Combined use of a broad-panel respiratory multiplex PCR and procalcitonin to reduce duration of antibiotics exposure in patients with severe community-acquired pneumonia (MULTI-CAP): a multicentre, parallel-group, open-label, individual randomised trial conducted in French intensive care units.

Introduction: At the time of the worrying emergence and spread of bacterial resistance, reducing the selection pressure by reducing the exposure to antibiotics in patients with community-acquired pneumonia (CAP) is a public health issue. In this context, the combined use of molecular tests and biomarkers for guiding antibiotics discontinuation is attractive. Therefore, we have designed a trial comparing an integrated approach of diagnosis and treatment of severe CAP to usual care.

Health technology assessment for the acute and preventive treatment of migraine: A position statement of the International Headache Society.

The Clinical Trials Subcommittee of the International Headache Society presents the first Health Technology Assessment for the Acute Treatment of Migraine Attacks and Prevention of Migraine. Health technology assessments are systematic evaluations of the properties, effects, and consequences of healthcare technologies; this position statement is designed to inform decision makers about access to and reimbursement for medications and devices for the acute and preventive treatment of migraine. This position statement extends beyond the already available guidelines on randomized controlled trials for migraine to incorporate real-world evidence and a synthetic approach for considering multiple data sources and modelling methods when assessing the value of migraine treatments.

Cardiovascular Magnetic Resonance May Avoid Unnecessary Coronary Angiography in Patients With Unexplained Left Ventricular Systolic Dysfunction: A Retrospective Diagnostic Pilot Study.

Background: Coronary angiography (CA) is usually performed in patients with reduced left ventricular ejection fraction (LVEF) to search ischemic cardiomyopathy. Our aim was to examine the agreement between CA and cardiovascular magnetic resonance (CMR) imaging among a cohort of patients with unexplained reduced LVEF, and estimate what would have been the consequences of using CMR imaging as the first-line examination.

Methods: Three hundred five patients with unexplained reduced LVEF of ≤45% who underwent both CA and CMR imaging were retrospectively registered.

Guidance for the Management of Patients with Vascular Disease or Cardiovascular Risk Factors and COVID-19: Position Paper from VAS-European Independent Foundation in Angiology/Vascular Medicine.

COVID-19 is also manifested with hypercoagulability, pulmonary intravascular coagulation, microangiopathy, and venous thromboembolism (VTE) or arterial thrombosis. Predisposing risk factors to severe COVID-19 are male sex, underlying cardiovascular disease, or cardiovascular risk factors including noncontrolled diabetes mellitus or arterial hypertension, obesity, and advanced age. The VAS-European Independent Foundation in Angiology/Vascular Medicine draws attention to patients with vascular disease (VD) and presents an integral strategy for the management of patients with VD or cardiovascular risk factors (VD-CVR) and COVID-19.

Cost-effectiveness of a cardiac contractility modulation device in heart failure with normal QRS duration.

Aims: The objective of this paper is to assess whether cardiac contractility modulation (via the Optimizer System) plus standard of care (SoC) is a cost-effective treatment for people with heart failure [New York Heart Association (NYHA) III, left ventricular ejection fraction of 25-45%, and narrow QRS] compared against SoC alone from the perspective of the English National Health Service.

Methods And Results: We developed a regression equation-based cost-effectiveness model, using individual patient data from three randomized control trials (FIX-HF-5 Phases 1 and 2, and FIX-HF-5C) to populate the majority of parameters. A series of regression equations predicted NYHA class over time, mortality, all-cause hospitalization rates, and health-related quality of life.

Therapeutic hypothermia for acute ischaemic stroke. Results of a European multicentre, randomised, phase III clinical trial.

Introduction: We assessed whether modest systemic cooling started within 6 hours of symptom onset improves functional outcome at three months in awake patients with acute ischaemic stroke.

Patients And Methods: In this European randomised open-label clinical trial with blinded outcome assessment, adult patients with acute ischaemic stroke were randomised to cooling to a target body temperature of 34.0-35.

Transcultural mediation programme in a paediatric hospital in France: qualitative and quantitative study of participants' experience and impact on hospital costs.

Objective: In France, immigrants with chronic diseases encounter numerous difficulties in gaining access to care and then in its initiation and organisation, difficulties only partly explained by socioeconomic factors. A consultation programme has been set up in Necker Hospital in Paris to help families and professionals deal with these situations. The objective of this study was to assess the economic impact and the experience of this consultation.

Paris MEM: a study protocol for an effectiveness and efficiency trial on the treatment of traumatic stress in France after the 2015-16 terrorist attacks.

Background: The Paris and Nice terrorist attacks affected a thousand of trauma victims and first-line responders. Because there were concerns that this might represent the first of several attacks, there was a need to quickly enhance the local capacities to treat a large number of individuals suffering from trauma-related disorders. Since Reconsolidation Therapy (RT) is brief, relatively easy to learn, well tolerated and effective, it appeared as the ideal first-line treatment to teach to clinicians in this context.

Cost-effectiveness of point-of-care viscoelastic haemostatic assays in the management of bleeding during cardiac surgery: protocol for a prospective multicentre pragmatic study with stepped-wedge cluster randomised controlled design and 1-year follow-up (the IMOTEC study).

Introduction: During cardiac surgery-associated bleeding, the early detection of coagulopathy is crucial. However, owing to time constraints or lack of suitable laboratory tests, transfusion of haemostatic products is often inappropriately triggered, either too late (exposing to prolonged bleeding and thus to avoidable administration of blood products) or blindly to the coagulation status (exposing to unnecessary haemostatic products administration in patients with no coagulopathy). Undue exposition to transfusion risks and additional healthcare costs may arise.

Comparative evaluation of minimally invasive 'tibial tuberoplasty' surgical technique versus conventional open surgery for Schatzker II-III tibial plateau fractures: design of a multicentre, randomised, controlled and blinded trial (TUBERIMPACT study).

Introduction: Fractures of the tibial plateau are in constant progression. They affect an elderly population suffering from a number of comorbidities, but also a young population increasingly practicing high-risk sports. The conventional open surgical technique used for tibial plateau fractures has several pitfalls: bone and skin devascularisation, increased risks of infection and functional rehabilitation difficulties.

Hereditary angioedema, emergency management of attacks by a call center.

Objective: Hereditary angiœdema (HAE) is a rare autosomal dominant disease characterized by recurrent, unpredictable, potentially life-threatening swelling. Objective is to assess the management of the acute HAE attacks in the real life setting through a call center in France.

Methods: A pre-specified ancillary study of SOS-HAE, a cluster-randomized prospective multicenter trial, was conducted.

Clinical and economic aspects of newborn screening for severe combined immunodeficiency: DEPISTREC study results.

Purpose: Severe combined immunodeficiency (SCID) refers to a group of genetic disorders characterized by greatly compromised cellular and humoral immunity. Children with SCID are asymptomatic at birth, but they die from infections within the first months of life if not treated. Quantification of T-cell receptor excision circles is an extremely sensitive screening method for detecting newborns who may have SCID.

Cost-effectiveness of the COPD Patient Management European Trial home-based disease management program.

Purpose: Efficient management of COPD represents an international challenge. Effective management strategies within the means of limited health care budgets are urgently required. This analysis aimed to evaluate the cost-effectiveness of a home-based disease management (DM) intervention vs usual management (UM) in patients from the COPD Patient Management European Trial (COMET).

Trends in resource use and effectiveness of ultrasound detection of fetal structural anomalies in France: a multiple registry-based study.

Objective: To analyse trends in the number of ultrasound examinations in relation to the effectiveness of prenatal detection of birth defects using population-based data in France.

Design: A multiple registry-based study of time trends in resource use (number of ultrasounds) and effectiveness (proportion of cases prenatally diagnosed).

Setting: Three registries of congenital anomalies and claims data on ultrasounds for all pregnant women in France.

Experimentation to favor innovation: Promoting the success of Article 51 transformation projects.

Article 51 of the French social security financing act for 2018 creates a derogatory funding system to support experimentation with new ways of organizing care pathways. The growing interest local leaders have expressed for this very open framework has led to a large number of candidate projects presented to public authorities in charge of the system. In order to sustain this dynamic movement and favor the success of these experimental projects, our work focused on clarifying the roles and responsibilities of project leaders and the need for a specific evaluation system that takes into account the evolving nature of these projects and the need for operational adaptability.

Is there an optimal strategy for real-time continuous glucose monitoring in pediatrics? A 12-month French multi-center, prospective, controlled randomized trial (Start-In!).

Aim: To compare the efficacy of three strategies for real-time continuous glucose monitoring (RT-CGM) over 12 months in children and adolescents with type 1 diabetes.

Methods: A French multicenter trial (NCT00949221) with a randomized, controlled, prospective, open, and parallel-group design was conducted. After 3 months of RT-CGM, patients were allocated to one of three groups: return to self-monitoring of blood glucose, continuous CGM (80% of the time), or discontinuous CGM (40% of the time).

Effectiveness and costs of non-invasive foetal RHD genotyping in rhesus-D negative mothers: a French multicentric two-arm study of 850 women.

Background: The determination of foetal Rhesus D (RHD) status allows appropriate use of IgRh prophylaxis by restricting its use to cases of RHD feto-maternal incompatibilities. There is a degree of uncertainty about the cost-effectiveness of foetal RHD determination, yet screening programs are being introduced into clinical practice in many countries. This paper evaluates the impact of non-invasive foetal Rhesus D (RHD) status determination on the costs of managing RHD-negative pregnant women and on the appropriate use of anti-D prophylaxis in a large sample of RHD-negative pregnant women using individual prospectively collected clinical and economic data.

Access to innovative drugs for metastatic lung cancer treatment in a French nationwide cohort: the TERRITOIRE study.

Background: Territorial differences in the access to innovative anticancer drugs have been reported from many countries. The objectives of this study were to evaluate access to innovative treatments for metastatic lung cancer in France, and to assess whether socioeconomic indicators were predictors of access at the level of the municipality of residence.

Methods: All incident cases of metastatic lung cancer hospitalised for a chemotherapy in public hospitals in 2011 were identified from the French National Hospital discharge database.

Quantifying patient centered outcomes associated with the use of bilateral endobronchial coil treatment in patients with severe emphysema.

Objective: To determine the impact of endobronchial coils on health-related quality-of-life (HRQoL). This paper utilizes trial data to identify the predictors of HRQoL in patients with severe emphysema, and subsequently estimates the impact of a new treatment on HRQoL (measured by utilities). These utility estimates are used to generate indicative long-term QALY estimates for a range of clinically plausible scenarios as a precursor to cost-effectiveness analyses.

Specialist Advice Support for Management of Severe Hereditary Angioedema Attacks: A Multicenter Cluster-Randomized Controlled Trial.

Study Objective: Hereditary angioedema is a rare disease associated with unpredictable, recurrent attacks of potentially life-threatening edema. Management of severe attacks is currently suboptimal because emergency medical teams are often unaware of new specific treatments. The objective of this trial is to test whether a dedicated national telephone care-management strategy would reduce resource use during severe hereditary angioedema attacks.

Friedreich and dominant ataxias: quantitative differences in cerebellar dysfunction measurements.

Background: Sensitive outcome measures for clinical trials on cerebellar ataxias are lacking. Most cerebellar ataxias progress very slowly and quantitative measurements are required to evaluate cerebellar dysfunction.

Methods: We evaluated two scales for rating cerebellar ataxias: the Composite Cerebellar Functional Severity (CCFS) Scale and Scale for the Assessment and Rating of Ataxia (SARA), in patients with spinocerebellar ataxia (SCA) and controls.