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Validation of a Portuguese Version of the Four-Item Migraine Interictal Burden Scale.
No full textBackground: Migraine is the most common neurological disorder and the second leading cause of disability. While the impact of migraine on daily life is often assessed by the frequency and intensity of attacks, the impairment between episodes is frequently overlooked. The four-item Migraine Interictal Burden Scale (MIBS-4) is a tool that can provide information and help in the evaluation of interictal disability.
Objective: To adapt and validate a translated version of the MIBS-4 for the Portuguese population.
Methods: A prospective, multicentric observational study was conducted between July 2023 and February 2024 in six tertiary headache centers across Portugal. The MIBS-4 was translated and back-translated to obtain a consensus version. The scale was administered to 459 patients with migraine, and its psychometric properties were assessed through correlations with established measures, including the Migraine Disability Assessment Scale (MIDAS), 12-item Short Form Survey (SF-12), and Hospital Anxiety and Depression Scale (HADS).
Results: A total of 459 patients were evaluated, including 430 women (93.7%), with an average age of 45.06 years. The MIBS-4 demonstrated good internal consistency with a Cronbach's alpha of 0.84 (95% confidence interval 0.81-0.86) and significant correlations with the MIDAS (r = 0.304, p < 0.001), SF-12 (r = 0.483, p < 0.001), and HADS (r = 0.211, p < 0.001), confirming its reliability and construct validity.
Conclusion: This analysis showed that the Portuguese version of the MIBS-4 showed identical psychometric properties to the original scale and can effectively assess interictal disability in patients with migraine in Portuguese speaking populations. Its validation provides a valuable tool for clinical practice and research, enabling improved assessment of migraine burden and contributing to better patient care in lusophone countries
O Strain Longitudinal Global do Ventrículo Esquerdo Está Associado com as Pressões de Enchimento e Débito Cardíaco no Contexto Ambulatório: Lições do CardioMEMS™
No full textIntroduction and objectives: Left ventricular global longitudinal strain (LVGLS) is an indicator of myocardial function in patients with heart failure with reduced ejection fraction (HFrEF) and preserved ejection fraction (HFpEF). Nevertheless, it is not clear whether LVGLS correlates with filling pressures and cardiac output (CO) in an ambulatory setting. We aimed to assess whether LVGLS is associated with invasive pulmonary artery pressures (PAP) and CO in outpatients using the invasive remote monitoring CardioMEMS™ system.
Methods: This single-center, prospective observational study included patients with HFrEF undergoing remote monitoring using the CardioMEMS™ system, between January 2020 and December 2022. Repeated transthoracic echocardiography (TTE) studies were performed in each patient and invasive hemodynamic data were obtained during the TTE studies using the CardioMEMS™ system. Univariate and multivariate models were used to assess the potential association between LVGLS and invasive PAP and CO.
Results: Twelve patients were included and 46 TTE studies were analyzed. LVGLS was correlated with diastolic (d) PAP (r=0.403, p=0.041) and CO (r=-0.426, p=0.039) in the univariate analysis. In multivariate models, LVGLS was an independent predictor of dPAP and CO, but not mean PAP or systolic PAP. The variation of LVGLS between TTE studies was correlated with the variation of dPAP during follow-up (r=0.60, p=0.017).
Conclusions: In a cohort of HFrEF patients under invasive hemodynamic remote monitoring, LVGLS was independently associated with invasive filling pressures and CO, in an outpatient setting. These findings reinforce the value of LVGLS for the management of outpatients with HFrEF.Introdução e objetivos:
O strain longitudinal global do ventrículo esquerdo (SLGVE) é um indicador de função miocárdica em doentes com insuficiência cardíaca com fração de ejeção reduzida (ICFEr) e com fração de ejeção preservada (ICFEp). No entanto, não é totalmente claro se o SLGVE se correlaciona com as pressões de enchimento e débito cardíaco (DC) no contexto ambulatório. Avaliámos a associação entre o SLGVE com as pressões (P) invasivas da artéria pulmonar (AP) e DC utilizando o sistema CardioMEMS™ de monitoração remota invasiva.
Métodos:
Este estudo unicêntrico, observacional e prospetivo incluiu doentes com ICFEr monitorados remotamente com o sistema CardioMEMS™, entre janeiro 2020 e dezembro 2022. Ecocardiograma transtorácicos (ETT) seriados foram executados em cada doente e os dados invasivos hemodinâmicos foram obtidos no momento de execução do ETT, usando o sistema CardioMEMS™. Modelos uni- e multivariados foram usados para avaliar a potencial associação entre o SLGVE e PAP e DC invasivos.
Resultados:
Doze doente foram incluídos e 46 ETT foram analisados. O SLGVE correlacionou-se com a PAP diastólica (r = 0,403, p = 0,041) e DC (r = −0,426, p = 0,039) na análise univariada. Nos modelos multivariados, o SLGVE foi um preditor independente da PAP diastólica e DC, mas não da PAP média e PAP sistólica. A variação do SLGVE entre ETT correlacionou-se com uma variação correspondente de PAP diastólica durante o seguimento (r = 0,60, p = 0,017).
Conclusões:
Numa coorte de doentes com ICFEr sob monitoração remota invasiva, o SLGVE está associado de forma independente com as pressões de enchimento e DC, no contexto ambulatório. Estes achados reforçam o valor do SLGVE na gestão dos doentes ambulatórios com ICFEr
Efficacy and Safety of IncobotulinumtoxinA in Older Patients with Upper Limb Spasticity: a Pooled Analysis.
No full text: The aim of this study was to compare the efficacy and safety of a single cycle of incobotulinumtoxinA versus placebo in pooled data from older patients (aged ≥65 years) with upper limb spasticity (ULS). : This study was a post hoc analysis of pooled data from seven prospective, multicenter, phase II or III trials of incobotulinumtoxinA in adult patients aged ≥65 years from across the world with post-stroke ULS or upper and lower limb spasticity, including a subgroup with moderate-to-severe ULS. Changes from baseline in ULS severity were evaluated using the (modified) Ashworth Scale across different spasticity patterns at 4 and 12 weeks after incobotulinumtoxinA injection. : In 267 older patients with ULS, including a subgroup of 207 with moderate-to-severe ULS, all ULS patterns statistically analyzed (elbow flexion, thumb-in-palm, clenched fist, wrist flexion, and pronated forearm) were improved more by incobotulinumtoxinA than placebo at week 4 ( < 0.05). For most of these patterns, the difference remained significant at week 12 ( < 0.05). IncobotulinumtoxinA was generally well tolerated. : This study, which analyzed data from the largest cohort of older patients in the literature, provides information regarding the use of incobotulinumtoxinA in ULS, the efficacy and favorable safety profile of incobotulinumtoxinA for the treatment of ULS in older patients, particularly in those with moderate-to-severe spasticity, was confirmed
Global Inequities in Diabetes Technology and Insulin Access and Glycemic Outcomes.
No full textImportance: Advanced diabetes technologies such as continuous glucose monitoring (CGM), continuous subcutaneous insulin infusion (insulin pumps [CSII]), and glucometers alongside insulin access represent the criterion standard for managing type 1 diabetes (T1D) in children. Global disparities in their access and reimbursement may be associated with glycemic outcomes.
Objective: To describe how accessibility and reimbursement of advanced diabetes technologies and insulin are associated with glycated hemoglobin (HbA1c) levels in centers participating in the SWEET initiative, an international pediatric diabetes registry.
Design, setting, and participants: This global multicenter cross-sectional study collected data from 81 centers in 56 countries. Web-based questionnaires were distributed to representatives of all 121 pediatric diabetes centers participating in the SWEET initiative from March 1 to May 31, 2024, and used to map accessibility of and reimbursement for CGM, CSII, glucometers, and insulin. Reimbursement data were compared with HbA1c levels using the SWEET Study dataset. Participants included 42 349 children with T1D.
Exposures: Responses were categorized into 4 groups based on the extent of reimbursement for diabetes technologies and insulin.
Main outcomes and measures: Mean HbA1c levels across centers calculated from measurements current as of December 31, 2023, analyzed by categories of accessibility of and reimbursement for diabetes technologies and insulin.
Results: Data collected from 81 of 121 SWEET centers (67%) across 56 countries included HbA1c levels from 42 349 children with T1D (22 021 male [52%]; mean [SD] age, 14.3 [4.4] years; mean [SD] diabetes duration, 6.0 [4.2] years). Universal access with complete reimbursement for all technologies and insulin was reported by 32 centers from 19 countries, while 8 countries reported no reimbursement for any technologies or insulin. Centers with full reimbursement for CSII, CGM, glucometers, and insulin showed mean HbA1c levels of 7.62% (95% CI, 7.59%-7.64%) to 7.75% (95% CI, 7.73%-7.77%) compared with 9.65% (95% CI, 9.55%-9.71%) to 10.49% (95% CI, 10.40%-10.58%) in centers with no reimbursement and/or no availability (P < .001 for all items).
Conclusions and relevance: This cross-sectional study found that HbA1c levels were associated with the accessibility of modern diabetes technologies and insulin. Efforts to ensure universal accessibility are required to reduce global inequities and glycemic outcomes for children with T1D.
Biologic Therapies and Quality of Life in Pediatric Patients with Asthma: A Systematic Review.
No full textBackground/Objectives: Pediatric asthma is the most prevalent chronic respiratory condition in children and adolescents worldwide and remains a major contributor to morbidity, school absenteeism, and the use of integrated healthcare services. The main goal of this systematic review is to synthesize the available evidence about the impact of biologic therapies on the quality of life in the pediatric population (children and adolescents) with asthma.
Methods: This systematic review followed the PRISMA guidelines. A comprehensive search was performed across PubMed, Scopus, and Web of Science for articles published between 2015 and 2025 in English, Portuguese, or Spanish. Studies were eligible if they included pediatric patients (<18 years) with asthma receiving biologic therapies (e.g., omalizumab, mepolizumab, dupilumab) and reported health-related quality of life (HRQoL) outcomes using validated instruments. Article selection followed PICOS criteria and excluded reviews, case reports, and editorials. Risk of bias was assessed using the Mixed Methods Appraisal Tool.
Results: A total of 576 articles were found and screened, and 8 studies were selected. The characteristics of the studies highlighted the involved countries, study design, number of participants, conditions, type of biologic intervention, duration, and follow-up. In addition, the importance of biologic therapies in patients' HRQoL was presented. All eight included studies reported statistically significant improvements in HRQoL using appropriate scales.
Conclusions: The studies reinforced the importance of biologic therapies to improve HRQoL in both patients and families/caregivers. Implications for health promotion, in particular, greater involvement of integrated healthcare comprising health professionals, family, school contexts, and the community, are discussed
ANCA Renal Risk Score in a Portuguese Cohort: Insights and Comparison with Berden Classification
No full textIntroduction: Antineutrophil cytoplasmic antibody (ANCA)‑associated vasculitis (AAV) is a rare cause of glomerulonephritis. Despite advances in immunosuppressive therapies, AAV with renal involvement often leads to poor renal outcomes and a high‑risk of end‑stage renal disease (ESRD). Berden classification categorizes renal histology in four classes, but performance varies across cohorts. A lack of precise findings of prognostic value led to development of ANCA Renal Risk Score (ARRS). We assessed ARRS predictive value for renal survival in a Portuguese cohort and compared it to Berden classification.
Methods: Observational and retrospective study analysing cases from native AAV kidney biopsies at a Portuguese
histomorphology centre (2004‑2023). Demographical data, percentage of normal glomeruli, interstitial fibrosis/tubular
atrophy and estimated glomerular filtration rate (eGFR) were recorded. Samples were categorized by Berden class and
ARRS. Descriptive and comparative analyses were performed.
Results: One hundred fifty eight patients, 53.8 % male, with mean age of 67.1 ± 14.1 years. Median ARRS was 6.5 ± 3.6,
with 10.8%, 44.3% and 44.9% being low, intermediate, and high‑risk, respectively. Overall mean time to dialysis was 20
months, renal survival at 36 months was 92.9%, 63.6% and 29.7% in low, intermediate and high‑risk groups. Patient
survival did not differ between groups. ROC curves showed statistical significance for both scores, with ARRS having the highest AUC (AUC <0.72, p<0.001 vs 0.65, p=0.03).
Conclusion: ARRS was effective in predicting renal survival in this cohort, outperforming Berden classification in determining progression to ESRD at 36 months. Incorporating serum creatinine, treatment modalities and patient comorbidities could further improve ARRS predictive value
Impact of Anatomical Resection on Non-transplantable Recurrence Among Patients with Hepatocellular Carcinoma: An International Multicenter Inverse Probability of Treatment Weighting Analysis.
No full textBackground: Among patients with hepatocellular carcinoma (HCC), the impact of anatomic resection (AR) versus non-anatomic resection (NAR) on non-transplantable recurrence (NTR) remains poorly defined. We sought to compare the risk of NTR among patients treated with AR versus NAR as the primary surgical strategy for HCC.
Patients and methods: Patients with HCC within Milan criteria who underwent curative-intent resection between 2000 and 2020 were identified from an international multi-institutional database. The inverse probability of treatment weighting (IPTW) method was utilized to compare short- and long-term outcomes among patients undergoing AR versus NAR.
Results: Among 1038 patients, 747 (72.0%) patients underwent AR, while 291 (28.0%) patients underwent NAR. After IPTW adjustment, patients who underwent AR had better 5-year recurrence-free survival than individuals treated with NAR (63.9 vs. 52.0%; hazard ratio [HR] 0.78; 95% confidence interval [CI] 0.62-0.99); however, there was no difference in 5-year overall survival (80.2 vs. 75.6%; HR 0.76; 95% CI 0.55-1.05). Notably, individuals who underwent AR were less likely to have a NTR versus individuals treated with NAR (3-year NTR 9.8 vs. 14.4%; HR 0.62; 95% CI 0.40-0.96). In particular, AR was associated with a lower risk of NTR among patients with a medium tumor burden score (TBS) (HR 0.53; 95% CI 0.28-0.99), while the benefit among patients with a low TBS was less pronounced (HR 0.73; 95% CI 0.40-1.32).
Conclusions: AR was associated with a lower risk of NTR and improved recurrence-free survival (RFS) among patients with HCC, especially individuals with higher TBS. An anatomically defined surgical approach should be strongly considered in patients with a higher HCC tumor burden
Practical and Multidisciplinary Review on Wilson Disease: The Portuguese Perspective.
No full textWilson disease (WD) is a genetic disorder of copper metabolism caused by mutations in the ATP7B gene resulting in toxic copper accumulation in several organs. WD can manifest as liver disease, a progressive neurological disorder, a psychiatric illness, or a combination of these. Other clinical manifestations can also occur. Diagnosis is challenging and typically requires a range of biochemical tests, imaging, genetic testing for ATP7B, and/or liver biopsy. WD is treatable with chelating agents, such as d-penicillamine and trientine, and/or zinc salts alongside with dietary copper restriction. Liver transplantation may be indicated in WD patients with severe hepatic disease, and cautiously considered in patients with neurological WD. Treatment success highly depends on patient adherence and treatment persistence. Therefore, effective interventions for improving patient adherence and close monitoring are key for preventing WD progression. In Portugal, there are no reference centers for WD, and patients are dispersed across numerous medical specialists. This review aimed to summarize the most recent and relevant information for the diagnosis, treatment, and monitoring of WD in Portugal, as well as possible interventions for stimulating adherence to treatment.A doença de Wilson é uma doença genética do metabolismo do cobre, causada por mutações no gene , que levam à acumulação tóxica de cobre em diversos órgãos. A doença de Wilson pode manifestar-se como doença hepática, perturbação neurológica progressiva, doença psiquiátrica ou como uma combinação destas patologias. Outras manifestações clínicas também podem ocorrer. O diagnóstico é complexo e normalmente requer a combinação de análises bioquímicas, imagiologia, testes genéticos para o gene e/ou biópsia hepática. A doença de Wilson é tratável com agentes quelantes, como a d-penicilamina e a trientina, e/ou sais de zinco, em conjunto com uma dieta com baixo teor de cobre. O transplante de fígado pode ser indicado em doentes com doença hepática grave, e deve ser cuidadosamente considerado em doentes com manifestações predominantemente neurológicas. O sucesso do tratamento é altamente dependente da adesão do doente e da persistência no tratamento. Portanto, intervenções eficazes para melhorar a adesão do doente ao tratamento, bem como a monitorização rigorosa, são cruciais para prevenir a progressão da doença de Wilson. Em Portugal não existem centros de referência para a doença de Wilson e os doentes encontram-se dispersos por numerosos especialistas médicos. Este artigo de revisão pretende reunir informação recente e relevante para o diagnóstico, tratamento e monitorização da doença de Wilson em Portugal, assim como possíveis intervenções para estimular a adesão ao tratamento
International Benchmark Values for Robotic Right Hepatectomy: Multicenter Study From 22 Expert Centers.
No full textObjective: This study aimed to identify benchmark values for robotic right hepatectomy (RH) based on a low-risk cohort treated at expert centers.
Background: Robotic liver surgery is emerging as a preferred minimally invasive approach to the liver. To enable conclusive comparisons with the standard open or laparoscopic approaches, reference values are needed.
Methods: Outcomes from consecutive patients undergoing robotic RH for malignant or benign indications at 22 international expert centers between 2018 and 2024 were analyzed. Low-risk, benchmark patients were without significant comorbidities such as portal hypertension, Child B cirrhosis, cardiac disease, chronic pulmonary disease, and renal failure. Patients undergoing robotic RH for donor hepatectomy were excluded. Fifteen reference values were derived from the 75th or the 25th percentile of the median values of all centers. Reference values were compared with a laparoscopic cohort from 4 centers and published benchmark values for laparoscopic and open RH.
Results: Of 357 patients, 172 (48%) qualified as the benchmark cohort. The main indications were hepatocellular carcinoma (31%) and colorectal liver metastases (27%). Reference values included: operative time (≤476 min), conversion rate (≤8.2%), bile leak (≤15.4%), major complications (≤23.1%), and comprehensive complication index at 90 days (≤15.6). Robotic RH compared favorably to a multinational cohort series of laparoscopic RH with lower conversion (10.0% vs ≤8.2%) and R1 rate (10.9% vs ≤0%). Compared to open robotic hepatectomy, cutoffs for major complications (≤50.0% vs ≤23.1%) and liver failure (≤22.0% vs ≤2.7%) were lower for robotic right hepatectomies.
Conclusion: This international benchmark study on robotic right hepatectomy (RRH) demonstrates that the robotic approach provides advantages compared with laparoscopic and open RH. RRH can be expected to become the minimally invasive approach of choice for tumors in the right liver
