Sports participation and lifestyle in middle-aged adults with congenital heart disease.

Background: Sports are associated with numerous physiological and psychological benefits. However, it is unclear to what extent adults with congenital heart disease(CHD) participate in sports and whether this is safe. Furthermore, little is known about lifestyle habits in this group.

Time-restricted versus standard-duration immunosuppression after allogeneic hematopoietic stem cell transplantation: Results of the prospective randomized HOVON-96 trial.

Cyclosporine A combined with mycophenolate mofetil (CsA/MMF) has become an established regimen for the prevention of graft-versus-host disease (GVHD) following non-myeloablative (NMA) allogeneic hematopoietic stem cell transplantation (alloHSCT). However, the optimal duration of immunosuppression (IS) has not yet been defined and overtreatment is of concern. We hypothesized that time-restricted IS with CsA/MMF would increase the proportion of patients with non-severe GVHD compared to standard-duration IS, thereby resulting in reduction of the relapse rate and improvement of progression-free survival (PFS) and overall survival (OS).

Mapping challenges in the accessibility of treatment products for urea cycle disorders: A survey of European healthcare professionals.

Current management guidelines for urea cycle disorders (UCDs) offer clear strategies, incorporating both authorized and non-authorized medicinal products (including intravenous formulations and products regulated as food). These varying product categories are subject to specific accessibility challenges related to availability, reimbursement, and pricing. The aim of this study is to identify potential obstacles to optimal UCD treatment implementation in European clinical practice.

The Early Childhood Development of Pediatric Burn Patients.

Our study aimed to provide a description of the early childhood development of pediatric burn patients relative to Dutch reference values, using both pre- and post-burn data from the Dutch Development Instrument and the -score. Data from the Dutch Development Instrument were used to calculate the -score and age-standardized -score. Similar to a growth chart, the -score was used to plot pediatric burn patients' development relative to Dutch reference values for their age.

Causes of death among patients diagnosed with chronic lymphocytic leukemia: A population-based study in the Netherlands, 1996-2020.

Chronic lymphocytic leukemia (CLL) manifests heterogeneously with varying outcomes. This population-based study examined causes of death (CODs), as registered by the physician who established the death, among 20,588 CLL patients diagnosed in the Netherlands between 1996 and 2020. Utilizing cause-specific flexible parametric survival models, we estimated cause-specific hazard ratios (HRs) and cumulative incidences of death due to CLL, solid malignancies, other hematological malignancies, infections, and other causes.

Playing sport as a central-line carrier: a survey to collect the European pediatric intestinal failure centers' view.

Background: The administration of home parenteral nutrition improves quality of life for patients with intestinal failure, thus fostering their will to actively participate to social activities. Nevertheless, sports participation can be risky for patients with a central venous catheter (CVC). Despite literature thoroughly proving the positive impact of sports on motor-psychosocial development, no consistent evidence assessing its role on central-line complications is available.

Co-creation of a step-by-step guide for specifying the test-management pathway to formulate focused guideline questions about healthcare related tests.

Background: Guideline development on testing is known to be difficult for guideline developers. It requires consideration of various aspects, such as accuracy, purpose of testing, and consequences on management and people-important outcomes. This can be outlined in a test-management pathway.

Development and validation of prediction models for fetal growth restriction and birthweight: an individual participant data meta-analysis.

Background: Fetal growth restriction is associated with perinatal morbidity and mortality. Early identification of women having at-risk fetuses can reduce perinatal adverse outcomes.

Objectives: To assess the predictive performance of existing models predicting fetal growth restriction and birthweight, and if needed, to develop and validate new multivariable models using individual participant data.

Oncogenic and immunological targets for matched therapy of pediatric blood cancer patients: Dutch iTHER study experience.

Over the past 10 years, institutional and national molecular tumor boards have been implemented for relapsed or refractory pediatric cancer to prioritize targeted drugs for individualized treatment based on actionable oncogenic lesions, including the Dutch iTHER platform. Hematological malignancies form a minority in precision medicine studies. Here, we report on 56 iTHER leukemia/lymphoma patients for which we considered cell surface markers and oncogenic aberrations as actionable events, supplemented with ex vivo drug sensitivity for six patients.

Development, validation and long-term evaluation of a liquid chromatography-tandem mass spectrometry method for simultaneous quantification of amiodarone, desethylamiodarone and mexiletine in human plasma and serum.

Amiodarone and mexiletine are used for ventricular arrhythmias, for which a combination therapy of both anti-arrhythmic drugs (AADs) is not uncommon. Therapeutic drug monitoring (TDM) can be beneficial for clinical guidance of therapy, especially to correctly identify adverse events. Desethylamiodarone, the active metabolite of amiodarone, accumulates over time and is associated with serious adverse events.

Proteome encoded determinants of protein sorting into extracellular vesicles.

Extracellular vesicles (EVs) are membranous structures released by cells into the extracellular space and are thought to be involved in cell-to-cell communication. While EVs and their cargo are promising biomarker candidates, sorting mechanisms of proteins to EVs remain unclear. In this study, we ask if it is possible to determine EV association based on the protein sequence.

Required knowledge for guideline panel members to develop healthcare related testing recommendations: a developmental study.

Objectives: To define the minimum knowledge required for guideline panel members (healthcare professionals and consumers) involved in developing recommendations about healthcare related testing.

Study Design And Setting: A developmental study with a multistaged approach. We derived a first set of knowledge components from literature and subsequently performed semistructured interviews with 9 experts.

International Migration and Cardiovascular Health: Unraveling the Disease Burden Among Migrants to North America and Europe.

Europe and North America are the 2 largest recipients of international migrants from low-resource regions in the world. Here, large differences in cardiovascular disease (CVD) morbidity and death exist between migrants and the host populations. This review discusses the CVD burden and its most important contributors among the largest migrant groups in Europe and North America as well as the consequences of migration to high-income countries on CVD diagnosis and therapy.

Gastric emptying and myoelectrical activity testing in children with esophageal atresia: A pilot study.

Objectives: Abnormalities of gastric function in children with esophageal atresia (EA) could potentially contribute to gastrointestinal symptoms and reduced quality of life (QOL). Therefore, we aimed to determine the feasibility and clinical usefulness of gastric function testing in children with EA.

Methods: The validated PedsQL Gastrointestinal Symptoms Questionnaire (PedsQL-GI) was completed to assess gastrointestinal symptoms and symptom-related QOL.

Orodental injury and mouthguard usage in Dutch and international field hockey.

Background: Mouthguards are used to prevent players from orodental injuries in field hockey. However, such injuries are still a common problem. This study describes the prevalence of orodental injury and the related mouthguard usage in field hockey.

Learning and metacognition under volatility in GD: Lower learning rates and distorted coupling between action and confidence.

Background And Aims: Decisions and learning processes are under metacognitive control, where confidence in one's actions guides future behaviour. Indeed, studies have shown that being more confident results in less action updating and learning, and vice versa. This coupling between action and confidence can be disrupted, as has been found in individuals with high compulsivity symptoms.

Studying the topology of peroxisomal acyl-CoA synthetases using self-assembling split sfGFP.

Peroxisomes are membrane-bounded organelles that contain enzymes involved in multiple lipid metabolic pathways. Several of these pathways require (re-)activation of fatty acids to coenzyme A (CoA) esters by acyl-CoA synthetases, which may take place inside the peroxisomal lumen or extraperoxisomal. The acyl-CoA synthetases SLC27A2, SLC27A4, ACSL1, and ACSL4 have different but overlapping substrate specificities and were previously reported to be localized in the peroxisomal membrane in addition to other subcellular locations.