Priorities for medical device regulatory approval: a report from the European Society of Cardiology Cardiovascular Round Table.

The European Union (EU) Medical Device Regulation increased regulatory scrutiny to improve the safety and performance of new medical devices. An equally important goal is providing timely access to innovative devices to benefit patient care. The European Society of Cardiology strongly advocates for the evolution of the Medical Device Regulation system to facilitate priority access for innovative devices for unmet needs and orphan cardiovascular (CV) medical devices in EU countries.

Concomitant medications in patients with metastatic urothelial carcinoma receiving enfortumab vedotin: real-world data from the ARON-2 study.

Patients with metastatic urothelial carcinoma (mUC) are typically elderly and often have other comorbidities that require the use of concomitant medications. In our study we evaluated the association of concomitant use of antibiotics (ATBs), proton pump inhibitors (PPIs), corticosteroids, statins, metformin and insulin with patient outcomes and we validated the prognostic role of a concomitant drug score in mUC patients treated with enfortumab vedotin (EV) monotherapy. Data from 436 patients enrolled in the ARON-2 retrospective study were analyzed according to the concomitant medications used at baseline.

Assessing the predicted impact of single amino acid substitutions in MAPK proteins for CAGI6 challenges.

New thermodynamic and functional studies have been recently conducted to evaluate the impact of amino acid substitutions on the Mitogen Activated Protein Kinases 1 and 3 (MAPK1/3). The Critical Assessment of Genome Interpretation (CAGI) data provider, at Sapienza University of Rome, measured the unfolding free energy and the enzymatic activity of a set of variants (MAPK challenge dataset). Thermodynamic measurements for the denaturant-induced equilibrium unfolding of the phosphorylated and unphosphorylated forms of the MAPKs were obtained by monitoring the far-UV circular dichroism and intrinsic fluorescence changes as a function of denaturant concentration.

More than meets the eye: phenomenological insights into the functioning of people with lipoedema.

Purpose: The aim of this study was to explore the functioning of people with lipoedema from their perspective.

Methods: This was a qualitative study following a phenomenological perspective using individual in-depth interviews with a convenience sample of 13 Dutch people with lipoedema. The data were analysed by using an interpretative phenomenological approach.

Asymptomatic and oligosymptomatic states of dysferlinopathy.

Dysferlinopathy is a phenotypically heterogeneous, inherited, progressive muscular dystrophy caused by mutations in the gene. Dysferlinopathy is marked by elevated serum creatine kinase (CK) and can in some cases manifest as hyperCKemia in asymptomatic or low-symptom states. Here, we describe the clinical signs and symptoms and laboratory and imaging results with quantitative MRI analysis of eight pediatric patients at asymptomatic and oligosymptomatic states of dysferlinopathy (aged 3-14 years).

International collaboration to improve knowledge on myotonic dystrophy type 2.

Background: The TREAT-NMD Global Registry Network is a global collaboration of neuromuscular disease registries, including myotonic dystrophy type 2 (DM2), which aims to facilitate collaborative research and clinical trials.

Objectives: This study aimed to assess DM2 patients included in the network, and to analyse their socio-demographic and clinical features.

Methods: Data were collected through email surveys sent to 16 TREAT-NMD myotonic dystrophy core member registries.

Rare Case of Valve-Sparing Root Reimplantation in a Patient With an Anomalous Circumflex Artery.

We present a case of valve-sparing root reimplantation in a patient with severe aortic regurgitation, in the setting of a bicuspid aortic valve with an aberrant circumflex coronary artery arising from the right coronary artery, thus rendering standard dissection of the aortic root during a David procedure challenging and risky.

Clinical utility of coronary CT angiography to guide PCI: a survey among P4 investigators.

Background: Coronary CT Angiography (CTA) is increasingly being used to plan percutaneous coronary intervention (PCI), offering detailed pre-procedural anatomical and physiological insights. The Precise Procedural and PCI Plan (P4) study evaluates the efficacy of CT- versus intravascular ultrasound-guided PCI. The aim of this study was to assess the utility of various CT-guided PCI tools and strategies as perceived by experienced operators within the context of the P4 study.

Progression-free survival as a potential surrogate end point for overall survival in advanced cervical carcinoma.

Objective: This systematic review and meta-analysis was performed to study the potential of progression-free survival as a surrogate end point for trials evaluating the use of systemic therapy in advanced cervical cancer.

Methods: We performed a systematic review selecting phase II and III randomized trials including locally advanced, recurrent, and metastatic cervical cancer, with mature progression-free survival and overall survival data. Our study adhered to the Reporting Items for Systematic Reviews and Meta-Analyses checklist and was registered in International Prospective Register of Systematic Reviews (CRD42023405604).

Valve sparing aortic root replacement in bicuspid aortic valves, the reimplantation technique: A multicenter study.

Objective: Reported outcome in patients with bicuspid aortic valves(BAV) undergoing valve-sparing root replacement(VSRR) is scarce. This study aims to evaluate outcomes in patients with BAV, utilizing the reimplantation (David)technique.

Methods: Consecutive adult patients, with BAV, aortic root aneurysm and/or valve insufficiency(AI) undergoing VSRR(reimplantation)), were included, from 5 centers experienced in reimplantation-procedure.

The 2024 international survey of platelet products and practice.

Background: Previously, two international surveys have addressed the wider application of platelet collection by apheresis technology and practical issues of platelet transfusion.

Study Design And Methods: A questionnaire was sent to persons with leadership roles related to blood banking and transfusion medicine in their countries/areas/centers, to document the implementation of modern technologies for platelet manufacturing, preservation, and transfusion risk reduction.

Results: Responses to the questionnaire finally came from 52 contributors in 40 countries/areas.

Phenotype variability and therapeutic response to Patisiran in patients with hereditary transthyretin amyloidosis: a Belgian real-world experience.

Introduction: Hereditary transthyretin amyloidosis (hATTRv) is a rare, genetic, adult-onset, multisystemic disorder which can affect diverse organs, including peripheral nerves, heart, kidneys, gastrointestinal tract, liver, skin and eyes. Currently, several disease-modifying treatments for hATTRv are available in Belgium including the TTR stabilizer tafamidis and TTR mRNA silencers patisiran and vutrisiran. Patisiran contains a small interfering RNA encapsulated into a lipid nanoparticle to deliver to hepatocytes, the main source of TTR protein production, thereby reducing TTR production.

Sex-Specific Association of Myocardial Fibrosis With Mortality in Patients With Aortic Stenosis.

Importance: Myocardial fibrosis in aortic stenosis (AS) may exhibit sex differences. However, its prognostic significance in women with AS remains unclear.

Objective: To investigate sex differences in myocardial fibrosis assessed by cardiovascular magnetic resonance (CMR) and evaluate its prognostic value in women and men with AS.

Residential exposome and the risk of coronary obstruction and myocardial ischemia detected by coronary computed tomography.

Background And Aims: To assess independent impacts of air and noise pollution, and residential green exposure on non-invasively derived coronary obstruction and myocardial ischemia.

Methods: Consecutive patients screened by coronary computed tomography angiography (CCTA) for stable chest pain between 01/01/2019 and 31/12/2020 were included in a registry. Exposure associations between residential PM2.

Developing and implementing a nurse-delivered and a web-based dyadic psychoeducational program for people with advanced cancer and their family caregivers: sharing experiences from a three-arm international randomized controlled trial (DIAdIC).

Background: Each year millions are diagnosed with cancer, impacting both patients and caregivers. Few interventions target both patients and family caregivers together, despite their shared experiences. While dyadic psychoeducational programs are gaining attention, evidence on developing and implementing these in international trials is limited.

Goals of care discussions and treatment limitation decisions in European acute geriatric units: a one-day cross-sectional study.

Background: It is important to pursue goal-concordant care and to prevent non-beneficial interventions in older people.

Aim: To describe serious illness communication and decision-making practices in hospitalised older people in Europe.

Setting/participants: Data on advance directives, goals of care (GOC) discussions and treatment limitation decisions were collected about patients aged 75-years and older admitted to 23 European acute geriatric units (AGUs).

Cardiovascular Safety in Oncology Clinical Trials: JACC: CardioOncology Primer.

The development of novel treatments has improved cancer outcomes but may result in cardiovascular toxicities. Traditional approaches to clinical trial safety evaluation have limitations in their ability to detect signals of cardiovascular risk. Mechanisms to increase power and specificity to clarify cardiovascular safety are required.

Deciding whether to do elective neck dissection in patients with salivary gland tumors with no evidence of neck lymph node metastasis.

Aim: The treatment of salivary gland cancers with clinically negative neck is controversial, with divergent guidelines about elective neck dissection (END). Even though these guidelines are widely used, they mostly rely on retrospective research that are subject to methodological flaws and selection bias.

Methods: A critical narrative review.

Are Patients with Prostate Imaging Reporting and Data System 5 Lesions Eligible for Active Surveillance? A Multicentric European Study.

Background And Objective: Patients with Prostate Imaging Reporting and Data System (PI-RADS) 5 lesions are at a high risk of clinically significant prostate cancer (PCa), extracapsular extension, and biochemical recurrence (BCR) after local treatment. Managing these patients with active surveillance (AS) can be particularly challenging when targeted biopsies indicate favorable-risk tumors. This study aims to evaluate the outcomes of patients with PI-RADS 5 lesions managed with AS.

First-year treatment response predicts the following 5-year disease course in patients with relapsing-remitting multiple sclerosis.

Predicting long-term prognosis and choosing the appropriate therapeutic approach in patients with Multiple Sclerosis (MS) at the time of diagnosis is crucial in view of a personalized medicine. We investigated the impact of early therapeutic response on the 5-year prognosis of patients with relapsing-remitting MS (RRMS). We recruited patients from MSBase Registry covering the period between 1996 and 2022.

Explainable time-to-progression predictions in multiple sclerosis.

Background: Prognostic machine learning research in multiple sclerosis has been mainly focusing on black-box models predicting whether a patients' disability will progress in a fixed number of years. However, as this is a binary yes/no question, it cannot take individual disease severity into account. Therefore, in this work we propose to model the time to disease progression instead.

INHERITED RETINAL DISEASE GENE PANEL IN POSTERIOR OR PANUVEITIS WITH DYSTROPHIC FEATURES.

Purpose: To evaluate the presence of American College of Medical Genetics and Genomics class 3, 4, and 5 genetic variants in inherited retinal disease (IRD) genes in posterior or panuveitis with dystrophic features (PUD) in a Belgian cohort.

Methods: Multicentric, retrospective study of PUD cases diagnosed between January 2012 and February 2022. Inherited retinal disease gene panels were analyzed in every patient.

Disease associated malnutrition in pediatrics - what is new?

Purpose Of Review: Disease associated malnutrition (DAM) remains an important concern in the care of hospitalized children and children with a chronic disease. This review focused on pediatric literature published since 2023 on the prevalence, assessment and treatment of DAM in different settings.

Recent Findings: The prevalence of DAM depends on a variety of factors.