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.

The NHLBI seeks to enable early translational research as a means of facilitating and accelerating the development of new clinical interventions. The NHLBI currently supports three research resource programs that provide regulatory, pharmacology and toxicology, and manufacturing services to NHLBI investigators:

• Production Assistance for Cell Therapies (PACT) is designed to develop novel cellular therapies that will aid investigators by providing production assistance in areas ranging from translational development to the scale-up of a product intended for use in human clinical trials.
• Science Moving towArds Research Translation and Therapy (SMARTT) Program accelerates the translation of research from demonstration of efficacy in vivo to submission of an investigational new drug (IND) application to the U.S. Food and Drug Administration.
• The Gene Therapy Resource Program (GTRP) includes preclinical-grade vectors, tests GLP pharmacology and toxicology, produces GMP- and pre–GMP-grade adeno-associated virus vectors and lentivirus vectors, assists regulatory affairs, and funds clinical trials.

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.