535 results match your criteria: "Willem Alexander Children's Hospital[Affiliation]"

Objective: Fetomaternal transfusion (FMT) is associated with increased perinatal mortality and morbidity, but data on postnatal outcomes are scarce. Our aim was to determine the incidence of adverse short-termand long-term sequelae of severe FMT.

Design: Retrospective cohort study.

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Background: Extracorporeal membrane oxygenation (ECMO) can act as a bridge to recovery in both pre-and postoperative patients with transposition of the great arteries (TGA). However, literature on its use in these patients is scarce.

Methods: Retrospective single-centre cohort study encompassing all TGA patients who received ECMO between January 2009 and March 2024.

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Objective: Despite lack of evidence supporting efficacy, prophylactic fresh frozen plasma and Octaplas transfusions may be administered to very preterm infants to reduce bleeding risk. International variation in plasma transfusion practices in neonatal intensive care units (NICUs) is poorly understood, therefore, we aimed to describe neonatal plasma transfusion practice in Europe.

Design: Prospective observational study.

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Background And Aims: Refractory celiac disease type II (RCDII) is characterized by a clonally expanded aberrant cell population in the small intestine. The role of other tissue-resident immune subsets in RCDII is unknown. Here, we characterized CD8 and CD4 T cells in RCDII duodenum at the single-cell level and .

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Background: Treatment for childhood cancer may increase the risk of long-term pulmonary complications and dysfunction. Pulmonary surveillance is recommended after established pulmonary toxic exposures, including bleomycin, busulfan, carmustine (BCNU), lomustine (CCNU), radiotherapy to a field exposing the lungs, and pulmonary surgery. However, the role of cyclophosphamide as a pulmonary toxic agent is debated.

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Variations and Opportunities in Postnatal Management of Hemolytic Disease of the Fetus and Newborn.

JAMA Netw Open

January 2025

Division of Neonatology, Department of Pediatrics, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, the Netherlands.

Importance: Preventive efforts in pregnancy-related alloimmunization have considerably decreased the prevalence of hemolytic disease of the fetus and newborn (HDFN). International studies are therefore essential to obtain a deeper understanding of the postnatal management and outcomes of HDFN. Taken together with numerous treatment options, large practice variations among centers may exist.

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Importance: Intraventricular hemorrhage (IVH) has been described to typically occur during the early hours of life (HOL); however, the exact time of onset is still unknown.

Objective: To investigate the temporal distribution of IVH reported in very preterm neonates.

Data Sources: PubMed, Embase, Cochrane Library, and Web of Science were searched on May 9, 2024.

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Loss-of-function GHSR variants are associated with short stature and low IGF-I.

J Clin Endocrinol Metab

January 2025

Division of Pediatric Endocrinology, Department of Pediatrics, Willem-Alexander Children's Hospital, Leiden University Medical Centre, Leiden, The Netherlands.

Context: The growth hormone (GH) secretagogue receptor, encoded by GHSR, is expressed on somatotrophs of the pituitary gland. Stimulation with its ligand ghrelin, as well as its constitutive activity, enhances GH secretion. Studies in knock-out mice suggest that heterozygous loss-of-function of GHSR is associated with decreased GH response to fasting, but patient observations in small case reports have been equivocal.

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Proximate cord insertion in monochorionic twins with selective fetal growth restriction.

Am J Obstet Gynecol MFM

January 2025

Division of Neonatology, Department of Pediatrics, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands (Spekman, Ros, van Klink, Groene, and Lopriore).

Background: Monochorionic (MC) twins share a single placenta which can be unequally shared, leading to selective fetal growth restriction (sFGR). Limited data is available on the prevalence and clinical consequences of proximate cord insertion (PCI) in sFGR pregnancies.

Objective: We aimed to investigate the prevalence of PCI in MC placentas with and without sFGR and per type of sFGR, and study the placental characteristics and perinatal outcome of PCI in sFGR pregnancies.

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Critical procedural steps in intrauterine transfusion: Delphi survey of international experts.

Ultrasound Obstet Gynecol

January 2025

Division of Fetal Medicine, Department of Obstetrics and Gynecology, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands.

Objective: To determine consensus, using Delphi methodology, on the critical procedural steps for intravascular intrauterine transfusion (IUT) for the treatment of fetal anemia.

Methods: We conducted a two-part Delphi survey of international experts in fetal intervention. The first round of the survey proposed 32 potentially critical steps for the IUT procedure.

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Introduction: Transposition of the great arteries (TGA), especially with intact ventricular septum (TGA-IVS), presents unique challenges during fetal-to-neonatal transition, which can contribute to developing persistent pulmonary hypertension of the newborn (PPHN).

Case Presentation: A male newborn with TGA-IVS, delivered via caesarean section, presented with hypoxemia and tachycardia immediately after birth (preductal SpO: 50-60%, post-ductal SpO: 70-75%). Echocardiography revealed a floppy interatrial septum and two interatrial connections with bidirectional shunting.

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Selective termination: a life-saving procedure for complicated monochorionic gestations.

J Perinat Med

December 2024

Fetal Care and Surgery Center (FCSC), Division of Fetal Medicine and Surgery, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.

Monochorionic twin pregnancies are a subset of twin pregnancies that face potential complications related to a shared circulation between the fetuses. These complications are related to anastomotic placental vessels connecting the cardiovascular systems of the two fetuses, which can result in significant sequela if one twin experiences intrauterine death. The sudden cardiovascular collapse in this scenario leads to a massive blood shift away from the healthy co-twin, significantly jeopardizing its life and long-term neurodevelopmental outcome.

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Background And Objective: Vancomycin is a glycopeptide antibiotic used for the treatment of severe gram-positive infections. Despite decades of clinical experience, optimized dosing for vancomycin in pediatric populations still warrants further investigation. Patients admitted to the pediatric intensive care unit (PICU) after cardiac surgery are often treated with vancomycin in case of (suspected) infection.

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International Multicenter Cohort Study on Beta-Blocker-Free Treatment Strategies for Catecholaminergic Polymorphic Ventricular Tachycardia Patients.

JACC Clin Electrophysiol

November 2024

Department of Molecular Pharmacology & Experimental Therapeutics (Windland Smith Rice Sudden Death Genomics Laboratory), Mayo Clinic, Rochester, Minnesota, USA; Department of Pediatric and Adolescent Medicine (Division of Pediatric Cardiology), Mayo Clinic, Rochester, Minnesota, USA; Department of Cardiovascular Medicine (Division of Heart Rhythm Services, Windland Smith Rice Genetic Heart Rhythm Clinic), Mayo Clinic, Rochester, Minnesota, USA. Electronic address:

Background: Catecholaminergic polymorphic ventricular tachycardia (CPVT) is a rare, potentially life-threatening genetic heart disease. Nonselective beta-blockers (BBs) are highly effective in reducing CPVT-triggered arrhythmic events. However, some patients suffer from unacceptable BB side effects and might require strategies without a BB.

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Purpose: is one of the most frequently mutated genes in intellectual disability cohorts. Thus, far few adult-aged patients with -related disorder have been described, which limits our understanding of the disease's natural history and our ability to counsel patients and their families.

Methods: Data on patients aged 18+ years with -related disorder were collected through an online questionnaire completed by clinicians and parents.

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Background And Objectives: Foetal and neonatal alloimmune thrombocytopenia (FNAIT) results from maternal platelet-directed antibodies and can result in severe intracranial haemorrhage (ICH) in foetuses and newborns. Screening for human platelet antigen-1a (HPA-1a)-directed antibodies during pregnancy could allow timely intervention with antenatal treatment and prevent ICH. We assessed the cost effectiveness of HPA-1a typing and anti-HPA-1a-screening as part of the prenatal screening programme.

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Complexity science is a discipline which explores how complex systems behave and how we interact with them. Though it is widely implemented outside medicine, particularly in the sciences involving human behavior, but also in the natural sciences such as physics and biology, there are only a few applications within medical research. We propose that complexity science can provide new and helpful perspectives on complex pediatric medical problems.

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Background: Early-onset Group B Streptococcus (EOGBS) infection leads to substantial morbidity and mortality in newborn infants. Intrapartum antibiotic prophylaxis (IAP) prevents EOGBS infection, but IAP strategies vary. The approach to the provision of IAP can be risk-based, universal or a combination of the two strategies.

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Background And Objectives: Routine coagulation screens at birth are still standard in some European neonatal intensive care units (NICUs), although interpretation of these results is complex in preterm infants. It is unclear to what extent local coagulation assay results agree with published reference ranges when using different analysers and reagents. We aimed to assess coagulation assay results on day 1 of life in very preterm infants admitted to the NICU.

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Objectives: To validate the prognostic accuracy of a previously published tool (HOPE calculator) using longitudinal analysis of amplitude-integrated electroencephalography (aEEG) background activity and sleep-wake cycling to predict favorable or adverse 2-year neurodevelopmental outcome in infants with hypoxic-ischemic encephalopathy (HIE) undergoing therapeutic hypothermia (TH), and to evaluate the predictive value for outcome at 5-8 years of age.

Study Design: Single-center retrospective cohort study in 117 infants who underwent TH for HIE between 2008 and 2022. We scored 2-channel aEEG BGPs, sleep-wake cycling, and seizure activity at 6-hour intervals for 84 hours.

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Background: Advances in haemolytic disease of the fetus and newborn have led to numerous treatment options. We report practice variations in the management and outcomes of haemolytic disease of the fetus and newborn in at-risk pregnancies.

Methods: In this international, retrospective, observational cohort study, data from cases with moderate or severe haemolytic disease of the fetus and newborn were retrieved from 31 centres in 22 countries.

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Newborn screening for SCID and severe T lymphocytopenia in Europe.

J Allergy Clin Immunol

November 2024

Laboratory for Paediatric Immunology, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands. Electronic address:

Article Synopsis
  • Newborn screening (NBS) programs in Europe started in the 1960s, expanding recently to include testing for severe combined immunodeficiency (SCID) through T-cell receptor excision circles (TRECs).
  • A survey sent to 46 European countries collected comprehensive data, with 38 countries responding, revealing that 17 have already implemented TREC-based NBS.
  • The findings indicate a need to reassess the definition of the target disease to include conditions beyond SCID and highlight the importance of international collaboration for improving universal TREC-based screening.
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