Disease progression strikingly differs in research and real-world Parkinson's populations.

Characterization of Parkinson's disease (PD) progression using real-world evidence could guide clinical trial design and identify subpopulations. Efforts to curate research populations, the increasing availability of real-world data, and advances in natural language processing, particularly large language models, allow for a more granular comparison of populations than previously possible. This study includes two research populations and two real-world data-derived (RWD) populations.

Disease progression strikingly differs in research and real-world Parkinson's populations.

Characterization of Parkinson's disease (PD) progression using real-world evidence could guide clinical trial design and identify subpopulations. Efforts to curate research populations, the increasing availability of real-world data and recent advances in natural language processing, particularly large language models, allow for a more granular comparison of populations and the methods of data collection describing these populations than previously possible. This study includes two research populations and two real-world data derived (RWD) populations.

Difficult-to-treat primary immune thrombocytopenia in adults: Prevalence and burden. Results from the CARMEN-France registry.

The aim of this study was to assess the prevalence and the burden of difficult-to-treat primary ITP (pITP), defined by the need for another ITP treatment after romiplostim and eltrombopag. Adult patients were selected in the prospective, real-world CARMEN-France registry up to December 2021. Out of 821 adult patients with pITP, 29 had difficult-to-treat ITP (3.

Impairment of platelet function in both mild and severe COVID-19 patients.

Abnormalities of platelet function were reported in patients with severe COVID-19 (severe-C), but few data are available in patients with mild COVID-19 (mild-C) and after COVID-19 recovery. The aim of this study was to investigate platelet parameters in mild-C patients (n = 51), with no evidence of pneumonia, and severe-C patients (n = 49), during the acute phase and after recovery, compared to 43 healthy controls. Both mild-C and severe-C patients displayed increased circulating activated platelets, low δ-granule content (ADP, serotonin), impaired platelet activation by collagen (light transmission aggregometry) and impaired platelet thrombus formation on collagen-coated surfaces under controlled flow conditions (300/s shear rate).

Long-term sutimlimab improves quality of life for patients with cold agglutinin disease: CARDINAL 2-year follow-up.

Cold agglutinin disease (CAD) is a rare form of autoimmune hemolytic anemia with a substantial burden on patient's quality of life. CARDINAL was a 2-part, open-label, single-arm, multicenter phase 3 study evaluating the C1s inhibitor, sutimlimab, for treatment of CAD. Part A consisted of the pivotal study phase, with the part B extension phase assessing long-term safety and durability of response including patient-reported outcomes, which is the focus of this report.

Experiences of parents and patients hospitalised in a child psychiatric unit for anorexia nervosa after reorganisation of care imposed by the COVID-19 Pandemic in France: a qualitative study-The QUALICOVID study.

Background: Anorexia nervosa is a serious, albeit common mental illness that generally occurs during adolescence. Although outpatient care is recommended, hospitalisation is sometimes required. There is a dedicated hospitalisation unit caring for children and adolescents presenting with anorexia nervosa in Nancy, France.

Insights Into the Low Rate of In-Pump Thrombosis With the HeartMate 3: Does the Artificial Pulse Improve Washout?

While earlier studies reported no relevant effect of the HeartMate 3 (HM3) artificial pulse (AP) on bulk pump washout, its effect on regions with prolonged residence times remains unexplored. Using numerical simulations, we compared pump washout in the HM3 with and without AP with a focus on the clearance of the last 5% of the pump volume. Results were examined in terms of flush-volume ( , number of times the pump was flushed with new blood) to probe the effect of the AP independent of changing flow rate.

On-Chip Platelet Activation Assessment: Microfluidic Emulation of Shear Stress Profiles Induced by Mechanical Circulatory Support Devices.

Mechanical circulatory support devices (MCSDs), although proved to be a pillar in the clinical setting of advanced heart failure, are afflicted by thromboembolic complications. Shear-mediated platelet activation has been recognized to drive thromboembolic events in patients implanted with MCSDs. Despite this, to date, a clinically reliable diagnostic test for assessing platelet response to stress stimuli is still missing.

The effect of turbulence modelling on the assessment of platelet activation.

Pathological platelet activation by abnormal shear stresses is regarded as a main clinical complication in recipients of cardiovascular mechanical devices. In order to improve their performance computational fluid dynamics (CFD) are used to evaluate flow fields and related shear stresses. CFD models are coupled with mathematical models that describe the relation between fluid dynamics variables, and in particular shear stresses, and the platelet activation state (PAS).

Electrical impedance vs. light transmission aggregometry: Testing platelet reactivity to antiplatelet drugs using the MICELI POC impedance aggregometer as compared to a commercial predecessor.

Background: Patients' responses to antiplatelet therapy significantly vary, with individuals showing high residual platelet reactivity associated with thrombosis. To personalize thrombosis management, platelet function testing has been suggested as a promising tool able to monitor the antithrombotic effect of antiplatelet agents in real-time. We have prototyped the MICELI, a miniature and easy-to-use electrical impedance aggregometer (EIA), measuring platelet aggregation in whole blood.

Accelerating diagnosis of Parkinson's disease through risk prediction.

Background: Characterization of prediagnostic Parkinson's Disease (PD) and early prediction of subsequent development are critical for preventive interventions, risk stratification and understanding of disease pathology. This study aims to characterize the role of the prediagnostic period in PD and, using selected features from this period as novel interception points, construct a prediction model to accelerate the diagnosis in a real-world setting.

Methods: We constructed two sets of machine learning models: a retrospective approach highlighting exposures up to 5 years prior to PD diagnosis, and an alternative model that prospectively predicted future PD diagnosis from all individuals at their first diagnosis of a gait or tremor disorder, these being features that appeared to represent the initiation of a differential diagnostic window.

Characterization of the competing role of surface-contact and shear stress on platelet activation in the setting of blood contacting devices.

Supraphysiological shear stress and surface-contact are recognized as driving mechanisms of platelet activation (PA) in blood contacting devices (BCDs). However, the competing role of these mechanisms in triggering thrombogenic events is poorly understood. Here, we characterized the dynamics of PA in response to the combined effect of shear stress and material exposure.

Health Assessment Questionnaire-Disability Index (HAQ-DI) use in modelling disease progression in diffuse cutaneous systemic sclerosis: an analysis from the EUSTAR database.

Background: Patients with diffuse cutaneous systemic sclerosis (dcSSc) have a poor prognosis. The importance of monitoring subjective measures of functioning and disability, such as the Health Assessment Questionnaire-Disability Index (HAQ-DI), is important as dcSSc is rated by patients as worse than diabetes or hemodialysis for quality of life impairment. This European Scleroderma Trials and Research (EUSTAR) database analysis was undertaken to examine the importance of impaired functionality in dcSSc prognosis.

Thrombotic Risk of Rotor Speed Modulation Regimes of Contemporary Centrifugal Continuous-flow Left Ventricular Assist Devices.

Contemporary centrifugal continuous-flow left ventricular assist devices (LVADs) incorporate dynamic speed modulation algorithms. Hemocompatibility of these periodic unsteady pump operating conditions has been only partially explored. We evaluated whether speed modulation induces flow alterations associated with detrimental prothrombotic effects.

Outcomes Associated with Dronedarone Use in Patients with Atrial Fibrillation.

The antiarrhythmic drug dronedarone was designed to reduce the extra-cardiac adverse effects associated with amiodarone use in treatment of patients with atrial fibrillation / atrial flutter (AF/AFL). This epidemiological study used a retrospective cohort design to compare risk of cardiovascular-related hospitalizations and death in AF/AFL patients treated with dronedarone versus other antiarrhythmic drugs (AADs). AF/AFL patients with incident dronedarone fills were matched by propensity score (PS) to incident users of other AADs.

Fluid dynamics characterization and thrombogenicity assessment of a levitating centrifugal pump with different impeller designs.

Technical guidelines nowadays recommend and regulate the use Computational Fluid Dynamics (CFD) to assess the performance of medical devices. CFD coupled to blood damage models has emerged as a powerful tool to evaluate the hemocompatibility of blood recirculating devices. The present study is aimed at evaluating the hydrodynamic performance and the thrombogenic potential of two prototypes of magnetically levitating centrifugal pumps.

Patients' experiences of Parkinson's disease: a qualitative study in glucocerebrosidase and idiopathic Parkinson's disease.

Background: Approximately 7-10% of Parkinson's disease (PD) patients carry a GBA (Glucocerebrosidase) mutation (GBA-PD patients), which may influence the disease's clinical course.

Objectives: This study aimed to explore the patient experience of GBA-PD and identify the most important symptoms and impacts to inform clinical trial measurement strategies.

Methods: Twenty PD patients (n = 15 GBA-PD; n = 5 idiopathic-PD) participated in qualitative interviews which explored concepts spontaneously reported or identified through a literature review.

The MICELI (MICrofluidic, ELectrical, Impedance): Prototyping a Point-of-Care Impedance Platelet Aggregometer.

As key cellular elements of hemostasis, platelets represent a primary target for thrombosis and bleeding management. Currently, therapeutic manipulations of platelet function (antithrombotic drugs) and count (platelet transfusion) are performed with limited or no real-time monitoring of the desired outcome at the point-of-care. To address the need, we have designed and fabricated an easy-to-use, accurate, and portable impedance aggregometer called "MICELI" (MICrofluidic, ELectrical, Impedance).

Platelet Adhesion and Thrombus Formation in Microchannels: The Effect of Assay-Dependent Variables.

Unlabelled: Microfluidic flow chambers (MFCs) allow the study of platelet adhesion and thrombus formation under flow, which may be influenced by several variables. We developed a new MFC, with which we tested the effects of different variables on the results of platelet deposition and thrombus formation on a collagen-coated surface.

Methods: Whole blood was perfused in the MFC over collagen Type I for 4 min at different wall shear rates (WSR) and different concentrations of collagen-coating solutions, keeping blood samples at room temperature or 37 °C before starting the experiments.

Uncertainty propagation of phase contrast-MRI derived inlet boundary conditions in computational hemodynamics models of thoracic aorta.

This study investigates the impact that uncertainty in phase contrast-MRI derived inlet boundary conditions has on patient-specific computational hemodynamics models of the healthy human thoracic aorta. By means of Monte Carlo simulations, we provide advice on where, when and how, it is important to account for this source of uncertainty. The study shows that the uncertainty propagates not only to the intravascular flow, but also to the shear stress distribution at the vessel wall.

Safety And Necessity Of Thermal Esophageal Probes During Radiofrequency Ablation For The Treatment Of Atrial Fibrillation.

Background: Radiofrequency ablation is extensively used to achieve pulmonary veins isolation for the cure of atrial fibrillation. Luminal esophageal temperature can be monitored by means of suitable probes to prevent the onset of lesions.

Objective: To compute the thermal field generated by the ablation, to investigate the interaction between the electromagnetic field and the probe sensors, and to provide a safe interpretation of the temperature detected by the probe, supported by clinical data.

Budgetary participation and performance: The mediating effects of medical managers' job engagement and self-efficacy.

Professional hybrids in healthcare have attracted a great deal of policy, managerial, and research interest. However, the current literature offers little guidance on (i) how hybrid roles can be supported by the hospital organization they work for as well as (ii) the cognitive and behavioral driving forces underpinning medical managers' managerial work that determine how they inhabit their roles and consequently meet the standards of performance that contribute to organizational effectiveness. Building on engagement theory and social cognitive theory and using data collected from clinical managers working in a large Italian public hospital, the current study focuses on the mediating role of psychological variables associated with the managerial role of medical managers, namely managerial job engagement and managerial self-efficacy, in the budgetary participation-job performance link.

Measuring treatment satisfaction in MS: Is the Treatment Satisfaction Questionnaire for Medication fit for purpose?

Background: The Treatment Satisfaction Questionnaire for Medication (TSQM) was designed to assess patient treatment satisfaction in chronic diseases. Its performance has not been examined in multiple sclerosis (MS). The 14 items of the TSQM cover four domains: Effectiveness, Side Effects, Convenience, and Global Satisfaction.

Multiple sclerosis relapses are associated with increased fatigue and reduced health-related quality of life - A post hoc analysis of the TEMSO and TOWER studies.

Background: Two pivotal phase 3 teriflunomide studies provided data on relapses, fatigue, and health-related quality of life (HRQoL) in patients with relapsing forms of multiple sclerosis (MS).

Objectives: Using pooled data from the TEMSO (NCT00134563) and TOWER (NCT00751881) studies, we investigated the association between relapse severity, and changes from baseline to Week 108 in fatigue and HRQoL outcomes.

Methods: Four definitions of relapse severity were applied in this analysis: sequelae post-relapse; relapse leading to hospitalization; relapse requiring intravenous corticosteroids; and intense relapse.