| Research line | Description | PI |
|---|---|---|
| Pediatric Viral Infectious Diseases | With the use of human organoid technology, researchers within OrganoVIR labs investigate the effects of picornaviruses, HIV, CMV and SARS-CoV2 on these models, to increase our understanding of the mechanism of disease in humans. | Adithya Sridhar |
| Development of human disease models | This research aims to establish human disease models for studying infectious diseases. Here, we use human stem cells to generated organotypic models of the human intestinal tract, human airway, and the central nervous system. These models are then used in the subsequent research lines for studying viral pathogenesis and antiviral testing. | Adithya Sridhar |
| Understanding Dengue virus neuropathogenesis | Advanced technologies, such as a brain-on-chip model, will be used to study dengue's impact on the nervous system. | Adithya Sridhar |
| Drug repurposing for chronic intestinal viral infections | In this research line, we use human intestinal organoids to test potential antivirals for patients suffering from chronic intestinal infections. Specifically, antiviral effects of clinically approved compounds is tested against patient isolated viruses in an human intestinal model. | Adithya Sridhar |
| PROs and PROMs for rare diseases | Development of relevant and personalized outcome measures for children and adults with rare genetic neurodevelopmental disorders such as Tuberous Sclerosis Complex and Fragile X syndrome. Current focus is on development op core outcome sets, PROMs and goal attainment scaling. | Agnies van Eeghen |
| Trials for complex behavioral manifestations of genetic neurodevelopmental disorders | Establishing the effectiveness of various interventions on neuropsychiatric manifestations of cannabis oil on psychiatric and behavioral manifestations of genetic neurodevelopmental disorders including Down Syndrome, Tuberous Sclerosis Complex, Fragile X Syndrome and Sanfilippo disease. | Agnies van Eeghen |
| Dementia in rare genetic neurodevelopmental disorders | Investigating dementia and blood biomarkers for neurodegeneration, in Fragile X Syndrome, Tuberous Sclerosis Complex, Dravet Syndrome, as well as heterogeneous populations with ID and epilepsy. | Agnies van Eeghen |
| Guidelines for ERN ITHACA | Chairing and supervising the development of European guidelines for rare genetic neurodevelopmental disorders and associated comorbidity, and optimizing methodology. | Agnies van Eeghen |
| Familiaire Hypercholesterolemie | Our research focuses on the treatment and outcomes of children with Familial Hypercholesterolemia (FH). FH is a genetic disorder that leads to high cholesterol levels, increasing the risk of cardiovascular disease from a young age. Our research aims to understand how early intervention can improve long-term health outcomes for individuals with FH. We are investigating various treatment strategies, monitoring their effects on cholesterol levels, and evaluating potential impacts on the prevention of heart disease. By closely tracking both short-term and long-term results, we hope to enhance the understanding of atherosclerosis in FH and develop optimized treatment protocols. Ultimately, our goal is to improve the quality of life and lifespan for children diagnosed with this condition. Furthermore we investigate the role of high Lp(a) in childhood. | Albert Wiegman |
| Strengthening Transgender Care for Adolescents | Adolescent transgender care is a relatively new field that is rapidly developing. It has its roots in the Netherlands and I have been at the forefront of what has become known as the ‘Dutch model’. It includes early medical intervention and is at present the internationally dominant care model and was shown to have positive effects in my first longer-term evaluation studies and in recent other outcome publications. Puberty blocking, with no permanent effects, relieves the acute psychological distress of transgender youth when they experience the physical changes of puberty. It provides time and rest before making decisions regarding masculinizing or feminizing hormones and gender affirming surgeries with life-long consequences. The increased visibility, acceptance and availability of medical gender affirming care is a positive development in a society that embraces diversity and tolerance. Meanwhile, transgender care for adolescents is surrounded by challenges and uncertainties, since medically intervening in a developing adolescent body remains an essential though controversial component of transgender care. Threats to providing affirming medical care exist in various countries. My current research addresses several questions. A topical concern involves the limited evidence base for the care model, especially for the more recent referred adolescents. A key concern is whether these ‘older’ birth assigned girls may present with a ‘new’ form of (rapid or sudden) onset adolescent GI/GD, who may regret their treatment and detransition later. There is a need for more insight how the diversity of developmental pathways and associated psychological difficulties is related to treatment outcomes. Meanwhile, youth, adolescents and their parents report an explicit wish to be more engaged in treatment-associated decisions. This asks for investigation of what good decision-making in gender affirming treatment should comprise. | Annelou de Vries |
| Medication for Inborn Errors of Metabolism | This research line is aimed at optimizing availability and quality of treatments for IEM, in cooperation with Medicine for Society. | Annet Bosch |
| PKU | This research line is aimed at Optimization of treatment and follow-up of patienten with Phenylketonuria (PKU). | Annet Bosch |
| Newborn Screening | This research line is aimed at: - Evaluation and optimization of current Dutch NBS programme - Studies into implementation of new diseases into the Dutch NBS programme | Annet Bosch |
| Riboflavin Transporter Deficiency | This research line is aimed at improving diagnostics, treatment and follow up of patients with Riboflavin Transporter Deficiency | Annet Bosch |
| Classical Galactosemia | This researchline is aimed: - Firstly at elucidating the pathophysiology of the long term complications in Classical Galactosemia, - Secondly at optimizing care, support and follow-up of patients with Classical Galactosemia | Annet Bosch |
| Neonatal pulmonology | This research line aims to unravel the (patho)physiological principles of neonatal lung function and disease. In addition, it aims to improve treatment of neonatal lung disease, thereby reducing the risk long-term respiratory morbidity, especially bronchopulmonary dysplasia (BPD). To achieve these goals we investigate new lung function monitoring tools, test new modes of respiratory support and pharmacological interventions. For this we perform(ed) pre-clinical experimental studies, observational cohort studies, randomized controlled trials, working together with both national and international research groups. | Anton van Kaam |
| Neonatal control of breathing | This research line aims to unravel the (patho)physiological principles of control of breathing in preterm infants. In addition, it aims to improve treatment of abnormal control of breathing. To achieve these goals we investigate new lung function monitoring tools, test new modes of respiratory support and pharmacological interventions. We perform(ed) observational cohort studies and randomized controlled trials, working together with both national and international research groups. | Anton van Kaam |
| Long-term outcome after preterm birth | This research line aims to identify risk factors for abnormal long-term outcome following preterm birth and use these risk factors to design prediction models. In addition, it aims to design and test new interventions to improve outcome of both children and their parents. | Anton van Kaam |
| Nutrition, gut health, growth, and metabolism in newborns | This research line aims to add the knowledge bank and improve neonatal care with regard to parenteral and enteral nutrition (clinically oriented), gastroenterology (incl microbiology and surgery), and basic nutritional science (e.g. organoids, human milk / donor milk quality). | Anton van Kaam |
| LEARNS | This research line aims to prevent a relapse in children with first episode of idiopathic nephrotic syndrome (INS) and to unravel to pathogenesis of INS. The LEARNS consortium consists of 5 work packages: - 1. RCT with combination treatment of prednisolone and levamisole or placebo; - 2. Health-related Quality-of Life (HRQoL); - 3. Pharmacokinetics and Pharmacodynamics of prednisolone and levamisole; - 4. IMMUNOMICS and Biobanking; - 5. In vitro podocytes research. | Antonia Bouts |
| Recurrence of nephrotic syndrome after pediatric kidney transplantation | Children with idiopathic steroid-resistant nephrotic syndrome (SRNS) have a high risk of developing end stage renal disease and recurrence after kidney transplantation. The treatment of recurrence is challenging. Within the European CERTAIN Working Group we investigates the recurrence rate, the (prophylactic) best treatment and outcome of children with SRNS/FSGS after kidney transplantation. | Antonia Bouts |
| TTV-TX | Torque Teno Virus (TTV) belongs to the Anelloviruses. The measurement of TTV in blood might be of additional value in the personalized dosing of immunosuppression in children after kidney transplantation, besides the blood concentration levels of immunosuppressive medication. A more personalized immunosuppressive treatment might prevent over- and under-immunosuppression with risk of rejection, infection and PTLD. Furthermore the Annelloma of the donor and recipient is investigated to study the transmission of the virus from donor to recipient. | Antonia Bouts |
| Family Mental health | This research line aims to improve family-focused approaches to mental health care by implementing effective strategies and developing and testing improved strategies. Professionals in child and adolescent mental health care services and adult mental health care services receive guidelines they can put into practice in order to give families optimal care. Questionnaires, focus groups and interviews are used to gather insights from professionals as well as information on effectivity from parents and their children. Longitudinal databases that include genetic data are used to improve the identification of families that are vulnerable for a less favourable course in order to provide more intense treatments to those in highest need. | Christel Middeldorp |
| Pediatric chronic intestinal failure | This research focuses on children with chronic intestinal failure and has the aim to better understand the complex underlying rare diseases, to improve nutritional management and catheter care, to improve transition from pediatric to adult care, to improve quality of life and to develop personalized medicine for children with rare congenital enteropathies. | Clara van Karnebeek |
| Pediatrische InfectieZiekte Onderzoek, PIZO | This research line primarily revolves around clinical pediatric viral infections. The projects investigate how viruses impact children’s health, aiming to develop better diagnostic tools and treatments. The research line focuses on long term health outcomes of children and adolescents born with HIV or CMV. | Dasja Pajkrt |
| Pediatric Viral Infectious Diseases | With the use of human organoid technology, researchers within OrganoVIR labs investigate the effects of picornaviruses, HIV, CMV and SARS-CoV2 on these models, to increase our understanding of the mechanism of disease in humans. | Dasja Pajkrt |
| Development of human disease models | This research aims to establish human disease models for studying infectious diseases. Here, we use human stem cells to generated organotypic models of the human intestinal tract, human airway, and the central nervous system. These models are then used in the subsequent research lines for studying viral pathogenesis and antiviral testing. | Dasja Pajkrt |
| Understanding Dengue virus neuropathogenesis | Advanced technologies, such as a brain-on-chip model, will be used to study dengue's impact on the nervous system. | Dasja Pajkrt |
| Drug repurposing for chronic intestinal viral infections | In this research, we use human intestinal organoids to test potential antivirals for patients suffering from chronic intestinal infections. Specifically, antiviral effects of clinically approved compounds is tested against patient isolated viruses in an human intestinal model. | Dasja Pajkrt |
| Paediatric IBD with focus on nutritional therapy | I am a pediatrician and epidemiologist. I discovered that Group B Streptococcus (GBS) disease, the most common cause of sepsis and meningitis in infants, is increasing and showed that current prevention guidelines are inadequate. The goal of my research is to develop effective and targeted prevention approaches for sepsis and meningitis in infants. I therefore started the NOGBS prospective nationwide cohort study that investigates GBS colonization and disease. NOGBS has already collected clinical data, blood and bacterial isolates from cases with GBS and E. coli (>500) and controls (>1000). In collaboration with the Wellcome Sanger Institute, I am identifying new diagnostic targets to improve GBS prevention. I am using a unique dataset that combines clinical data, long-term surveillance data and bacterial sequencing of invasive and colonizing bacteria. We are currently performing the largest GBS bacterial genome-wide association study to date. Outcome data, essential for cost-effectiveness research, was lacking for GBS disease. I therefore started a collaboration that established long-term outcomes for nearly 3000 Dutch and Danish infants with GBS disease. Vaccination against GBS during pregnancy is a promising new prevention strategy. NOGBS joined the international PREPARE consortium to establish immune correlates of protection against GBS disease. These correlates will be used in clinical trials. | Douwe Visser |
| Stem cell therapy for kidney disease | NEOGRAFT is an innovative ‘high-risk – high gain’ translational project, which tackles a global problem of chronic kidney diseases by establishing a novel type of cell therapy, using as source the neonatal kidney stem/progenitor cells (nKSPC). The ultimate goal of the project is to: i. reconditio kidney grafts prior to transplantation and extend number of kidneys suitable for transplantation in a growing population of patients on a transplantation waiting list; ii. regenerate kidneys affected by chronic kidney diseases. For doing that, nKSPC need to be characterized in-depth to gain fundamental insights in developmental, immunomodulatory and regenerative potential of the cells. Additionally, in this project we make the first steps towards the establishment of a potent and safe cell source suited for consequent clinical trials. | Elena Levtchenko |
| ArtDECO – Aetiology of renal and urinary tract anomalies defines diagnostic efficacy and clinical outcome | ArtDECO is a multicenter research framework that aims to connect the molecular etiology with the clinical outcome of children with congenital anomalies of the kidney and urinary tract (CAKUT) by: - 1. The establishment of a nationwide data- and biobank of patients with CAKUT - 2. Gene discovery studies for monogenic causes of CAKUT. - 3. Genome-wide association studies to unravel the oligo-polygenic backgrounds of CAKUT - 4. Gene-environment interaction studies using maternal questionnaire data - 5. Functional pipeline for CAKUT containing in vivo and in vitro model systems Homepagina - Artdeco Study | Elena Levtchenko |
| FINEart – Foetal INvestigations into the Etiology of anomalies of renal tissue | FINEart aims to identify the cellular mechanism underlying kidney dysplasia, which is the most important cause of kidney failure in childhood, by the establishment of a spatial transcriptionally-resolved atlas of normal and abnormal kidney development. We investigate unique fetal samples with normal and abnormal kidney development by using cutting edge spatial transcriptional technologies. This research is conducted in Amsterdam UMC in collaboration with RWTH Uniklinik in Aachen. Projectenoverzicht - Nierstichting | Elena Levtchenko |
| Optimal and personalized treatment for patients with Primary Hyperoxaluria (PH) | The research line comprises clinical trials, clinical efficacy studies (ODAP lumasiran), observational clinical studies and basic studies aimed at personalized treatment for patients with Primary Hyperoxaluria: - 1. Clinical trials: participation in PHYOX (Novo/Nordisk/Dicerna) 1,2,3 and 5 and Illuminate studies 1,3 (Alnylam): RNAi therapies, nedosiran and lumasiran for PH1-3 patients - 2. Clinical efficacy: Orphan Drug Access Protocol for lumasiran in PH1 (ODAP lumasiran): controlled and limited access to lumasiran therapy for PH1, collaborative study of all Dutch Academic sites, united insurance companies and Medicijn voor de Maatschappij (Carla Hollak) - 3. Observational registry studies: a. RHINE study: collaborative studies of Oxaleurope & ERKNet, endorsed by NovoNordisk: delay of diagnosis in PH, comparison of outcomes of PH, cystinuria and Dent’s disease b. Oxaleurope outcomes studies PH (cardiac involvement, real world outcome data RNAi in PH) - 4. Stable isotope studies: a. DIOX study: intestinal oxalate absorption in PH patients, healthy controls and Non-PH kidney stone patients by using stable isotopes b. Endogenous oxalate production in PH patients under nesodiran and lumasiran by using iv stable isotopes – Grant of the Kidney Foundation - 5. Lab: a. Role of chitotriosidase in the pathophysiology of tubule-interstitial damage in PH1 b. Pyridoxine dependent pathways in PG | Elena Levtchenko |
| New therapy for cystinosis | Cystinosis is a genetic disorder affecting kidney function due to cystine accumulation. Current treatment with cysteamine slows progression but has many side effects. Our research suggests cystinosis-affected cells struggle to use cysteine for energy production. This project aims to stimulate this metabolic pathway using vitamins (vitamin B5 or vitamin B3) to improve energy metabolism in kidney cells. Using patient-derived cell models and advanced metabolic analysis, we study cystine and cysteine dynamics. Since vitamins are already available, our findings could quickly translate into improved patient treatment. | Fanny Oliveira Arcolino |
| Global Pediatric Oncology | Globally, each year more than 400,000 children are confronted with cancer. While the far majority (>80%) of children lives in so-called low- and middle income countries (LMIC), their chance of cure typically is 20% or less. This is the opposite for children with cancer in high-income countries (HIC): less than 20% of all children reside there with >80% chance of cure. This research program is aimed to help bridging this gap and fits nicely in the WHO Global Initiative on Childhood Cancer. This initiative aims to achieve at least 60% cure for children with cancer worldwide. Important pillars in this program are datamanagement, better care including anticancer treatment and supportive care, capacity building, research and advocacy. | Gertjan Kaspers |
| Childhood acute myeloid leukemia | Children with acute myeloid leukemia (AML) currently have a cure rate of 80% in high-income countries such as The Netherlands. However, that comes with a cost in terms of significant morbidity during treatment and frequent late effects after completion of therapy. In low- and middle-income countries, survival rates are typically well below 30%. In other words, treatment must further improve all over the world. Important | Gertjan Kaspers |
| Neuroscientic outcome measurement | This research line aims to determine the impact of disease and treatment on the brain of children and young adults. The work focuses on the development and application of neuroscientific outcome measurements in typically developing and clinical groups, assessing neurocognitive functioning in children and adults (e.g. the in-house developed ‘Emma Toolbox for Neurocognitive Functioning’), advanced analysis of neurocognitive test performance (e.g. neurocognitive networks), structural and functional brain networks (e.g. DTI resting-state fMRI), and early neurocognitive functioning in babies and infants (e.g. eye-tracking). | Jaap Oosterlaan |
| Clinical outcome prediction for precision medicine | This research line is aimed at better prediction of patient outcome, contributing to the transition towards precision medicine by improving clinical outcome prediction. Together with prof. dr. Mark Hoogendoorn & dr. Frank Bennis, we investigate the added value of machine learning models for clinical outcome prediction. We are also developing a machine learning pipeline optimized for high-dimensional data. | Jaap Oosterlaan |
| Data-driven health care innovation | This research line aims to innovate health care using structured clinical data, by integrating care, evaluation and scientific research using structured care paths. This work is focused on the development of structured multidisciplinary clinical (follow-up) programs that make use of structured electronic clinical registration and integrate with other clinical data sources (e.g. medical devices, patient reported outcome measurements). Consequently, the structured clinical data flows into rich and ever-accumulating databases that are re-used for care evaluation and scientific research aimed at data-driven care innovation. The Emma Children's Hospital Follow Me program is the blueprint for this work. | Jaap Oosterlaan |
| Pediatric acute lung injury and respiratory support | This research line aims to determine the impact and treatment of acute lung injury in critically ill children. The work focuses on pathophysiological mechanisms and epidemiology of severe viral respiratory infections and ARDS, childhood respiratory trajectories, the effectiveness of various respiratory support modalities (e.g. mechanical ventilation and non-invasive ventilation), potential adverse effects of treatment (e.g. ventilator- and hyperoxia-induced lung injury), and opportunities for optimization of treatment (e.g. ventilation mask personalization). | Jeroen Hutten |
| Control of breathing in preterm infants | The research focuses on monitoring respiration in preterm infants using transcutaneous electromyography of the diaphragm (dEMG) and exploring treatments for apnea in preterm infants. This includes both bench studies and clinical trials. Over the past few years, dEMG has been validated as a reliable method for cardiorespiratory monitoring. Further investigations are underway to determine whether dEMG can be utilized to synchronize spontaneous breathing in preterm infants with ventilators. Additionally, the research examines the effects of hypoxia resulting from apnea and assesses the safety and efficacy of doxapram as a potential treatment for apnea in preterm infants. | Jeroen Hutten |
| Technical innovations to timely identify critically ill children | Over the past years this has been my major topic of research. Most recent success is that I secured a large EU grant (3.4 million euro) as the PI on the IMPALA project. | Job Calis |
| Circulatory failure & Severe anaemia: | Over the past 20 years several studies concerning severe anaemia (my PhD and consecutive PhD’s I supervised) and circulation were done. | Job Calis |
| Severe infectious diseases in paediatrics | Improving care of life threatening infections. I supervised 4 PhD students (Sigaloff, Boender, Boerma, Huibers) in the field of HIV care and studied severe bacterial and viral infections in African children, PICU in the Netherlands and the carribean. | Job Calis |
| Setting up critical care units and care in LMIC | Newest area of research which focuses on setting up High and intensive care facilities and services in LMIC. We published our experience setting up the first PICU in Malawi, reviewed the literature on this topic and combine health systems with medical education and | Job Calis |
| Critical illness in resource poor settings | In this research line we aim to improve timely recognition of pediatric critical illness through innovative monitoring. In addition, we aim to discriminate with innovative techniques between different forms of shock | Job Calis |
| Pediatric acute lung injury and respiratory support | This research line aims to determine the impact and treatment of acute lung injury in critically ill children. The work focuses on pathophysiological mechanisms and epidemiology of severe viral respiratory infections and ARDS, childhood respiratory trajectories, the effectiveness of various respiratory support modalities (e.g. mechanical ventilation and non-invasive ventilation), potential adverse effects of treatment (e.g. ventilator- and hyperoxia-induced lung injury), and opportunities for optimization of treatment (e.g. ventilation mask personalization). | Job van Woensel |
| Respiratory insufficiency and mechanical ventilation | This research line aims to investigate both (patho)physiological as well as clinical aspects of (imminent) respiratory insufficiency in pediatric patients. | Job van Woensel |
| Evidence for nursing practice | In this research line we aim to find evidence for clinical nursing practice on the PICU. | Job van Woensel |
| Longterm follow up critical illness | In this research line we aim evaluate the longterm consequences of critical illness and PICU treatments. | Job van Woensel |
| Pediatric chronic intestinal failure | This research focuses on children with chronic intestinal failure and has the aim to better understand the complex underlying rare diseases, to improve nutritional management and catheter care, to improve transition from pediatric to adult care, to improve quality of life and to develop personalized medicine for children with rare congenital enteropathies. | |
| Pediatric inguinal hernia (HERNIIA) | - 1. National RCT for studying optimal treatment of pediatric inguinal hernia repair: open vs laparoscopic surgery. Amsterdam UMC is leading hospital. - 2. Create application for early recovery after surgery. | |
| Intestinal anastomotic healing and leakage | This study assesses crucial factors involved in intestinal anastomotic healing and leakage after colorectal surgery. | |
| Poepen op school | Almost 70% of the children don’t dare to use the toilet at school. This is especially important for vulnerable children treated for anorectal malformation and Hirschsprung disease. | |
| FLUOPATCH | Outcome of pediatric thyroid gland surgery is largely determined by the functioning of the parathyroid glands. | |
| The SCT study | Sacrococcygeal teratoma (SCT) is a congenital germ cell tumor that needs to be resected early after birth. We explored the outcomes of surgery in a previous global study. | |
| KBC-study | This study assesses the gastrointestinal outcomes of children born with an abdominal wall defect (gastroschisis and omphalocele) | |
| Appendicitis in children | Appendicitis in children is a common disease. Recently old dogma’s on this well known entity are challenged | |
| Anorectal malformation | Anorectal malformations are rare and complex congenital anomalies severely affecting HR-QOl in children. Multisdisciplinary team | |
| Rare and complex motility disorders | Anorectal malformations are rare and complex congenital anomalies severely affecting HR-QOl in children. | |
| Micro-CT | Micro-CT imaging is a novel technique recently introduced by our group in the pediatric surgical research. With this technique we aim to gain new insights in micro-anatomy of pediatric surgical conditions | |
| Paediatric IBD with focus on nutritional therapy | The increasing incidence of Crohn’s disease causes considerable morbidity and poses challenges for our health care due to costs of therapy/hospitalisation/surgery. We and others have shown in randomized trials that dietary self-management using the Crohn’s exclusion diet (CDED) can induce remission in children and adults, increase quality of life and reduce the need for immune-suppressing medication. Our research programme assesses biomarkers/multi-omics and novel technology (including heart-rate variability) to improve intestinal healing with dietary therapy. Analysis of HRV-data of patients from disease to remission and during maintenance of remission, will enable integration of biomarkers (CRP/calprotectin) and immune-phenotyping together with multi-omics analyses (epigenetics, microbiome, metabolome). To date, disease management is guided by markers of inflammation in blood and feces together with return of symptoms, which are late signs. Our programme hopes to facilitate the development of non-invasive bio-signatures to help patients improve self-management. In addition to this novel investigator-initiated research programme, we also participate in several industry-sponsored clinical trials and collaborate with other centers in Europe and Canada on IBD-related research projects with other members of the ESPGHAN-Porto IBD group. | Johan van Limbergen |
| Hemophilia and bleeding disorders | Inhibitor development, ADA (anti drug antibodies): immunological and epidemiological determinants. Outcome measurement in hemophilia. | Johan van Limbergen |
| Pediatric trombosis | The research line focuses on two topics: anticoagulation in children and long-term outcomes after thrombosis. In our anticoagulation research, we strive to determine the optimal dosage for children of, among other things, low-molecular-weight heparin, with the fewest side effects. We also investigate how to treat thrombosis as optimally as possible. This way, we aim to ensure that children have the best possible outcome from their thrombosis. In our long-term outcome studies, we examine the complications children develop with different types of thrombosis. A pulmonary embolism causes very different symptoms than a thrombosis in the leg. By accurately identifying the symptoms children experience after their thrombosis, and the risk factors for developing them, we can tailor follow-up to the anticipated problems for each child. | Karin Fijnvandraat |
| Sickle Cell Disease | This research line aims to reduce the burden of pain caused by Vaso Occlusive Episodes in Sickle Cell Disease by early identification and preventive interventions. The second domain of research in SCD is the effect of Sickle Cell Disease on the brain. | Karin Fijnvandraat |
| Transfusion in sickle cell disease and neonates | This research line aims to improve transfusion practices in patients with SCD and neonates by analysing efficacy and safety of red blood cell and thrombocyte transfusions. | Karin Fijnvandraat |
| Patient Reported Outcome Measures | This research line focuses on evaluating the psychometric properties of various PROMs, contributing to the development of novel systems for measuring patient-reported outcomes (PROs), such as PROIMIS, creating CorePRO sets and studying the implementation of PROMs in routine clinical care. | Lotte Haverman |
| Pediatrische psychologie | This line of research focuses both on assessing psychological aspects in children growing up with a chronic illness and their parents, as well as on developing new psychological treatments and investigating the effectiveness of these interventions. | Lotte Haverman |
| Long term child and parental outcomes after perinatal problems | This research line aims to chart long-term child and parental outcomes after perinatal problems (preterm birth, asphyxia) and neonatal and psychosocial interventions thereon. | Lotte Haverman |
| Pediatric Gastroenterology | The main focus is on disorders of gut-brain interaction, such as infant colic, gastro-esophageal reflux (disease), abdominal pain, constipation and fecal in continence. In addition we study rare motility disorders such achalasia, esophageal atresia, pediatric intestinal pseudo-obstruction and Hirschsprung disease. The aim is to unravel anatomic and (patho)physiological abnormalities and mechanisms, investigate the best diagnostics to use and evaluate a scala of new treatments of these functional and organic gastrointestinal disorders. Also the focus is on MAFLD, in which we study symptomatology, diagnostics and treatment. Lastly we aim to unravel the microbiome in premature infants and search for better tools to earlier detect sepsis. | Marc Benninga |
| ATTACK-GD3 trial; investigator initiated intervention study | This research line is aimed at: - Ambroxol as a potential small molecule to treat Gaucher type 3 patients. - Design: n-of-1 case series | Marion Brands |
| Sanfilippo syndrome, MPS III | This research line is aimed at: - Better understanding the phenotype of Sanfilippo syndrome and participation in current trials. - Outstanding research question at the moment are the behavioural and sleep problems in MPS III | Marion Brands |
| Gyrate Atrophy of the Choroid and Retina | This research line is aimed at: - Translational research in Gyrate Atrophy of the Choroid and Retina | Marion Brands |
| Longterm follow-up of patients with Fabry disease | This research line is based on following children and adults with Fabry disease. The effects of treatment (expensive orphan drugs) are evaluated here, and efforts are made to better understand this lysosomal storage disorder. | Marion Brands |
| Neuroscientic outcome measurement | This research line aims to determine the impact of disease and treatment on the brain of children and young adults. The work focuses on the development and application of neuroscientific outcome measurements in typically developing and clinical groups, assessing neurocognitive functioning in children and adults (e.g. the in-house developed ‘Emma Toolbox for Neurocognitive Functioning’), advanced analysis of neurocognitive test performance (e.g. neurocognitive networks), structural and functional brain networks (e.g. DTI resting-state fMRI), and early neurocognitive functioning in babies and infants (e.g. eye-tracking). | Marsh Königs |
| Clinical outcome prediction for precision medicine | This research line is aimed at better prediction of patient outcome, contributing to the transition towards precision medicine by improving clinical outcome prediction. Together with prof. dr. Mark Hoogendoorn & dr. Frank Bennis, we investigate the added value of machine learning models for clinical outcome prediction. We are also developing a machine learning pipeline optimized for high-dimensional data. | Marsh Königs |
| Data-driven health care innovation | This research line aims to innovate health care using structured clinical data, by integrating care, evaluation and scientific research using structured care paths. This work is focused on the development of structured multidisciplinary clinical (follow-up) programs that make use of structured electronic clinical registration and integrate with other clinical data sources (e.g. medical devices, patient reported outcome measurements). Consequently, the structured clinical data flows into rich and ever-accumulating databases that are re-used for care evaluation and scientific research aimed at data-driven care innovation. The Emma Children's Hospital Follow Me program is the blueprint for this work. | Marsh Königs |
| The developing HPA axis | This research line focuses on developmental aspects of HPA axis activity. The overall aim is to tailor glucocorticoid (replacement) therapy to the needs in the very young. | Martijn Finken |
| Paediatric IBD with focus on nutritional therapy | I am a pediatrician and epidemiologist. I discovered that Group B Streptococcus (GBS) disease, the most common cause of sepsis and meningitis in infants, is increasing and showed that current prevention guidelines are inadequate. The goal of my research is to develop effective and targeted prevention approaches for sepsis and meningitis in infants. I therefore started the NOGBS prospective nationwide cohort study that investigates GBS colonization and disease. NOGBS has already collected clinical data, blood and bacterial isolates from cases with GBS and E. coli (>500) and controls (>1000). In collaboration with the Wellcome Sanger Institute, I am identifying new diagnostic targets to improve GBS prevention. I am using a unique dataset that combines clinical data, long-term surveillance data and bacterial sequencing of invasive and colonizing bacteria. We are currently performing the largest GBS bacterial genome-wide association study to date. Outcome data, essential for cost-effectiveness research, was lacking for GBS disease. I therefore started a collaboration that established long-term outcomes for nearly 3000 Dutch and Danish infants with GBS disease. Vaccination against GBS during pregnancy is a promising new prevention strategy. NOGBS joined the international PREPARE consortium to establish immune correlates of protection against GBS disease. These correlates will be used in clinical trials. | Merijn Bijlsma |
| Pediatric chronic intestinal failure | This research focuses on children with chronic intestinal failure and has the aim to better understand the complex underlying rare diseases, to improve nutritional management and catheter care, to improve transition from pediatric to adult care, to improve quality of life and to develop personalized medicine for children with rare congenital enteropathies. | Merit Tabbers |
| Paediatric IBD with focus on nutritional therapy | I am a pediatrician and epidemiologist. I discovered that Group B Streptococcus (GBS) disease, the most common cause of sepsis and meningitis in infants, is increasing and showed that current prevention guidelines are inadequate. The goal of my research is to develop effective and targeted prevention approaches for sepsis and meningitis in infants. I therefore started the NOGBS prospective nationwide cohort study that investigates GBS colonization and disease. NOGBS has already collected clinical data, blood and bacterial isolates from cases with GBS and E. coli (>500) and controls (>1000). In collaboration with the Wellcome Sanger Institute, I am identifying new diagnostic targets to improve GBS prevention. I am using a unique dataset that combines clinical data, long-term surveillance data and bacterial sequencing of invasive and colonizing bacteria. We are currently performing the largest GBS bacterial genome-wide association study to date. Outcome data, essential for cost-effectiveness research, was lacking for GBS disease. I therefore started a collaboration that established long-term outcomes for nearly 3000 Dutch and Danish infants with GBS disease. Vaccination against GBS during pregnancy is a promising new prevention strategy. NOGBS joined the international PREPARE consortium to establish immune correlates of protection against GBS disease. These correlates will be used in clinical trials. | Michaël Boele van Hensbroek |
| Riboflavin Transporter Deficiency | This research line is aimed at improving diagnostics, treatment and follow up of patients with Riboflavin Transporter Deficiency. | Nicole Wolf |
| Pediatric inguinal hernia (HERNIIA) | - 1. National RCT for studying optimal treatment of pediatric inguinal hernia repair: open vs laparoscopic surgery. Amsterdam UMC is the leading hospital. - 2. Create application for early recovery after surgery. | |
| Intestinal anastomotic healing and leakage | This study assesses crucial factors involved in intestinal anastomotic healing and leakage after colorectal surgery. | |
| Poepen op school | Almost 70% of the children don’t dare to use the toilet at school. This is especially important for vulnerable children treated for anorectal malformation and Hirschsprung disease. | |
| FLUOPATCH | Outcome of pediatric thyroid gland surgery is largely determined by the functioning of the parathyroid glands. | |
| The SCT study | Sacrococcygeal teratoma (SCT) is a congenital germ cell tumor that needs to be resected early after birth. We explored the outcomes of surgery in a previous global study. | |
| KBC-study | This study assesses the gastrointestinal outcomes of children born with an abdominal wall defect (gastroschisis and omphalocele). | |
| Appendicitis in children | Appendicitis in children is a common disease. Recently old dogma’s on this well known entity are challenged. | |
| Anorectal malformation | Anorectal malformations are rare and complex congenital anomalies severely affecting HR-QOl in children. Multisdisciplinary team | |
| Rare and complex motility disorders | Anorectal malformations are rare and complex congenital anomalies severely affecting HR-QOl in children. | |
| Micro-CT | Micro-CT imaging is a novel technique recently introduced by our group in the pediatric surgical research. With this technique we aim to gain new insights in micro-anatomy of pediatric surgical conditions | |
| Pediatric acute lung injury and respiratory support | This research line aims to determine the impact and treatment of acute lung injury in critically ill children. The work focuses on pathophysiological mechanisms and epidemiology of severe viral respiratory infections and ARDS, childhood respiratory trajectories, the effectiveness of various respiratory support modalities (e.g. mechanical ventilation and non-invasive ventilation), potential adverse effects of treatment (e.g. ventilator- and hyperoxia-induced lung injury), and opportunities for optimization of treatment (e.g. ventilation mask personalization). | Reinout Bem |
| Metabolism and Metabolic Brain Disorders | This research line aims to identify new metabolic disorders and resolve the pathophysiology of different IMDs based on the detailed knowledge of metabolism with the ultimate goal to develop strategies for the treatment of patients affected by different IMDs. Particular emphasis is focused on Hyperoxaluria together with Dr. Sander Garrelfs, Prof. dr. Jaap Groothoff c.s., Gyrate Atrophy together with Prof.dr. Clara van Karnebeek c.s. among others IMDs. | Ronald Wanders |
| Evaluation of hormonal treatment in transgender adolescents | This research line aims to evaluate treatment trajectories and the efficacy and safety of endocrine treatment for transgender adolescents. A wide range of treatment outcomes has been or is being assessed including continuation/discontinuation of endocrine treatment, growth/adult height, development of desired sex characteristics (such as breast development, voice change), body image, sexual function, safety data such as changes in bone mineral density, and outcomes with regard to fertility/fertility preservation. Research is performed in close collaboration with internist-endocrinologists, psychologists, psychiatrists, gynaecologists, surgeons and ethicists. | Sabina Hannema |
| Improving care for children and adolescents with a difference of sex development (DSD) | This research line focuses on evaluating outcomes in individuals with a difference of sex development (DSD) in order to improve care. Care pathways have been structured according to the Follow Me programme, allowing continuous data-driven health care innovation. Topics of interest include gonadal function and pathology and optimizing hormonal treatment to induce puberty. | Sabina Hannema |
| Pediatric acute lung injury and respiratory support | This research line aims to determine the impact and treatment of acute lung injury in critically ill children. The work focuses on pathophysiological mechanisms and epidemiology of severe viral respiratory infections and ARDS, childhood respiratory trajectories, the effectiveness of various respiratory support modalities (e.g. mechanical ventilation and non-invasive ventilation), potential adverse effects of treatment (e.g. ventilator- and hyperoxia-induced lung injury), and opportunities for optimization of treatment (e.g. ventilation mask personalization). | Suzanne Terheggen |
| Pediatric Immunology | This research line aims to determine the impact of immune dysfunction in genetically determined so-called “inborn errors of immunity (IEIs)” and various conditions of induced (acquired) innate or adaptive immune dysregulation. To these conditions, (auto)inflammatory and B-cell immune-mediated disorders (B-IMDs) are being focused on. In the realm of infection and inflammatory diseases in children we try to find an RNA- and/or protein-based signature to distinguish large categories of disease as a “super-CRP” in a large European consortium to categories to guide diagnostics and treatment in a faster and more certain fashion. | Taco Kuijpers |
| Generation P: Microbiome and pediatric gut health | This research line aims to determine the impact of microbiome and metabolome on early gut health and health outcomes beyond infancy. The work focuses on the correlation between antibiotic exposure, feeding pattern and microbial colonization, related to gut health outcomes, including growth, neurocognitive outcome, asthma en alleries. Furthermore, this research line focused on the development of microbiota-based biomarkers for necrotizing enterocolitis and gut-derived neonatal sepsis. | Tim de Meij |
| Microbiome and metabolome in pediatric IBD Therapeutic drug monitoring in MTX | This research line aims to determine the impact of microbiome and metabolome on disease course in pediatric IBD and to predict therapy response. The work focuses on the correlation between gut microbes and metabolomics in de novo pediatric IBD. Furthermore, this research line focused on therapeutic drug monitoring in MTX and on cancer surveillance in pediatric IBD. | Tim de Meij |
| DREAMS (Dutch REsearch in child and Adolescent Mental health) | DREAMS is a collaboration between 4 child- and adolescent psychiatry centers in the Netherlands: Accare (Groningen), Karakter (Nijmegen), LUMC Curium (Leiden) en Levvel (Amsterdam). Together, DREAMS provides care for approximately 25.000 children per year (0-18 years old). The aim of DREAMS is to do large scale research into risk and protective factors of mental health problems, treatment effects, general wellbeing and long-term outcomes. | Tinca Polderman |
| Bronchopulmonary dysplasia | This research line investigate prediction, diagnosis, pathophysiology, prevention and treatment of bronchopulmonary dysplasia (BPD) in preterm infants. The work focuses on reducing the incidence and the burden of BPD in preterm infants by prevention, but also treatment in established BPD, as well as monitoring of the long term outcome of BPD. | Wes Onland |
| Malnutrition in childhood | This research line is grouped around the broad theme of undernutrition in children with an acute illness in low-resource settings (Low- and Middle-Income Countries). Vulnerable patient groups (cerebral palsy, severe acute malnutrition) are the focus of attention and triage (how to recognise illness in an early stage?) and risk prediction (can we identify a poor outcome in advance?) are main areas of interest. Data collection is done outside of Europe and done with academic partner institutions abroad (Malawi, Yemen, Kenia,Bangladesh). | Wieger Voskuijl |
| Familiaire Hypercholesterolemie | Our research focuses on the treatment and outcomes of children with Familial Hypercholesterolemia (FH). FH is a genetic disorder that leads to high cholesterol levels, increasing the risk of cardiovascular disease from a young age. Our research aims to understand how early intervention can improve long-term health outcomes for individuals with FH. We are investigating various treatment strategies, monitoring their effects on cholesterol levels, and evaluating potential impacts on the prevention of heart disease. By closely tracking both short-term and long-term results, we hope to enhance the understanding of atherosclerosis in FH and develop optimized treatment protocols. Ultimately, our goal is to improve the quality of life and lifespan for children diagnosed with this condition. Furthermore we investigate the role of high Lp(a) in childhood. | Willemijn Corpeleijn |
Last edited: 05-03-2026