Blood Epidemiology and Clinical Therapeutics

Blood Epidemiology and Clinical Therapeutics is sponsored by National Heart, Lung, and Blood Institute (NHLBI), NIH. Funds clinical research in non-malignant blood diseases and cell therapies, including epidemiologic studies and clinical trials for sickle cell disease.

Division of Blood Diseases and Resources | NHLBI, NIH Divisions, Offices and Centers Division of Blood Diseases and Resources Division of Blood Diseases and Resources The Division of Blood Diseases and Resources (DBDR) is a leader in research on the causes, prevention, and treatment of non-neoplastic blood diseases. DBDR assumes a major responsibility in ensuring the adequacy and safety of the nation’s blood supply.

It also supports scientific advances in stem cell biology and new gene and cell-based therapies developed to repair and regenerate human tissues and organs. Blood diseases affect millions of people each year in the United States.

These congenital and acquired diseases, including the anemias, venous thromboembolism, hemophilia, and other bleeding disorders, impact the normal biology of red and white blood cells, platelets, bone marrow, vascular endothelium, and plasma proteins. They also manifest a wide range of symptoms, generating the need for preventive, therapeutic, and curative approaches to restoring health.

The DBDR is divided into three branches that are structured to facilitate team science in priority areas of blood science. Together, the branches foster the training of the next generation of blood scientists, coordinate blood research activities, and promote communication across the Division, the NHLBI, the NIH, and partner federal agencies.

Related Scientific Focus Areas Stimulating and Supporting Blood Science Research to Improve Clinical Care Division of Blood Diseases and Resources With a particular focus on understanding how blood diseases impact diverse populations, research within the DBDR has affected how physicians diagnose, assess, and treat across the spectrum of blood disorders.

The research supporting these discoveries spans discovery, translational, and clinical science and includes these key aspects: Generating critical knowledge in disorders of blood, hemostasis, and vascular systems, and translating this knowledge into the discovery of diagnostic biomarkers and therapeutic targets.

Research areas include systems biology, glycomics, thrombotic and hemostatic disorders, and vascular dysfunction in the pathogenesis of severe malaria. Establishing multicenter clinical research programs for rare and uncommon blood diseases to enhance participant recruitment, data collection, and analysis.

Programs include the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) and the National Myelodysplastic Syndromes (MDS) Study Informing the prevention and treatment of the complications of sickle cell anemia (SCA).

Research includes the Stroke Prevention Trial in Sickle Cell Anemia (STOP), Optimizing Primary Stroke Prevention in Children With Sickle Cell Anemia (STOP II), Transcranial Doppler With Transfusions Changing to Hydroxyurea (TWiTCH), and the Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG).

Developing and disseminating data science resources in patient-reported outcomes to facilitate phenotyping and cross-study analyses for clinical practice guidelines.

Resources include the Pediatric Quality of Life Inventory (PedsQL) Sickle Cell Disease module, the Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me), the Patient-Reported Outcomes Measurement Information System (PROMIS), the Validation of Pediatric Patient-Reported Outcomes in Chronic Diseases (PEPR), and Consensus Measures for Phenotypes and eXposures (PhenX).

Ensuring the safety of the nation’s blood supply, improving blood transfusion benefits, and reducing associated risks. Research includes the Recipient Epidemiology and Donor Evaluation Study-III (REDS-III), which the NHLBI recently expanded to ascertain the potential threat to the blood supply posed by the emergence of Zika virus.

Facilitating the Translation of Discoveries Into Therapeutics Division of Blood Diseases and Resources The NHLBI seeks to enable early translational research as a means of facilitating and accelerating the development of new clinical interventions.

The NHLBI Catalyze Program provides a comprehensive suite of support and services to help translate basic scientific discoveries into viable therapeutics, devices, and diagnostics ready for human testing.

Leading Precision Medicine Activities for Blood Diseases Division of Blood Diseases and Resources The DBDR and other NHLBI divisions are involved with NHLBI’s Trans-Omics for Precision Medicine (TOPMed) Program , which will support the Institute’s larger precision medicine activities and complement the NIH All of Us Research Program.

TOPMed will collect and couple whole-genome sequencing and other –omics data, such as DNA methylation signatures and RNA expression profiles, with molecular, behavioral, imaging, environmental, and clinical data from studies focused on heart, lung, blood, and sleep disorders.

The TOPMed Program will provide more opportunities for the NHLBI community to pursue research that will further the understanding and improve the care of people with blood disorders. Julie Panepinto, M. D.

, MSPH Blood Epidemiology and Clinical Therapeutics Branch The Blood Epidemiology and Clinical Therapeutics Branch provides oversight, support, and stimulation of epidemiologic, clinical, and implementation research throughout the spectrum of blood science. This spectrum includes benign blood diseases, as well as cell therapies such as hematopoietic stem cell transplants, transfusion products, and novel cell therapies.

This branch also helps develop and disseminate data science resources in patient-reported outcomes. View funding information for the Blood Epidemiology and Clinical Therapeutics Branch . Molecular Cellular and Systems Blood Science Branch The Molecular, Cellular, and Systems Blood Science Branch seeks to advance discovery science focused on the physiology and pathophysiology of blood, bone marrow, and blood vessels.

This Branch promotes a systems biology approach to understanding the critical role of blood, bone marrow, and vascular endothelium in animal and human organs and organisms. It also applies fundamental genetic, proteomic, metabolomic and related data analysis tools and/or approaches to better understand hematologic physiology and pathophysiology.

The Branch also administers and serves as liaison for NHLBI resources in data science and models for understanding bleeding disorders. View funding information for the Molecular Cellular and Systems Blood Science Branch .

Translational Blood Science and Resources Branch The Translational Blood Science and Resources Branch supports the creation and development of blood-focused therapeutics, the facilitation and promotion of discovery science from bench to first-in-human studies, and the coordination of workforce development and opportunities for small-business programs.

This emphasis includes the oversight, support, and stimulation of post-discovery science, preclinical research, early phase clinical studies and trials, and commercialization initiatives (the Small Business Innovation Research and Small Business Technology Transfer programs) in blood sciences. This Branch also administers and serves as liaison for NHLBI resources related to translational research.

View funding information for the Translational Blood Science and Resources Branch . andrei. kindzelski@nih.

gov The Office of the Director oversees all Divisional activities.

This includes planning and coordinating the activities of all Branches responsible for supporting and managing discovery, translational, and clinical research programs across the blood sciences; continuously assessing the Institute's national and international health programs related to all blood science disciplines and resources; fostering and coordinating interdivisional, trans-NHLBI, trans-NIH and interagency collaborative and cooperative research arrangements; developing and maintaining the necessary scientific management capability in the Division to foster and guide effective programs in blood sciences and resources management; planning, coordinating, and directing special activities that transcend program lines, including minority, small business, and education research programs; providing program analysis and administrative support services for the Division; and overseeing Divisional programs to support the workforce in nonmalignant hematology.

Related Scientific Focus Areas Blood Disorders and Blood Safety ‘Low-intensity’ blood stem cell transplants for sickle cell appear safe for lung health NIH study finds lung function remained stable or improved in adults after transplant So-called low-intensity blood stem cell transplants, which use milder conditioning agents than standard stem cell transplants, do not appear to damage the lungs and may help improve lung function in some patients with sickle cell disease (SCD), according to a three...

Division of Blood Diseases and Resources Sickle Cell Disease Advisory Committee - Agenda, January 14, 2025 10:00 a. m. - 3:00 p.

m. ET The Sickle Cell Disease Advisory Committee (SCDAC) makes recommendations on research priorities conducted or supported by NHLBI. The Com...

Division of Blood Diseases and Resources Division of Blood Diseases and Resources The National Heart, Lung and Blood Institute One Rockledge Center, Suite 400 6705 Rockledge Dr. MSC 7950