Clinical efficacy of efgartigimod combined with intravenous methylprednisolone in the acute phase of neuromyelitis optica spectrum disorders.

Background: Neuromyelitis Optica Spectrum Disorders (NMOSD) comprise a group of autoimmune-mediated, inflammatory, demyelinating central nervous system diseases caused by aquaporin-4 (AQP4) IgG autoantibodies. Efgartigimod is a human IgG Fc fragment that reduces antibody titers by targeting the neonatal Fc receptor (FcRn). This study documents the efficacy of efgartigimod combined with intravenous methylprednisolone (IVMP) in the acute phase of NMOSD.

International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USA.

Introduction: The clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of clinical outcome assessment for Rett syndrome and (2) identify what is needed to ensure that fit-for-purpose clinical outcome assessments are available for clinical trials.

Methods: Clinical outcome assessments for the top 10 priority domains identified in the Voice of the Patient Report for Rett syndrome were compiled and available psychometric data were extracted.

snRNA-seq stratifies multiple sclerosis patients into distinct white matter glial responses.

Poor understanding of the cellular and molecular basis of clinical and genetic heterogeneity in progressive multiple sclerosis (MS) has hindered the search for new effective therapies. To address this gap, we analyzed 632,000 single-nucleus RNA sequencing profiles from 156 brain tissue samples of MS and control donors to examine inter- and intra-donor heterogeneity. We found distinct cell type-specific gene expression changes between MS gray and white matter, highlighting clear pathology differences.

Real-world status of multimodal treatment of Stage IIIA-N2 non-small cell lung cancer in Japan: Results from the SOLUTION study, a non-interventional, multicenter cohort study.

Objectives: There is limited consensus on resectability criteria for Stage IIIA-N2 non-small cell lung cancer (NSCLC). We examined the patient characteristics, N2 status, treatment decisions, and clinical outcomes according to the treatment modality for Stage IIIA-N2 NSCLC in Japan.

Materials And Methods: Patients with Stage IIIA-N2 NSCLC in Japan were consecutively registered in the SOLUTION study between 2013 and 2014.

Visual quality of life in NMOSD and MOGAD: profiles, dynamics and associations with ageing and vision.

Objective: In this multicentric study, we were interested in the vision-related quality of life and its association with visual impairment in neuromyelitis optica spectrum disorders (NMOSD) and myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) in comparison to multiple sclerosis (MS) and healthy controls.

Methods: We analysed extracted data from the German NEMOS registry including National Eye Institute Visual Function Questionnaire (NEI-VFQ) scores, high and low contrast visual acuity (HCVA, LCVA), visually evoked potentials (VEP) and the scores for the expanded disability status scale (EDSS) and other neurological tests which assessed their disease-related impairment. The mean follow-up time of our patients was 1.

Results of carotid stenting in patients with contralateral internal carotid artery occlusion: a retrospective single-center analysis and 22 years of experience.

Purpose: Patients with contralateral carotid artery occlusion (CCO) represent a subgroup of patients at risk for revascularization procedures. The choice of appropriate revascularization procedure (carotid endarterectomy (CEA) or carotid artery stenting (CAS)) in these patients is controversial. The aim of this study is to share the results of clinical and radiological follow-up after CAS in these patients and to contribute to the literature by evaluating the efficacy and safety of stenting.

MPO-ANCA-positive eosinophilic granulomatosis with polyangiitis complicated by alveolar hemorrhage treated with mepolizumab as an induction therapy: Case report.

Eosinophilic granulomatosis with polyangiitis (EGPA) is a systemic vasculitis preceded by bronchial asthma or allergic sinusitis and accompanied by peripheral blood eosinophilia. Immunosuppressive drugs, such as cyclophosphamide in addition to high-dose glucocorticoids, are recommended for induction of remission in patients with severe EGPA. Although mepolizumab is widely recognized as remission induction therapy in non-fatal/non-organ disabling or relapsed/refractory EGPA, its efficacy and safety in induction of remission for severe cases have been ambiguous.

Association of critically short telomeres with brain and blood markers of ageing and Alzheimer's disease in older adults.

Background: Accumulation of critically short telomeres (CST) is implicated in decreased tissular regenerative capacity and increased susceptibility to degenerative diseases such as Alzheimer's disease (AD). Telomere shortening has also been associated with age-related brain changes. However, it remains unclear whether CST accumulation is directly associated with AD markers or instead amplifies age-related effects, potentially increasing susceptibility of developing AD in cognitively healthy older adults.

Safety and efficacy of intrathecal antibodies to Nogo-A in patients with acute cervical spinal cord injury: a randomised, double-blind, multicentre, placebo-controlled, phase 2b trial.

Background: Spinal cord injury results in permanent neurological impairment and disability due to the absence of spontaneous regeneration. NG101, a recombinant human antibody, neutralises the neurite growth-inhibiting protein Nogo-A, promoting neural repair and motor recovery in animal models of spinal cord injury. We aimed to evaluate the efficacy of intrathecal NG101 on recovery in patients with acute cervical traumatic spinal cord injury.

Bibliometric and visual analysis of medication therapy management from 2003 to 2023.

Medication therapy management (MTM) includes various clinical pharmacy services that greatly influence society and economy. Despite extensive research in recent years, there is currently a lack of bibliometric analysis on this subject. Articles and reviews on MTM published from 2003 to 2023 were identified and selected from the Web of Science Core Collection.

Dual Antiplatelet Treatment up to 72 Hours After Ischemic Stroke Stratified by Risk Profile: A Post Hoc Analysis.

Background: Risk profile of recurrence may influence the effect of antiplatelet therapy. This study aimed to evaluate the efficacy and safety of clopidogrel-aspirin initiated within 72 hours after symptom onset for acute mild stroke or high-risk transient ischemic attack stratified by risk profile.

Methods: This is a secondary post hoc analysis of the INSPIRES (Intensive Statin and Antiplatelet Therapy for Acute High-risk Intracranial or Extracranial Atherosclerosis) randomized clinical trial that enrolled patients 35 to 80 years old with acute mild ischemic stroke or high-risk transient ischemic attack between 2018 and 2022.

Cerebral spinal fluid analyses and therapeutic implications for leptomeningeal metastatic disease.

Purpose: To review applications of cerebral spinal fluid (CSF) biomarkers for the diagnosis, monitoring and treatment of leptomeningeal metastatic disease (LMD) among patients with metastatic solid tumors.

Methods: A narrative review identified original research related to CSF biomarkers among patients with metastatic solid tumors and LMD. Pre-clinical research (e.

Changes in Cardiovascular Health, Genetic Risk, and Cardiometabolic Diseases: Evidence From a Large-Scale Cohort Study.

Background: Evidence has firmly established the association between superior cardiovascular health (CVH) and reduced susceptibility to cardiometabolic diseases (CMDs). In reality, CVH experiences dynamic fluctuations throughout individuals' lifespans. However, the association between changes in CVH and the impact on CMDs among individuals with different genetic risks remains unclear.

Safety and Efficacy of Intravenous Thrombolysis by Infarct Type in Patients with No Visible Occlusion: A Secondary Analysis of the AcT Trial.

Background: About 25% of patients with acute ischemic stroke have lacunar infarct on follow-up imaging. In this secondary analysis from the AcT (Alteplase Compared With Tenecteplase) trial, we assessed if there is variation in safety or efficacy of intravenous thrombolysis by infarct type in patients with no visible occlusion. We also determined if this effect differed between tenecteplase and alteplase.

Sodium Oxybate-Treated Familial Myoclonus-Dystonia Syndrome Due to Novel SGCE Variant.

Myoclonus-dystonia syndrome (MDS, OMIM #159900) is an autosomal-dominant movement disorder caused by heterozygous variants in the epsilon sarcoglycan gene (SGCE) and characterized by a combination of myoclonic jerks, dystonia, and psychiatric comorbidities. Patients with MDS have a normal life expectancy with markedly reduced quality of life. Here, we report four family members diagnosed with MDS of variable severity due to a novel heterozygous splicing variant in SGCE (c.

Randomized Study Comparing First-Line Dual Versus Single-Stent Retriever Technique: TWIN2WIN.

Background: The double-stent retriever (SR) technique has been described as an effective rescue technique when single-SR fails to induce recanalization. We aimed to assess the safety and efficacy of first-line double-SR in patients with stroke undergoing thrombectomy.

Methods: This was a multicenter, randomized, controlled, blinded adjudicated primary outcome study.

Dual antiplatelet therapy after minor strokes or high-risk TIA: Evidence from the Austrian stroke registry.

Background And Purpose: Three large, randomized trials demonstrated the benefit of short-term dual antiplatelet therapy (DAPT) versus monotherapy after non-cardioembolic minor stroke or high-risk transient ischemic attack (TIA). The aim of this study was to evaluate effects of DAPT versus monotherapy on functional outcomes and safety in a real-life setting.

Methods: Patients with minor stroke (NIHSS <4) or high-risk TIA (ABCD2 score ≥4) of non-cardioembolic origin without major vessel occlusion or revascularization therapy (thrombolysis or thrombectomy) treated between 2018 and 2023 were analyzed based on a prospective nationwide stroke unit registry.

Remote ischaemic conditioning for neurological disorders-a systematic review and narrative synthesis.

Introduction: Remote ischaemic conditioning (RIC) refers to the use of controlled transient ischemic and reperfusion cycles, commonly of the upper or lower limb, to mitigate cellular damage from ischaemic injury. Preclinical studies demonstrate that RIC may have a neuroprotective effect and therefore could represent a novel therapeutic option in the management of neurological disorders. The aim of this review is to comprehensively describe the current clinical evidence of RIC in neurological disorders.

The clinical and biochemical effectiveness and safety of cholic acid treatment for bile acid synthesis defects: a systematic review.

Background: Bile acid synthesis defects (BASDs) can be severely disabling involving the liver and nervous system, potentially due to elevated levels of toxic C-bile acid intermediates. Cholic acid (CA) supplementation is hypothesized to decrease bile acid production, stimulate bile secretion and -flow, and slowing down disease progression. This systematic review assesses the clinical and biochemical effectiveness, and safety of CA in BASDs patients.

Disentangling relationships between Alzheimer's disease plasma biomarkers and established biomarkers in patients of tertiary memory clinics.

Background: Several plasma biomarkers for Alzheimer's disease (AD) have demonstrated diagnostic and analytical robustness. Yet, contradictory results have been obtained regarding their association with standard diagnostic markers of AD. This study aims to investigate the specific relationship between the AD biomarkers currently used in clinical practice and the plasma biomarkers.

Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy.

Importance: Limited studies have assessed the long-term benefit/risk of gene therapy for Leber hereditary optic neuropathy (LHON).

Objective: To determine the safety and efficacy of lenadogene nolparvovec in patients with LHON due to the MT-ND4 gene variant for up to 5 years after administration.

Design, Setting, And Participants: The RESCUE and REVERSE Long-Term Follow-up Study (RESTORE), conducted from 2018 to 2022, is the 5-year follow-up study of the 2 phase 3 clinical studies RESCUE (Efficacy Study of Lenadogene Nolparvovec for the Treatment of Vision Loss Up to 6 Months From Onset in LHON Due to the MT-ND4 Mutation) and REVERSE (Efficacy Study of Lenadogene Nolparvovec for the Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the MT-ND4 Mutation).

Prevalence and solving strategies of drug-related problems in adult psychiatric inpatients - a systematic review.

Introduction: Most psychiatric inpatients receive psychopharmacological treatment indicated for their mental diseases. The aim of this systematic review is to give clinical pharmacists and physicians a comprehensive summary of common drug-related problems (DRPs) in adult psychiatric inpatients and of potential interventions to solve them in clinical practice.

Methods: Six databases and registers were searched for English, German and French articles published between 1999 and 2023 with content regarding the prevalence and/or type or interventions to solve DRPs in adult psychiatric inpatients.

Knowledge of Risk Factors for Dementia and Attitudes on a Dementia Prevention Program by Age and Ethnicity in Arizona.

Background: Dementia disproportionately affects Hispanic communities, which may be partially attributable to disparities in resources to address modifiable risk factors. Addressing risk factors at younger ages would likely confer greater benefit than at older ages. Interest among Hispanic and younger persons participating in a dementia prevention program is unknown.

DBS in the restoration of motor functional recovery following spinal cord injury.

The landscape of therapeutic deep brain stimulation (DBS) for locomotor function recovery is rapidly evolving. This review provides an overview of electrical neuromodulation effects on spinal cord injury (SCI), focusing on DBS for motor functional recovery in human and animal models. We highlight research providing insight into underlying cellular and molecular mechanisms.