Willem-Alexander Kinderziekenhuis

Paediatric haematopoietic stem cell transplantation research priorities: Results from an international multi‐stakeholder priority setting partnership

Summary Co‐designed research in paediatric HSCT is limited. We sought to determine research priorities which represent the shared priorities of patients, parents, carers and healthcare professionals (HCP) within Australia, New Zealand, the Netherlands and the United Kingdom. An i...

Evaluation of nationwide variation in post-haemorrhagic ventricular dilatation management following national guideline implementation in the Netherlands

Abstract Purpose Post-haemorrhagic ventricular dilatation (PHVD) is a severe complication of germinal matrix–intraventricular haemorrhage in preterm infants and is associated with significant morbidity and adverse neurodevelopmental outcomes. In 2023, national recommendations for...

Adherence to treat-to-target recommendations in juvenile idiopathic arthritis in clinical practice

Abstract Objective To assess adherence to Treat-to-Target (T2T) recommendations for juvenile idiopathic arthritis (JIA) in clinical practice. Methods A nested cohort study in the multicenter international prospective UCAN JIA cohort was performed. Patient characteristics, clinica...

Selective immunoglobulin A deficiency and coeliac disease: Immune response dynamics

Abstract Objective To analyse the serological immune response in paediatric patients with selective immunoglobulin A deficiency (SIgAD) and coeliac disease (CeD), focusing on immunoglobulin G antibody dynamics after starting the gluten‐free diet (GFD). Methods Data from SIgAD pat...

Integrating optimal patient-centered care: patient- and family-reported experiences after complex treatment for rare diseases

Abstract Background Although life expectancy after complex treatments for rare diseases during childhood has increased, long-term medical, psychosocial and societal impacts remain for children and their families. Evaluating patient and family experiences is essential for the deve...

Defining global priorities in hematopoietic transplantation and cellular therapy: a statement paper from EBMT Global ComMittee

Abstract Hematopoietic cell transplantation, cellular and gene therapies, and (CAR)-T cells have revolutionized outcomes in hematologic disorders. However, major disparities persist in their availability, accessibility, and affordability, particularly in low- and middle-income co...

The Effect of Antenatal Paracetamol on Breathing Effort of Premature Infants at Birth: A Feasibility Trial

Background: Prostaglandin E2 (PGE2) suppresses perinatal breathing. Reducing antenatal PGE2 concentrations with paracetamol could stimulate spontaneous breathing in premature infants at birth. We aimed to assess feasibility and potential effects of antenatal paracetamol. Methods:...