denotes an abstract that is clinically relevant.
denotes that this is a ticketed session.
Hematology Disease Topics & Pathways:
Research, Biological therapies, Fundamental Science, clinical trials, adult, Clinical Practice (Health Services and Quality), Bone Marrow Failure Syndromes, Inherited Marrow Failure Syndromes, Translational Research, epidemiology, Genetic Disorders, genomics, Clinical Research, health outcomes research, Diversity, Equity, and Inclusion (DEI) , hematopoiesis, pediatric, Diseases, Gene Therapy, patient-reported outcomes, Therapies, registries, young adult , Biological Processes, emerging technologies, Technology and Procedures, Study Population, Human, imaging, Transplantation, machine learning
Description:
Anemia is the most common hematologic disorder in children worldwide. For many children, anemia is not only a cause of short-term ill-health, it also has a lifelong impact. This session will describe how a range of state-of-the-art technologies are being developed and applied to address challenging questions about the pathogenesis, diagnosis, and management of anemia in children. It will also illustrate how these technologies can be used to deliver practical advances in the management of childhood anemia and show that these benefits can be achieved way beyond the labs where they were first developed including resource-poor settings.
Dr. Deena Iskander will demonstrate the utility of single cell studies in understanding normal human erythropoiesis and its dysregulation in Diamond-Blackfan anemia (DBA). She will describe the clinical heterogeneity of this disease and how single cell RNA sequencing has helped to identify new genotype-phenotype correlations that contribute to disease heterogeneity. Finally, Dr. Iskander will discuss putative mechanisms of erythroid failure revealed by these studies and in turn, potential new therapeutic avenues in DBA.
Dr. Wilbur Lam will discuss new point-of-care and patient-facing technologies to screen for and monitor anemia. He will specifically describe a visual, color-based anemia test that requires a single drop of capillary blood as well as an entirely non-invasive smartphone app that can estimate blood hemoglobin levels with a "fingernail selfie" and how these two complementary technologies can be used synergistically. Dr. Lam will also discuss the accuracy of these technologies and how improvements are being implemented. Lastly, he will discuss the various clinical use cases of these anemia detection technologies for children and adolescents.
Dr. Obiageli Nnodu will describe the strategy and progress in delivering novel therapies in sickle cell disease in Africa. She will focus on advanced therapies that include exchange blood transfusion, hematopoietic stem cell transplantation and Gene Therapy. The strategies she will present highlight the importance of an integrated approach that includes optimizing healthcare, advocacy, research, and training with partnerships at different levels. The goal of this work is to facilitate access of SCD populations in Africa to advanced therapies and participation of healthcare providers, policy makers, educators and researchers from Africa to deliver disease-modifying and curative therapies.
Dr. Deena Iskander will demonstrate the utility of single cell studies in understanding normal human erythropoiesis and its dysregulation in Diamond-Blackfan anemia (DBA). She will describe the clinical heterogeneity of this disease and how single cell RNA sequencing has helped to identify new genotype-phenotype correlations that contribute to disease heterogeneity. Finally, Dr. Iskander will discuss putative mechanisms of erythroid failure revealed by these studies and in turn, potential new therapeutic avenues in DBA.
Dr. Wilbur Lam will discuss new point-of-care and patient-facing technologies to screen for and monitor anemia. He will specifically describe a visual, color-based anemia test that requires a single drop of capillary blood as well as an entirely non-invasive smartphone app that can estimate blood hemoglobin levels with a "fingernail selfie" and how these two complementary technologies can be used synergistically. Dr. Lam will also discuss the accuracy of these technologies and how improvements are being implemented. Lastly, he will discuss the various clinical use cases of these anemia detection technologies for children and adolescents.
Dr. Obiageli Nnodu will describe the strategy and progress in delivering novel therapies in sickle cell disease in Africa. She will focus on advanced therapies that include exchange blood transfusion, hematopoietic stem cell transplantation and Gene Therapy. The strategies she will present highlight the importance of an integrated approach that includes optimizing healthcare, advocacy, research, and training with partnerships at different levels. The goal of this work is to facilitate access of SCD populations in Africa to advanced therapies and participation of healthcare providers, policy makers, educators and researchers from Africa to deliver disease-modifying and curative therapies.