PRX004 in variant amyloid transthyretin (ATTRv) amyloidosis: results of a phase 1, open-label, dose-escalation study.

Background: The investigational monoclonal antibody PRX004 is designed to specifically target and deplete TTR amyloid. Here, we report on the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary clinical activity of PRX004 in patients with ATTRv amyloidosis.

Methods: This global, multicentre, phase 1 trial comprised a 3 + 3 dose-escalation phase and a long-term extension (LTE) phase (NCT03336580).

Brain MRI in patients with V30M hereditary transthyretin amyloidosis.

Background: Central nervous system dysfunction is common in longstanding hereditary transthyretin amyloidosis (ATTRv) caused by the V30M (p.V50M) mutation. Neuropathology studies show leptomeningeal amyloid deposition and cerebral amyloid angiopathy (CAA).

Neurological Involvement in a Portuguese Cohort of IgG4-Related Disease.

Introduction: Neurological involvement in immunoglobulin G4-related disease (IgG4-RD) is increasingly recognized. Its diagnosis can be challenging due to clinical mimics and difficulty in obtaining nervous system biopsies. The aim of this study was to describe a cohort of neurological IgG4-RD patients.

Distinct disease-modifying therapies are associated with different blood immune cell profiles in people with relapsing-remitting multiple sclerosis.

Disease modifying therapies (DMTs) used for treating people with relapsing-remitting multiple sclerosis (pwRRMS) target the immune system by different mechanisms of action. However, there is a lack of a comprehensive assessment of their effects on the immune system in comparison to treatment-naïve pwRRMS. Herein, we evaluated the numbers of circulating B cells, CD4 and CD8 T cells, regulatory T cells (Tregs), natural killer (NK) cells and NKT cells, and their subsets, in pwRRMS who were treatment-naïve or treated with different DMTs.

Idiopathic inflammatory myopathies - The burden of disease: Cohort analysis focusing on damage and comorbidities.

Introduction/background: Idiopathic Inflammatory Myopathies (IIM) continue to be a major clinical challenge worldwide. The exact aetiopathogenesis of this chronic and disabling disease remains elusive, preventing the development of novel and effective therapeutic strategies and leading to a high incidence of damage. The complexity of treating these diseases is even greater due to the numerous comorbidities that affect these patients.

An improved method for determining free amino acids by RP-HPLC/DAD with o-phthalaldehyde derivatization: Method evaluation in beers and wines.

Rapid methods for amino acid determination are desired to reduce running times, and the main factors involved in the rapid separation of these compounds in HPLC are the columns, solvents, and gradient. The present study refers to a method optimization to rapidly analyze 19 amino acids in RP-HPLC/DAD with pre-column derivatization using o-phthalaldehyde. To evaluate the method's robustness, Indian Pale Ale-IPA beers and wines from the San Francisco Valley-SFV and Chapada Diamantina-CHD, Brazil, were analyzed.

Teriflunomide treatment outcomes in multiple sclerosis: A Portuguese real-life experience.

Teriflunomide is an oral disease-modifying therapy for relapsing-remitting multiple sclerosis patients. A decline in physical and cognitive functions, which negatively impacts their quality of life (QoL), is observed in relapsing-remitting multiple sclerosis patients. The aim of this study was to characterise adult Portuguese relapsing-remitting multiple sclerosis patients treated with teriflunomide in routine clinical practice concerning their quality of life, comorbidities, treatment effectiveness, satisfaction, compliance and safety.

People with Primary Progressive Multiple Sclerosis Have a Lower Number of Central Memory T Cells and HLA-DR Tregs.

The importance of circulating immune cells to primary progressive multiple sclerosis (PPMS) pathophysiology is still controversial because most immunotherapies were shown to be ineffective in treating people with PPMS (pwPPMS). Yet, although controversial, data exist describing peripheral immune system alterations in pwPPMS. This study aims to investigate which alterations might be present in pwPPMS free of disease-modifying drugs (DMD) in comparison to age- and sex-matched healthy controls.

Effect of commercial yeasts (Saccharomyces cerevisiae) on fermentation metabolites, phenolic compounds, and bioaccessibility of Brazilian fermented oranges.

Brazil is the largest producer of oranges worldwide, as well as one of the largest producers of orange juice. Alcoholic fermented beverages have been considered a marketable alternative for oranges. In this study, four S.

Predictors of cognitive dysfunction in hereditary transthyretin amyloidosis with liver transplant.

Background: Cognitive dysfunction is part of the broad spectrum of clinical manifestations in older untreated hereditary transthyretin amyloidosis patients with peripheral polyneuropathy.

Objective: The objective of this study is to systematically explore cognitive dysfunction in ATTRV30M amyloidosis patients whose disease course was modified by liver transplant (LT).

Methods: A series of 269 carriers of TTRVal30Met mutation treated with LT underwent a neuropsychological assessment.

Low Memory T Cells Blood Counts and High Naïve Regulatory T Cells Percentage at Relapsing Remitting Multiple Sclerosis Diagnosis.

Objective: The aim of this study is to assess the peripheral immune system of newly diagnosed patients with relapsing remitting multiple sclerosis (RRMS) and compare it to healthy controls (HC).

Methods: This cross-sectional study involves 30 treatment-naïve newly diagnosed patients with RRMS and 33 sex- and age-matched HC. Peripheral blood mononuclear cells were analyzed regarding: i) thymic function surrogates [T cell receptor excision circles (TRECs) and recent thymic emigrants (RTEs)]; ii) naïve and memory CD4 and CD8 T cells subsets; iii) T helper (Th) phenotype and chemokine receptors expression on CD8 T cells subsets; iv) regulatory T cell (Tregs) phenotype; and exclude expression of activating/inhibitory receptors by natural killer (NK) and NKT cells.

Autoimmune encephalitis: suspicion in clinical practice and mimics.

Despite the existence of well-established diagnostic criteria for autoimmune encephalitis, there are diseases capable of mimicking it. This study sought to retrospectively evaluate the reasons for testing and the final diagnosis of patients admitted to a Neurology ward tested for anti-NMDAR antibodies and estimate sensitivity and specificity of current diagnostic criteria. The threshold for testing was lower than that of the prevailing diagnostic criteria, and the proportion of autoimmune encephalitis mimics was high.

Correlated prevalence of hydrocele and microfilaremia in Amazon (Belém, 1951-2005).

Background & Objectives: For decades, the city of Belém in Brazil's eastern Amazon was the second city in the country with highest prevalence of cases of filariasis due to Wuchereria bancrofti infection. However, this prevalence decreased over time until reaching null records, concomitantly with a decrease in frequency of recorded hydrocele cases. In this context, we analyzed cross-sectional data to evaluate the degree of correlation between prevalence of positive blood microfilariae results during surveillance screening occurred along 54 years (1951-2005) and prevalence of hydrocele cases recorded in the same time period.

Kappa free light chains: Diagnostic performance in multiple sclerosis and utility in a clinical laboratory.

Background: Automated, technically simple analytical methods offering objective results are highly valued in clinical laboratories. Kappa free light chains (KFLC) in cerebrospinal fluid (CSF) are promising multiple sclerosis (MS) biomarkers, particularly kappa (K) index.

Methods: KFLC were determined in CSF and serum samples of patients diagnosed with MS, clinically/radiologically isolated syndrome (N, 39), and controls (N, 152; inflammatory and non-inflammatory neurological disorders).

Pediatric neuromyelitis optica spectrum disorders in Portugal: A multicentre retrospective study.

Introduction: Neuromyelitis optica spectrum disorders (NMOSD) are more prevalent in adulthood, with few cases reported in pediatric age (<18 years). In this group, anti-aquaporin 4 (AQP4) antibodies are less frequent, while antibodies against myelin oligodendrocyte glycoprotein (MOG) are more commonly detectable than in adults.

Objective And Methods: Description of pediatric NMOSD cases identified in a national multicentric NMOSD Portuguese registry.

Cognitive dysfunction and mortality in multiple sclerosis: Long-term retrospective review.

Background: Cognitive dysfunction as a predictor of clinical progression and mortality in multiple sclerosis (MS) is still a matter of debate.

Objective: The aim of this study was to explore the long-term outcome associated with neuropsychological performance in a cohort of patients with MS.

Methods: A series of 408 MS patients had previously undergone a comprehensive neuropsychological assessment and a contemporaneous neurological evaluation (T1).

Graves' disease with spontaneous resolution following ocrelizumab in primary progressive multiple sclerosis.

Immune reconstitution therapies (IRT), which include antibody-based cell-depleting therapies targeting CD52+ (alemtuzumab) or CD20+ (rituximab, ocrelizumab) leukocytes, are approved for the treatment of multiple sclerosis. Thyroid autoimmunity is a common adverse effect of alemtuzumab treatment, Graves' disease (GD) being the most prevalent manifestation. To date, thyroid autoimmunity events have not been reported with CD20-targeting monoclonal antibodies.

Depression and anxiety in multiple sclerosis patients: The role of genetic variability of interleukin 1β.

Background: Mood disorders, as depression and anxiety, are frequent in Multiple Sclerosis (MS) patients. High pro-inflammatory cytokine levels (e.g.

Pituitary metastasis unveiling a lung adenocarcinoma.

Summary: Pituitary metastasis (PM) can be the initial presentation of an otherwise unknown malignancy. As PM has no clinical or radiological pathognomonic features, diagnosis is challenging. The authors describe the case of a symptomatic PM that revealed a primary lung adenocarcinoma.

Pregnancy in Patients With AQP4-Ab, MOG-Ab, or Double-Negative Neuromyelitis Optica Disorder.

Objective: To analyze the effects of pregnancy on neuromyelitis optica spectrum disorder (NMOSD) according to patients' serostatus and immunosuppressive therapy (IST).

Methods: We performed a retrospective multicenter international study on patients with NMOSD. Patients were tested for aquaporin-4 (AQP4) and myelin oligodendrocyte glycoprotein (MOG) antibodies (Ab).

Prognostic value of odor identification impairment in multiple sclerosis: 10-Years follow-up.

Background: Olfactory dysfunction has been linked to clinical severity variables in multiple MS populations. Though, its prognostic value is still unknown.

Objective: The aim of this study was to explore the long-term outcome associated with Brief-Smell Identification Test (B-SIT) performance in a cohort of MS patients.