Post-hoc analyses from the ADAPT clinical study demonstrate aggregate sustained benefit of Efgartigimod in generalized myasthenia gravis.

Objectives: This post-hoc analysis evaluates the long-term efficacy of efgartigimod versus placebo in adult patients with generalized myasthenia gravis (gMG) with acetylcholine-receptor autoantibodies (AChR-Ab+), based on data from the ADAPT RCT and its open-label extension ADAPT+.

Methods: Changes from baseline in Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores were assessed by treatment group over the ADAPT (up to 20 weeks) and ADAPT+ time horizon (extended to 64 weeks for efgartigimod group patients). Response to treatment was defined as 5-point reduction in QMG or 3-point reduction in MG-ADL vs.

Psychometric properties of MG-ADL items and MG-ADL score: An assessment of distributional characteristics, validity and factor structure in two large datasets.

Background: The Myasthenia Gravis-Activities of Daily Living scale (MG-ADL) is an 8-item outcome measure to assess symptoms and functional limitations in myasthenia gravis (MG) patients. The MG-ADL score is an equally weighted level sum score that is used as primary outcome measures in clinical trials, in clinical practice, and as an end-point in health economic evaluation. This data analysis aims to obtain detailed knowledge of measurement properties of MG-ADL items and the MG-ADL score.

Concordance between patient- and physician-reported Myasthenia Gravis Activities of Daily Living (MG-ADL) scores.

Introduction/aims: Myasthenia gravis (MG) is a neuromuscular disease characterized by abnormal skeletal muscle fatiguability. The MG Activities of Daily Living (MG-ADL) scale assesses eight symptoms and is often used as primary endpoint in MG clinical trials where it is completed by neurologists. However, in observational studies, patients frequently complete the MG-ADL scale independently of their neurologist.

Association Between Myasthenia Gravis-Activities of Daily Living (MG-ADL) and EQ-5D-5L Utility Values: The Additional Effect of Efgartigimod on Utilities.

Introduction: For patients with generalized myasthenia gravis (gMG), the association between symptom severity, often measured with the Myasthenia Gravis Activities of Daily Living (MG-ADL) instrument, and utility values is unknown.

Methods: Data was analyzed from the phase 3 ADAPT trial, which included adult patients with gMG randomly assigned to treatment with efgartigimod + conventional therapy (EFG + CT) or placebo + CT (PBO + CT). MG-ADL total symptom scores and the EQ-5D-5L, a measure of health-related quality of life (HRQoL), were collected biweekly up to 26 weeks.

Patient-reported burden of myasthenia gravis: baseline results of the international prospective, observational, longitudinal real-world digital study MyRealWorld-MG.

Objectives: Myasthenia gravis (MG) is a rare, chronic, autoimmune neuromuscular disease which can affect functional and mental aspects of health and health-related quality of life (HRQoL). This study aims to obtain detailed knowledge of the impact of MG on HRQoL in a broad population from the perspective of the patient.

Design: Prospective, observational, digital, longitudinal real-world study.

An EQ-5D-Y-3L Value Set for Belgium.

Background: In Belgium, a value set for children and adolescents for a generic health-related quality-of-life measure is not available. To inform drug pricing and make resource allocation decisions for children and adolescents, national tax-payers' preferences for youth health states should be known.

Objective: We aimed to obtain a value set for EQ-5D-Y-3L in Belgium, following the international youth valuation protocol for data collection.

Exploration of the Reasons Why Health State Valuation Differs for Children Compared With Adults: A Mixed Methods Approach.

Objectives: Evidence comparing utilities for adults and children consistently report higher utility values for child health states. This study investigates the reasons why child health states are valued differently.

Methods: A total of 80 respondents (United Kingdom, Belgium, The Netherlands) participated in 1.

Exploring the Comparability of Face-to-Face Versus Video Conference-Based Composite Time Trade-Off Interviews: Insights from EQ-5D-Y-3L Valuation Studies in Belgium and Spain.

Background: Face-to-face interviews are recommended for the collection of composite time trade-off (cTTO) data. The coronavirus disease 2019 (COVID-19) social distancing measures made researchers consider videoconferencing interviews as an alternative mode of administration, but little evidence about the implementation of videoconferencing in valuation studies is available. This study provides insights into the effect of videoconferencing on the quality of data, evaluating interviewers' and respondents' engagement level in videoconferences compared with face-to-face interviews.

Thrombectomy is a cost-saving procedure up to 24 h after onset.

Introduction And Aim: The treatment of ischemic stroke due to large-vessel occlusion has been revolutionized by mechanical thrombectomy (MT), as multiple trials have consistently shown improved functional outcomes compared to standard medical management both in the early and late time windows after symptom onset. However, MT is an interventional procedure that is more costly than best supportive care (BSC).

Methods: We set out to study the cost-utility and budget impact of MT + BSC versus BSC alone for large-vessel occlusion using a combined decision tree and Markov model.

Patient-reportedimpact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG).

Introduction: Myasthenia gravis (MG) is a rare, chronic, autoimmune disease, mediated by immunoglobulin G antibodies, which causes debilitating muscle weakness. As with most rare diseases, there is little patient-reported data with which to understand and address patient needs. This study explores the impact of MG in the real world from the patient perspective.

Estimating health state utilities in hemophagocytic lymphohistiocytosis.

Background: Hemophagocyti.c lymphohistiocytosis (HLH) is a rare and severe disorder characterized by abnormal activation of the immune system. Primary HLH causes prolonged fever, spleen and liver enlargement, and organ dysfunction, usually in infancy and early childhood and is fatal if left untreated.

Evaluation of the cost-effectiveness of dexrazoxane for the prevention of anthracycline-related cardiotoxicity in children with sarcoma and haematologic malignancies: a European perspective.

Background: Anthracycline-treated childhood cancer survivors are at higher risk of cardiotoxicity, especially with cumulative doses received above 250 mg/m. Dexrazoxane is the only option recommended for cardiotoxicity prevention in high-risk patients supported by randomised trials but its cost-effectiveness in paediatric cancer patients has not been established.

Methods: A cost-effectiveness model applicable to different national healthcare system perspectives, which simulates 10,000 patients with either sarcoma or haematologic malignancies, based upon baseline characteristics including gender, age at diagnosis, cumulative anthracycline dose and exposure to chest irradiation.

The combined impact of dependency on caregivers, disability, and coping strategy on quality of life after ischemic stroke.

Background: To estimate the additional impact of coping and of being dependent on caregivers, over and above the large effects of disability on utility after ischemic stroke.

Methods: A total of 539 patients were recruited into an observational, retrospective study when returning for a check-up between 3 and 36 months after an ischemic stroke. Patients' modified Rankin Scale (mRS), dependency on caregivers, the Brandtstädter and Renner Coping questionnaire (with summary scores: Tenacity of Goal Pursuit (TGP) and Flexible Goal Adjustment (FGA) coping styles), EQ-5D-3 L and co-morbidities were evaluated.

The relationship between Home-time, quality of life and costs after ischemic stroke: the impact of the need for mobility aids, home and car modifications on Home-time.

Home-time (the number of days spent at home during the first 3 months after stroke) shows a strong association with the modified Rankin scale (mRS). We studied whether Home-time was also a determinant of quality-of-life and medical care costs after ischemic stroke, and assessed factors delaying discharge home. Five hundred and sixty nine patients participated in a retrospective study when returning for an in-person visit after an ischemic stroke.

Hospital financing of ischaemic stroke: determinants of funding and usefulness of DRG subcategories based on severity of illness.

Background: Several Western and Arab countries, as well as over 30 States in the US are using the "All-Patient Refined Diagnosis-Related Groups" (APR-DRGs) with four severity-of-illness (SOI) subcategories as a model for hospital funding. The aim of this study is to verify whether this is an adequate model for funding stroke hospital admissions, and to explore which risk factors and complications may influence the amount of funding.

Methods: A bottom-up analysis of 2496 ischaemic stroke admissions in Belgium compares detailed in-hospital resource use (including length of stay, imaging, lab tests, visits and drugs) per SOI category and calculates total hospitalisation costs.

What Impact Does Venous Thromboembolism and Bleeding Have on Cancer Patients' Quality of Life?

Background: Venous thromboembolism (VTE) is common in cancer patients and its treatment is associated with a high risk of recurrent VTE (rVTE) and bleeding.

Objectives: To analyze data from the Comparison of Acute Treatments in Cancer Hemostasis (CATCH) trial to describe the impact of rVTE and bleeding events on health-related quality of life.

Methods: The three-level EuroQol five-dimensional questionnaire (EQ-5D) data were collected monthly for up to 7 months in patients starting anticoagulation for newly diagnosed VTE.

Modified Rankin scale as a determinant of direct medical costs after stroke.

Background Resource use in the acute and subacute phases after stroke depends on the degree of disability. Aims To determine if direct costs after stroke also vary by level of disability as measured using the modified Rankin scale at the chronic stage after stroke. Methods In a multicentre study, we collected acute and chronic in- and outpatient resource use in survivors of ischemic stroke stratified by levels of disability according to the modified Rankin Scale.

Health state utilities in non-small cell lung cancer: An international study.

Aim: Quality of life weights (utilities) are an important input in economic evaluation and evidence suggests that there can be important differences between countries. This study was designed to capture utilities for metastatic non-small cell lung cancer and common grade III/IV toxicities associated with treatment from local populations in the United Kingdom, Australia, France, China, Taiwan, and Korea. Toxicities included neutropenia, febrile neutropenia, fatigue, diarrhea, nausea and vomiting, rash, bleeding, hypertension, and hair loss.

Bendamustine-rituximab: a cost-utility analysis in first-line treatment of indolent non-Hodgkin's lymphoma in England and Wales.

Objective: To evaluate the cost-effectiveness of bendamustine-rituximab (B-R) compared with CHOP-R (cyclophosphamide, doxorubicin, vincristine, prednisone, rituximab) and CVP-R (cyclophosphamide, vincristine, prednisone, rituximab) as first-line treatment for patients with advanced indolent non-Hodgkin's lymphoma (NHL).

Methods: A patient-level simulation was adapted from the model used by the University of Sheffield School of Health and Related Research (ScHARR) in a health technology appraisal of rituximab for first-line treatment of follicular lymphoma. This approach allowed modelling of the complex treatment pathways in indolent NHL.

Investigating incoherence gives insight: clopidogrel is equivalent to extended-release dipyridamole plus aspirin in secondary stroke prevention.

Objective: To identify confounding factors that may explain the incoherence between direct and indirect evidence in a published analysis comparing extended-release dipyridamole (ERDP) plus aspirin to clopidogrel for the reduction of stroke.

Study Design And Setting: An existing analysis was updated with new studies from a systematic literature review. Clinicians reviewed the studies for potential confounders.

Patient preferences for first-line oral treatment for mild-to-moderate ulcerative colitis: a discrete-choice experiment.

Background: Patients with ulcerative colitis (UC) frequently require long-term therapy to prevent relapse. Treatments such as 5-aminosalicylic acid (5-ASA [mesalazine]) are efficacious and well tolerated, but adherence to treatment is often poor.

Objective: This discrete-choice experiment (DCE) was conducted to estimate differences in patient preferences for 5-ASA treatment in mild-to-moderate UC based on levels of self-reported adherence.

Cost of hospitalization for cerebrovascular disorders in Belgium.

Background: There is only scarce information on the incidence and costs of stroke in Belgium. Knowledge of these figures permits targeted allocation of resources and aids cost efficacy estimates.

Methods: We analysed a nationwide administrative database used for reimbursement of hospitals in Belgium.

Methylphenidate delivery mechanisms for the treatment of children with attention deficit hyperactivity disorder: heterogeneity in parent preferences.

Objectives: Extended-release therapies avoid the need for children with attention-deficit/hyperactivity disorder (ADHD) to take medication at school. Recently a transdermal delivery system has been developed which can allow symptom control all day long but with greater dosing flexibility. This study explored the parents' preferences regarding oral and transdermal therapy.

The immediate early genes Fos and Egr1 become STAT1 transcriptional targets in the absence of STAT3.

Signal transducer and activators of transcription (STAT)1 and STAT3 cross-regulate their activity downstream of gp130 cytokines, and eliminating STAT3 leads to IFN-γ-like responses to IL-6 correlating with prolonged STAT1 phosphorylation. Here we demonstrate that the increased gp130-mediated induction of the IFN-γ-responsive interferon regulatory factor 1 gene observed in STAT3(-/-) cells correlates with prolonged STAT1 binding to its promoter. Intriguingly, gp130-mediated induction of the immediate early genes FBJ osteosarcoma oncogene and early growth response 1 is also prolonged in STAT3(-/-) cells, with STAT1 binding to their promoters.

Cost-effectiveness of adjunctive eptifibatide in patients undergoing coronary stenting in Germany.

Objective: To determine the cost-effectiveness of adding eptifibatide to the standard treatment for selected high-risk patients undergoing coronary stenting in Germany. Furthermore, to investigate the impact of several extrapolation methods on the results.

Methods: A Markov model was developed to reflect the clinical events in this specific patient population, including target vessel revascularization, myocardial infarction, and death.