The sphingolipidoses Fabry disease, Gaucher disease and Acid sphingomyelinase deficiency (ASMD) are the three most common lysosomal storage diseases for which treatment is currently available. Timely diagnosis with estimation of the disease severity and possibilities of follow-up of patients, whether or not under therapy, is crucial for providing good care and for the prevention of possible lethal complications. With this research we provide an efficient and sensitive detection method; its implementation in clinical practice could optimize the diagnosis and follow-up of patients with Gaucher, Fabry and ASMD.
View Article and Find Full Text PDFBackground: Chronic infection and inflammation of the lungs contribute significantly to disease progression in persons with cystic fibrosis (pwCF). Treatment regimens are largely based on isolating the putative causative pathogen(s) from respiratory samples using basic culturing methods. While this strategy has shown to be highly valuable in the management of CF, the approach is time-consuming and often misses detection of pathogenic microbes that are more difficult to culture, including Mycobacterium spp.
View Article and Find Full Text PDFBackground: Paediatric acute myeloid leukaemia (AML) is a challenging disease in low- and middle-income countries with limited African data. This study reviewed the existing literature regarding paediatric AML in Africa.
Methods: A literature search was performed using PubMed, Embase, Google Scholar, Research4Life, and African Journals Online for published studies on paediatric AML in Africa, with keywords acute myeloid leukaemia, children, Africa, and country-specific names for all the African countries.
Introduction: Real-world data on children with severe asthma is scarce. We report characteristics of children with severe asthma already on biologics, enrolled in the Severe Paediatric Asthma Collaborative in Europe, a clinical research collaboration of the European Respiratory Society.
Methods: We describe patient's characteristics including asthma control assessed with Global Initiative for Asthma (GINA) criteria, composite asthma severity index (CASI), exacerbations, unscheduled medical attendances, lung function and quality of life in children on biologic treatment because of severe asthma.
Objective: To present reference data for nocturnal oximetry (NOx) in infants without respiratory disorders and evaluate their diagnostic accuracy for distinguishing moderate-to-severe upper airway obstruction (UAO) from mild/no UAO.
Materials And Methods: Infants (aged 1-12 months) without respiratory disease hospitalized for common disorders (e.g.
Objective: To explore the effect of body mass index (BMI) variability on cardiometabolic risk in children with obesity.
Study Design: Children between 8 and 18 years, entering a 12-month inpatient or 18-month outpatient obesity treatment were studied. BMI, body composition, blood pressure, high-sensitivity C-reactive protein, lipid profile, insulin sensitivity, and endothelial function were assessed at baseline and follow-up (every 3 months for outpatients; at 12 and 18 months for inpatients).
Respiratory syncytial virus (RSV) remains a major global health issue. Therapeutic options are limited, but new prophylactics, all targeting the fusion (F) glycoprotein, were recently licensed. Although F sequence variation is limited, it is unclear if and how this variability translates to phenotypical differences.
View Article and Find Full Text PDFBackground: Excessive drinking among adolescents in Western Europe is prevalent, posing significant health risks and societal costs. Comprehensive data on adolescent drinking patterns is crucial for developing effective prevention strategies. Data on alcohol intoxication among adolescents provide valuable insights in this context.
View Article and Find Full Text PDFBackground: Premature neonates have an increased risk of long-term effects, including impaired respiratory and neurodevelopment. During NICU admission, they are exposed to endocrine-disrupting phthalates and alternative plasticizers. The objective was to study the association between cumulative NICU-based plasticizer exposure and respiratory and neurodevelopmental outcome during the first year of life.
View Article and Find Full Text PDF: This study investigates the incidence of acute alcohol intoxication (AAI) in adolescents in Belgium, by comparing nationwide estimations based on health insurance data with a recently published hospital chart study in Antwerp. In this way, the scope of AAI among adolescents in Belgium can be estimated more precisely. : Health insurance data collected by the Intermutualistic Agency (IMA) regarding 12- to 17-year-olds admitted at emergency departments in Antwerp in 2019-2021 and receiving a blood test to screen for blood alcohol concentration were validated by data derived from a recently conducted retrospective hospital chart study regarding AAI among adolescents in Antwerp.
View Article and Find Full Text PDFJ Paediatr Child Health
December 2024
Aim: Creating an overview of the existing literature about peer support in paediatrics, with a focus on children with chronic diseases.
Methods: An online search was conducted in MEDLINE and Web of Science. English, Dutch or French articles published between 1 January 2000 and 10 May 2023 were included, based on title and abstract.
Background: Computer Aided Lung Sound Analysis (CALSA) aims to overcome limitations associated with standard lung auscultation by removing the subjective component and allowing quantification of sound characteristics. In this proof-of-concept study, a novel automated approach was evaluated in real patient data by comparing lung sound characteristics to structural and functional imaging biomarkers.
Methods: Patients with cystic fibrosis (CF) aged > 5y were recruited in a prospective cross-sectional study.
Background: Alcohol consumption is prevalent among students, with a common tendency to overestimate peers' alcohol use, contributing to increased consumption. This misperception is evident among Flemish students. This study aimed to develop and assess a Social Norms Approach (SNA) intervention targeting Flemish students to correct misperceptions and subsequently reduce alcohol use.
View Article and Find Full Text PDFBackground: Diagnosis and treatment of obstructive sleep apnea (OSA) in infants and young children is challenging because of its clinical heterogeneity and lack of age-specific guidelines.
Aim: We report the management and treatment outcome of OSA in children below 2 years of age. Treatment decisions were based upon the pattern of upper airway (UA) obstruction, clinical presentation and OSA severity.
BMJ Open
March 2024
Introduction: Diffuse intrinsic pontine glioma (DIPG) and paediatric high-grade glioma (pHGG) are aggressive glial tumours, for which conventional treatment modalities fall short. Dendritic cell (DC)-based immunotherapy is being investigated as a promising and safe adjuvant therapy. The Wilms' tumour protein (WT1) is a potent target for this type of antigen-specific immunotherapy and is overexpressed in DIPG and pHGG.
View Article and Find Full Text PDFThis case-control study aimed to investigate the association between short-term (1 to 5 days) and medium-term (31 days) exposure to air pollutants (PM, PM, BC, NO) at home/daycare and the risk of 'severe bronchiolitis' (defined as 'requiring hospitalization for bronchiolitis') in children under 2 years in Antwerp, Belgium. We included 118 cases and 79 controls admitted to three general hospitals from October 2020 to June 2021. Exposure levels were predicted using an interpolation model based on fixed measuring stations.
View Article and Find Full Text PDFIntroduction: Asthma is a chronic condition that affects millions of adolescents and young adults (AYA) worldwide. The transition from pediatric to adult care presents unique challenges for this population, affecting their self-management, quality of life and overall health outcomes. This systematic review aims to consolidate the available evidence on challenges encountered by AYA with asthma during the transition period from child to AYA and on the key elements of transitional care for AYAs with asthma including the outcomes achieved, ultimately enhancing outcomes.
View Article and Find Full Text PDFBackground: Asthma is the most prevalent chronic respiratory condition in children. An asthma exacerbation (AE) is a frequent reason for emergency department (ED) visits. An important step in the management of a moderate to severe AE is the administration of systemic corticosteroids (SCS) within 1 h after ED presentation.
View Article and Find Full Text PDFThe aim of this review is to summarise evidence that became available after publication of the 2017 European Respiratory Society statement on the diagnosis and management of obstructive sleep apnoea syndrome (OSAS) in 1- to 23-month-old children. The definition of OSAS in the first 2 years of life should probably differ from that applied in children older than 2 years. An obstructive apnoea-hypopnoea index >5 events·h may be normal in neonates, as obstructive and central sleep apnoeas decline in frequency during infancy in otherwise healthy children and those with symptoms of upper airway obstruction.
View Article and Find Full Text PDFAntibiotics and cystic fibrosis transmembrane conductance regulator (CFTR) modulators play a pivotal role in cystic fibrosis (CF) treatment, but both have limitations. Antibiotics are linked to antibiotic resistance and disruption of the airway microbiome, while CFTR modulators are not widely accessible, and structural lung damage and pathogen overgrowth still occur. Complementary strategies that can beneficially modulate the airway microbiome in a preventive way are highly needed.
View Article and Find Full Text PDFBackground: Obesity is linked to several health complication, including Metabolic Dysfunction Associated Steatotic Liver Disease (MASLD). Adipose tissue hypoxia has been suggested as an important player in the pathophysiological mechanism leading to chronic inflammation in obesity, and in the progression of MASLD. The study aims to investigate the effect of progressive obesity on adipose and liver tissue hypoxia.
View Article and Find Full Text PDFFront Pediatr
November 2023
Background: Although cystic fibrosis (CF) standards of care have been produced and regularly updated, they are not specifically targeting at the adult population. The ECFS Standards of Care Project established an international task force of experts to identify quality standards for adults with CF and assess their adherence.
Methods: This study was composed of two phases.