Meaningful scientific research on rare disorders such as pediatric myelodysplastic syndrome (MDS) and bone marrow failure disorders (BMF) can only advance with the collaboration of multiple centers.
The Pediatric MDS and BMF Registry is a National Institutes of Health (NIH)- funded research program that is dedicated to improving the understanding and treatment of pediatric MDS and BMF. The main goal of the Pediatric MDS and BMF Registry is to collaborate with national and international centers to advance scientific knowledge in the field and work towards the discovery of new targets for therapy.
Our registry, the first of its kind in the United States, is modeled after the European Working Group on MDS (EWOG-MDS), which was founded in 1993 by expert clinicians from Germany, Austria, Denmark, Italy and the Netherlands. We aim to work together with our European colleagues to advance scientific knowledge of MDS and BMF. Charlotte Niemeyer, MD, a world-renowned expert in MDS, serves as a consultant to the project and a member of our Steering Committee.
The Pediatric MDS and BMF Registry is supervised by our Steering Committee, which is responsible for oversight of the tissue bank, data sharing, and authorship. Our Steering Committee works in collaboration with research staff at Children’s to ensure that our goals are met.