May 10, 2022

The purpose of the 1st NCI ImpacT21 symposium is to bring together the academic and hematology-oncology community engaged in scientific and clinical research in leukemia in children and adolescent and young adults with Down syndrome to address current gaps in knowledge, identify barriers in clinical trials enrollment and improve overall outcomes for this at-risk vulnerable population.

Children, and adolescents and young adults (AYA) with Down syndrome (DS) have an increased risk of developing leukemia—specifically a 150-fold increased risk of developing myeloid leukemia (ML-DS) and a 24-fold increased risk of developing acute lymphoblastic leukemia (ALL). Approximately 10-30% of newborns with DS are diagnosed with transient abnormal myelopoiesis (TAM), which resolves spontaneously in the majority, yet 20% develop ML-DS during the first 4 years of life. The basis for this increased risk of leukemia remains unclear. Insights into TAM may help us understand how early fetal hematopoietic development promotes leukemogenesis. Although specific chemotherapy protocols have resulted in excellent outcomes for ML-DS, the success rate of treatment remains 10-20% lower for children with ALL and DS, due to a higher risk of relapse and toxicity of treatment. Children with DS and leukemia often have not been able to have early access to novel therapeutics due to the concern of increased toxicity. More recently, however, inclusion of patients with DS has been considered in the design of clinical trials for new agents. In addition, data on late effects of leukemia therapy in children and AYAs with DS is limited in long term survivors.

Virtual meeting program

The primary goal of this virtual meeting is to inform and engage the broader medical community regarding the need for research and clinical trials for children and AYA with DS leukemia.

The symposium will include leading national and international experts in the field who will discuss disease biology, alongside treatment and clinical trial considerations. The target audience includes clinicians and scientists in academia and private practices, serving pediatric and AYA patient populations. 

Overall Goal

The overall goal of this symposium is to bring together leading scientific and clinical investigators of acute leukemias in children and adolescents with trisomy 21, to survey the current state of the field and foster collaborations to address knowledge gaps and improve outcomes.

Organizing Committee

Co-Chair: Anupam Verma, MD, FAAP. Associate Research Physician, Hematologic Malignancies Section, Pediatric Oncology Branch, CCR, NCI. anupam.verma@nih.gov

Co-Chair: Karen Rabin, M.D., Ph.D. Associate Professor, Pediatric Hematology/Oncology, Director, Leukemia Program, Texas Children's Cancer Center, Baylor College of Medicine. krrabin@texaschildrens.org

Johann (Hans) Hitzler, MD, FRCP(C), FAAP. Professor, Department of Pediatrics, University of Toronto, Division of Hematology/Oncology, Section Leukemia LymphomaThe Hospital for Sick Children. johann.hitzler@sickkids.ca

Philip J. Lupo, PhD, MPH. Professor of Pediatrics, Baylor College of Medicine, Endowed Chair in Molecular Epidemiology, Director, Epidemiology and Population Sciences Program, Texas Children’s Cancer and Hematology Center. philip.lupo@bcm.edu

Nirali N. Shah, MD, MHSc. NIH Lasker Clinical Research ScholarNIH Distinguished ScholarHead, Hematologic Malignancies SectionPediatric Oncology Branch, CCR, NCI. nirali.shah@nih.gov


Registration is currently closed.