Demographic, clinical, and treatment characteristics of the juvenile primary fibromyalgia syndrome cohort enrolled in the Childhood Arthritis and Rheumatology Research Alliance Legacy Registry.

BackgroundTo describe the demographic, clinical, and treatment characteristics of youth diagnosed with juvenile primary fibromyalgia syndrome (JPFS) who are seen in pediatric rheumatology clinics.MethodsInformation on demographics, symptoms, functioning, and treatments recommended and tried were obtained on patients with JPFS as part of a multi-site patient registry (the Childhood Arthritis and Rheumatology Research Alliance Legacy Registry). Data were summarized using descriptive statistics. In a subset of patients completing registry follow-up visits, changes in symptoms, pain, and functioning were evaluated using growth modeling.ResultsOf the 201 patients with JPFS enrolled in the registry, most were Caucasian/White (85%), non-Hispanic (83%), and female (84%). Ages ranged from 9 to 20 years (M = 15.4 + 2.2). The most common symptoms reported were widespread musculoskeletal pain (91%), fatigue (84%), disordered sleep (82%), and headaches (68%). Pain intensity was rated as moderate to severe (M = 6.3 + 2.4/10). Scores on measures of functioning indicated mild to moderate impairment, with males observed to report significantly greater impairments. For the 37% of the initial cohort having follow-up data available, indicators of function and well-being were found to either worsen over time or remain relatively unchanged.ConclusionsThe symptoms of JPFS remained persistent and disabling for many patients treated by pediatric rheumatologists. Further study appears warranted to elucidate gender differences in the impact of JPFS symptoms. Work also is needed to identify accessible and effective outpatient treatment options for JPFS that can be routinely recommended or implemented by pediatric rheumatology providers

Clinical phenotypes and biologic treatment use in juvenile dermatomyositis-associated calcinosis

Abstract Background Few risk factors have been identified for the development of calcinosis among patients with Juvenile Dermatomyositis, and currently no clinical phenotype has been associated with its development. We analyzed a large database of patients to further elucidate any relationships among patients with and without calcinosis. Method The CARRA legacy registry recruited pediatric rheumatology patients from 55 centers across North America from 2010 through 2014, including over 650 subjects with Juvenile Dermatomyositis. We compared the demographic characteristics, clinical disease features and treatment histories of those with and without calcinosis using univariate and multivariate logistic regression. Results Of the 631 patients included in the analysis, 84 (13%) had a current or prior history of calcinosis. These patients were statistically more likely to have longer durations of disease prior to diagnosis and treatment, have lipodystrophy and joint contractures, and to have received intravenous immune globulin or rituximab as treatments. Conclusions Calcinosis is found more often in patients with prolonged active disease, severe disease, and certain clinical features such as lipodystrophy and joint contractures. When these factors are combined with other known associations and predictors, groups of at-risk patients can be more effectively identified, treated and studied to improve overall outcomes

Predictors of long-term survival after percutaneous aortic valvuloplasty: Report of the mansfield scientific balloon aortic valvuloplasty registry

AbstractPercutaneous balloon aortic valvuloplasty was used to prospectively treat 492 elderly, symptomatic, nonsurgical patients suffering from severe aortic stenosis in 27 centers in North America and Europe. At 1 year the overall survival rate was 64% and the event-free survival rate (survival free of valve replacement or repeat valvuloplasty) was 43%. Clinical, catheterization and procedural variables were assessed to define prognostic variables.Univariate analysis revealed that patients who survived had a lesser frequency of previous myocardial infarction (2% versus 6%, p < 0.005), lower incidence of severe ventricular dysfunction (22% versus 48%, p < 0.001) and lower incidence of symptoms of heart failure (60% versus 75%, p < 0.02). History of angina (56% versus 45%, p = NS) and syncope (23% versus 16%, p = NS) were similar for both groups. Values obtained at cardiac catheterization that differed in survivors and nonsurvivors included lower pulmonary artery systolic pressure (43 ± 1 versus 54 ± 2 mm Hg, p < 0.001), lower mean pulmonary artery pressure (28 ± 1.0 versus 36 ± 1.0 mm Hg, p < 0.001) and larger initial valve area (0.52 ± 0.01 versus 0.47 ± 0.02 cm2, p = 0.006).Discriminate function analysis was performed to identify variables that independently predicted improved probability of survival. Eight variables were significantly and independently predictive. These included age, initial cardiac output, initial left ventricular systolic pressures, initial left ventricular end-diastolic pressures, presence of coronary artery disease, New York Heart Association dyspnea classification, number of balloon inflations and final valve area. From this analysis, patient survival probabilities at 1 year varying between 80% and 20% could be calculated. Although both initial and final severity of aortic stenosis were prognostically important on univariate analysis, discriminant function analysis revealed that only final severity of aortic stenosis was prognostic.These observations suggest that subgroups of patients with lower or higher mortality rates can be identified after balloon aortic valvuloplasty. The presence of left ventricular dysfunction, the presence of coronary artery disease and the use of multiple balloon inflations adversely affect long-term prognosis. Conversely, effective valvuloplasty provides modest improvement in survival probability

Exploring the structural relationship between interviewer and self-rated affective symptoms in Huntington’s disease

This study explores the structural relationship between self-report and interview measures of affect in Huntington’s disease. The findings suggest continued use of both to recognize the multidimensionality within a single common consideration of distress

Doentes Oncológicos com Síndrome Coronária Aguda Não Têm Maior Risco Hemorrágico Quando Comparados com Doentes com Características Similares - Uma Análise de Emparelhamento de Score de Propensão do Registo ProACS

Introduction: The management of acute coronary syndrome (ACS) in malignancy is challenging due to higher bleeding risk. Methods: We analyzed patients with cancer (active or in the previous five years) prospectively included in the ProACS registry between 2010 and 2019. Our aim was to assess safety (major bleeding, primary endpoint) and secondary efficacy endpoints (in-hospital mortality and combined in-hospital mortality, reinfarction and ischemic stroke) of ACS treatment. Propensity score matching analysis (1:1) was further performed to better understand predictors of outcomes. Results: We found 934 (5%) cancer patients out of a total of 18 845 patients with ACS. Cancer patients had more events: major bleeding (2.9% vs. 1.5%), in-hospital mortality (5.8% vs. 3.4%) and the combined endpoint (7.4% vs. 4.9%). The primary endpoint was related to cancer diagnosis (OR 1.97), previous bleeding (OR 7.09), hemoglobin level (OR 4.94), atrial fibrillation (OR 3.50), oral anticoagulation (OR 3.67) and renal dysfunction. Mortality and the combined secondary endpoint were associated with lower use of invasive coronary angiography and antiplatelet and neurohormonal blocker therapy. After propensity score matching (350 patients), there were no statistically significant differences in endpoints between the populations. Conclusion: Bleeding risk was not significant higher in the cancer population compared to patients with similar characteristics, nor were mortality or ischemic risk. The presence of cancer should not preclude simultaneous ACS treatment.info:eu-repo/semantics/publishedVersio

Atrial Fibrillation Better Care Pathway Adherent Care Improves Outcomes in Chinese Patients With Atrial Fibrillation

BACKGROUND: Atrial fibrillation (AF) is a complex disease associated with comorbidities and adverse outcomes. The Atrial fibrillation Better Care (ABC) pathway has been proposed to streamline the integrated and holistic approach to AF care. OBJECTIVES: This study sought to evaluate patients’ characteristics, incidence of adverse events, and impact on outcomes with ABC pathway–adherent management. METHODS: The study included consecutive AF patients enrolled in the nationwide, ChioTEAF registry (44 centers, 20 Chinese provinces from October 2014 to December 2018), with available data to evaluate the ABC criteria and on the 1-year follow-up. RESULTS: A total of 3,520 patients (mean age 73.1 ± 10.4 years, 43% female) were included, of which 1,448 (41.1%) were managed as ABC pathway adherent. The latter were younger and had comparable CHA(2)DS(2)-VASc and lower HAS-BLED (mean 71.7 ± 10.3 years of age vs 74.1 ± 10.4 years of age; P < 0.01; 3.54 ± 1.60 vs 3.44 ± 1.70; P = 0.10; and 1.95 ± 1.10 vs 2.12 ± 1.20; P < 0.01, respectively) scores compared with ABC-nonadherent patients. At 1-year follow-up, patients managed adherent to the ABC pathway had a lower incidence of the primary composite outcome of all-cause death or any thromboembolic event (1.5% vs 3.6%; P < 0.01) as compared with ABC-nonadherent patients. On multivariate analysis, ABC pathway–adherent care was independently associated with a lower risk of the composite endpoint (OR: 0.51; 95% CI: 0.31-0.84). CONCLUSIONS: Adherence to the ABC pathway for integrated care in a contemporary nationwide cohort of Chinese AF patients was suboptimal. Clinical management adherent to the ABC pathway was associated with better outcomes

Perinatal insults and neurodevelopmental disorders may impact Huntington's disease age of diagnosis

Introduction: The age of diagnosis of Huntington's disease (HD) varies among individuals with the same HTT CAG-repeat expansion size. We investigated whether early-life events, like perinatal insults or neurodevelopmental disorders, influence the diagnosis age. Methods: We used data from 13,856 participants from REGISTRY and Enroll-HD, two large international multicenter observational studies. Disease-free survival analyses of mutation carriers with an HTT CAG repeat expansion size above and including 36 were computed through Kaplan-Meier estimates of median time until an HD diagnosis. Comparisons between groups were computed using a Cox proportional hazard survival model adjusted for CAG-repeat expansion length. We also assessed whether the group effect depended on gender and the affected parent. Results: Insults in the perinatal period were associated with an earlier median age of diagnosis of 45.00 years (95%CI: 42.07–47.92) compared to 51.00 years (95%CI: 50.68–51.31) in the reference group, with a CAG-adjusted hazard ratio of 1.61 (95%CI: 1.26–2.06). Neurodevelopmental disorders were also associated with an earlier median age of diagnosis than the reference group of 47.00 years (95% CI: 43.38–50.62) with a CAG-adjusted hazard ratio of 1.42 (95%CI: 1.16–1.75). These associations did not change significantly with gender or affected parent. Conclusions: These results, derived from large observational datasets, show that perinatal insults and neurodevelopmental disorders are associated with earlier ages of diagnosis of magnitudes similar to the effects of known genetic modifiers of HD. Given their clear temporal separation, these early events may be causative of earlier HD onset, but further research is needed to prove causation

Understanding a woman's heart: Lessons from 14 177 women with acute coronary syndrome

INTRODUCTION: Coronary artery disease is becoming the leading cause of death in women in Western society. However, the available data shows that women are still underdiagnosed and undertreated with guideline-recommended secondary prevention therapy, leading to a significantly higher rate of in-hospital complications and in-hospital mortality. OBJECTIVE: The main objective of this work is to assess the approach to acute coronary syndrome (ACS) in Portugal, including form of presentation, in-hospital treatment and in-hospital complications, according to gender and in three different periods. METHODS: We performed an observational study with retrospective analysis of all patients included between 2002 and 2019 in the Portuguese Registry of Acute Coronary Syndromes (ProACS), a voluntary, observational, prospective, continuous registry of the Portuguese Society of Cardiology and the National Center for Data Collection in Cardiology. RESULTS: A total of 49 113 patients (34 936 men and 14 177 women) were included. Obesity, hypertension, diabetes (p<0.001 for all) and dyslipidemia (p=0.022) were all more prevalent in women, who were more frequently admitted for non-ST segment elevation ACS (p<0.001), and more frequently presented with atypical symptoms. Women had more time until needle and until reperfusion, which is less accessible to this gender (p<0.001). During hospitalization, women had a significantly higher risk of in-hospital mortality (OR 1.94 [1.78-2.12], p<0.001), major bleeding (OR 1.53 [1.30-1.80], p<0.001), heart failure (OR 1.87 [1.78-1.97], p<0.001), atrial fibrillation (OR 1.55 [1.36-1.77], p<0.001), mechanical complications (OR 2.12 [1.78-2.53], p<0.001), cardiogenic shock (OR 1.71 [1.57-1.87], p<0.001) and stroke (OR 2.15 [1.76-2.62], p<0.001). Women were more likely to have a normal coronary angiogram or coronary lesions with <50% luminal stenosis (p<0.001 for both), and thus a final diagnosis other than ACS. Both during hospitalization and at hospital discharge, women were less likely to receive guideline-recommended secondary prevention therapy. CONCLUSION: In women admitted for ACS, revascularization strategies are still underused, as is guideline-recommended secondary prevention therapy, which may explain their higher incidence of in-hospital complications and higher unadjusted mortality.Introduc¸ão: A doenc¸a arterial coronária está-se a tornar a principal causa de morte no mundo ocidental no género feminino. Contudo, os dados de que dispomos mostram que as mulheres são ainda subdiagnosticadas e subtratadas com as terapias de prevenc¸ão secundária recomendadas, levando a taxas significativamente mais altas de complicac¸ões intra-hospitalares e mortalidade intra-hospitalar. Objetivo: Avaliar a abordagem nacional às síndromas coronárias agudas, incluindo forma de apresentac¸ão, tratamento intra-hospitalar e complicac¸ões intra-hospitalares, de acordo com o género e em três períodos distintos. Métodos: Estudo observacional com análise retrospetiva de todos os doentes incluídos entre 2002 e 2019 no Registo Nacional de Síndromas Coronárias Agudas (RNSCA), um registo voluntário, observacional, prospetivo e contínuo da Sociedade Portuguesa de Cardiologia e do Centro Nacional de Colec¸ão de Dados em Cardiologia. Resultados: Foram incluídos 49 113 doentes (34 936 homens e 14 177 mulheres). Obesidade, hipertensão arterial, diabetes mellitus (p < 0,001 para todos) e dislipidémia (p = 0,022) foram mais prevalentes nas mulheres, que são mais frequentemente admitidas por síndroma coronária aguda sem supradesnivelamento do segmento ST (p < 0,001) e mais frequentemente se apresentam com sintomas atípicos. As mulheres têm tempos mais longos até agulha e até reperfusão, esta última menos frequente neste género (p < 0,001). Durante hospitalizac¸ão, as mulheres têm um risco significativamente maior de mortalidade intra-hospitalar (OR 1,94 [1,78;2,12], p < 0,001), hemorragia major (OR 1,53 [1,30;1,80], p < 0,001), insuficiência cardíaca (OR 1,87 [1,78;1,97], p < 0,001), fibrilhac¸ão auricular (OR 1,55 [1,36;1,77], p < 0,001), complicac¸ões mecânicas (OR 2,12 [1,78;2,53], p < 0,001), choque cardiogénico (OR 1,71 [1,57;1,87], p < 0,001) e acidente vascular cerebral (OR 2,15 [1,76;2,62], p < 0,001). É mais provável que as mulheres tenham uma coronariografia normal ou lesão coronárias com estenose luminal < 50% (p < 0,001 para ambos) e, assim, um diagnóstico final alternativo a síndroma coronária aguda. Seja durante hospitalizac¸ão ou à alta hospitalar, é menos provável que as mulheres recebam as terapias de prevenc¸ão secundária recomendadas. Conclusão: Em mulheres admitidas com síndroma coronária aguda as estratégias de revascularizac¸ão são subutilizadas, assim como as terapias de prevenc¸ão secundária recomendadas, podendo justificar a maior incidência de complicac¸ões intra-hospitalares e maior mortalidade não ajustada.info:eu-repo/semantics/publishedVersio

Impact of Multimorbidity and Polypharmacy on Clinical Outcomes of Elderly Chinese Patients with Atrial Fibrillation

Background: The co-incidence of multiple morbidities and polypharmacy is common amongst patients with atrial fibrillation (AF); however, data on their impact on clinical outcomes are scarce in Asian cohorts. Objective: To evaluate the impact of multimorbidity and polypharmacy on clinical outcomes and AF management among elderly Chinese patients. Methods: The ChiOTEAF registry is a prospective, multicenter nationwide study conducted from October 2014 to December 2018. Endpoints of interest were the composite outcome of all-cause death/any thromboembolism (TE), all-cause death, cardiovascular death, TE events, major bleeding, as well as AF management. Results: The eligible cohort included 6341 individuals (mean age 74.7 ± 10.7; 39.1% female), of whom 4644 (73.2%) had multimorbidity (defined as two or more chronic diseases), and 2262 (35.7%) were treated with five or more medications. There were 2775 (43.8%) patients on anticoagulant (OAC) use. On multivariate analysis, (i) multimorbidity was associated with a higher odds ratio of the composite outcome (OR: 2.04; 95% CI: 1.49–2.79), all-cause death (OR: 1.82; 95% CI: 1.31–2.54), cardiovascular death (OR: 2.05; 95% CI: 1.13–3.69), any TE (OR: 2.69; 95% CI: 1.29–5.62), and major bleeding (OR: 2.61; 95% CI: 1.25–5.45); (ii) polypharmacy was associated with a lower odds ratio of all-cause death (OR: 0.78; 95% CI: 0.63–0.96). The use of OAC was safe and was associated with a lower odds ratio of the composite outcome and all-cause death in all subgroups of patients. Conclusions: Multimorbidity and polypharmacy were common among elderly AF Chinese patients. Multimorbidity was an independent predictor of adverse clinical outcomes. The use of OAC was safe and significantly improved survival amongst AF patients with multimorbidity and polypharmacy

Perinatal insults and neurodevelopmental disorders may impact Huntington's disease age of diagnosis

INTRODUCTION: The age of diagnosis of Huntington's disease (HD) varies among individuals with the same HTT CAG repeat expansion size. We investigated whether early-life events, like perinatal insults or neurodevelopmental disorders, influence the diagnosis age. METHODS: We used data from 13,856 participants from REGISTRY and Enroll-HD, two large international multicenter observational studies. Disease-free survival analyses of mutation carriers with an HTT CAG repeat expansion size above and including 36 were computed through Kaplan-Meier estimates of median time until an HD diagnosis. Comparisons between groups were computed using a Cox proportional hazard survival model adjusted for CAG-repeat expansion length. We also assessed whether the group effect depended on gender and the affected parent. RESULTS: Insults in the perinatal period were associated with an earlier median age of diagnosis of 45.00 years (95%CI: 42.07–47.92) compared to 51.00 years (95%CI: 50.68–51.31) in the reference group, with a CAG-adjusted hazard ratio of 1.61 (95%CI: 1.26–2.06). Neurodevelopmental disorders were also associated with an earlier median age of diagnosis than the reference group of 47.00 years (95% CI: 43.38–50.62) with a CAG-adjusted hazard ratio of 1.42 (95%CI: 1.16–1.75). These associations did not change significantly with gender or affected parent. CONCLUSIONS: These results, derived from large observational datasets, show that perinatal insults and neurodevelopmental disorders are associated with earlier ages of diagnosis of magnitudes similar to the effects of known genetic modifiers of HD. Given their clear temporal separation, these early events may be causative of earlier HD onset, but further research is needed to prove causation