Child research

Pediatric Rheumatology:
Collaboration is essential
The Pediatric Rheumatology Department, Wilhelmina Children’s
Hospital is comprised of both basic science and clinical research groups.
These include a joint pediatric rheumatology research group, led by
Bas Vastert, MD, PhD and Jorg van Loosdregt-Vastert, PhD; a translational
immunology lab headed by Femke van Wijk, PhD; and research by several
clinical pediatric rheumatology groups, including Joost Swart, MD, PhD,
Nico Wulffraat, MD, PhD and Sytze de Roock, PhD (Juvenile Idiopathic
Arthritis, JIA) and Annet van Royen, MD, PhD (Juvenile Dermatomyositis).
Together, we have a longstanding track record of studying
chronic inflammatory disorders in children.
bvastert@umcutrecht
Bas Vastert, MD,
PhD is a pediatric
rheuma-tologist with
expertise in translational
immunology. He’s
passionate about
developing novel,
biologically informed
therapeutic strategies
to personalize the
treatment for juvenile
idiopathic arthritis.
This truly translational research collaboration, where basic immunological scientists and clinician
scientists work together, lies at the heart of the acknowledgement of our department as a EULAR
Center of Excellence in pediatric rheumatology – we’re only the 2nd pediatric rheumatology
center worldwide. And thanks to the efforts of Nico, who has led several European Union funded
collaborations including Pharmachild (European Pharmacovigilance register) and SHARE
(developing evidence and consensus based guidelines on rare pediatric rheumatic diseases),
our ability to have a real impact on patient care is excellent.
Juvenile Idiopathic Arthritis (JIA)
We cover a broad spectrum of chronic inflammatory diseases in children, with a particular interest
in JIA, a chronic autoimmune disease characterized by arthritis and clinical presentation of
systemic inflammation. In addition, we’re focused on finding off-label uses for existing drugs or
alternative combinations of drugs in order to improve treatment strategies, and we perform
clinical trials based on the fundamental research we perform in our labs.
In 2014, we reported the first prospective cohort study of a first-line therapy with recombinant
IL-1Ra in children with new-onset JIA; the majority of patients responded quickly and could stop
treatment within one year. Based on research in our department, we postulate that timing of
recombinant IL-1Ra administration could be change disease progression. Since then, we’ve
initiated a the Early Stop of targeted Treatment in Systemic JIA (ESTIS), a ZonMw Rational
Pharmacotherapy-sponsored clinical trial, where we’re testing IL-18 as a therapy response
biomarker in systemic JIA in a multicenter clinical trial in the Netherlands.
Another example of off-label use is nicotinamide, also known as vitamin B3, which is used as a
dietary supplement and to treat acne and skin cancers. Most recently, we’ve planned a study to test
whether nicotinamide can act as a regulatory T-cell-inducing compound in a stop strategy of
biologicals and/or methotrexate (a chemotherapeutic agent and immune system suppressant),
which are used as maintenance treatments in non-systemic JIA.
From an immunology perspective
We’re also understanding epigenetic and epitranscriptomic regulatory mechanisms that modulate
immune homeostasis and might be dysregulated in chronic inflammatory (autoimmune) diseases
like JIA. We’re using JIA as a model to study fundamental immune-regulatory mechanisms, and
by combining our findings and observations from both the lab and the clinic, we can gain
insight into the complex interplay between the molecules and pathways involved in
pediatric inflammatory conditions.
We also focus on juvenile dermatomyositis (JDM) from an immunological point of view, where we’re
identifying distinctive and overlapping molecular mechanisms in order to develop early precision
treatment approaches. Together Annet and Femke, have discovered and validated a novel robust
jloosdre@umcutrecht.nl
Jorg van Loosdregt,
PhD is a fundamental
immunologist who
aims to translate
fundamental discoveries
into novel
therapeutic strategies.
He loves collaborating
with experts in other
fields of research.
28 UMC Utrecht - Child research