neoplasias Esofágicas/diagnóstico , Neoplasias Esofágicas/etiologia , Adenocarcinoma/patologia , Adenocarcinoma/terapia , Adenocarcinoma/diagnóstico , Esofagoscopia/métodos , Estadiamento de Neoplasias , Progressão da Doença , Fatores de Risco , Lesões Pré-Cancerosas/patologia , Lesões Pré-Cancerosas/terapia , Lesões Pré-Cancerosas/diagnóstico
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