Blood Disorders and Blood Safety

Blood diseases affect millions of people each year. These inherited and acquired diseases, including the anemias, venous thromboembolism, hemophilia, and other bleeding disorders, can affect red and white blood cells, platelets, bone marrow, vascular endothelium, or plasma proteins. These conditions can cause blood cells to function abnormally, which may lead to disease or affect overall health. Blood safety involves keeping the Nation’s blood supply free of contaminants or infectious agents.

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Research Making a Difference
Why It's Important
Key Accomplishments
Opportunities & Challenges
Advancing the Research
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Research Making a Difference

Rapid result test may transform sickle cell disease screening for millions."

Soon after birth, babies in the United States are tested for sickle cell disease. In sub-Saharan Africa, however, where the majority of sickle cell disease cases can be found, newborn screening programs are rare, and as a result, there is a missed opportunity for early initiation of lifesaving treatment.

WHY IT'S IMPORTANT

NHLBI has several major research goals in blood diseases. One is to ensure the adequacy and safety of the nation’s blood supply. A second is to support efforts to treat and cure blood disorders, such as supporting scientific advances in stem cell biology and new gene and cell-based therapies to repair and regenerate human tissues and organs. By funding and supporting research on the causes, prevention, and treatment of blood disorders, we help researchers make new discoveries, leading to better medical care for the millions of people affected by blood disorders around the world.

KEY ACCOMPLISHMENTS

The NHLBI-led Cure Sickle Cell Initiative is a patient-focused research effort to develop genetic therapies for use in clinical research.
NHLBI-sponsored research has helped and continues to help Americans with sickle cell disease live longer and stay well in adulthood.
NHLBI-supported scientists developed lifesaving treatments for patients with hemophilia and are now working on gene therapy approaches to cure the disease.
NHLBI-funded research helped develop therapies that increased the survival rates for patients who have aplastic anemia.

OPPORTUNITIES & CHALLENGES

NHLBI’s Strategic Vision guides the Institute’s research activities. Many of the objectives, compelling questions, and critical challenges identified in the plan focus on blood biology, blood disorders, and blood safety. Training the next generation of blood scientists is also a high priority for the NHLBI.

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- Blood Disorders and Blood Safety

Researchers are working across scientific disciplines to advance blood disorders research. The NHLBI assists in this effort by enabling early translational research to accelerate the development of new clinical interventions. Currently, we are supporting research resource programs that provide regulatory, pharmacology, toxicology, and manufacturing services to NHLBI investigators who conduct blood science research. The hope is for patients with blood disorders to live long and full lives and for the research to improve blood safety measures both in the United States and around the world.

Advancing the Research

NHLBI is advancing blood disorders and blood safety research in many ways. Learn more about some of our key efforts related to sickle cell disease, anemia, blood transfusions, and more.

We Perform Research

The NHLBI Division of Intramural Research and its Hematology Branch, Sickle Cell Branch, and Sickle Cell Program include investigators who are actively engaged in research on blood disorders and blood safety.

We Fund Research

The research we fund today will help improve our future health. Our Division of Blood Diseases and Resources is a leader in research on the causes, prevention, and treatment of blood diseases (other than cancers of blood cells). The NHLBI’s Division of Cardiovascular Sciences also supports some blood science–related research.

The Promise of Precision Medicine

Through NHLBI’s Trans-Omics for Precision Medicine (TOPMed) program, researchers will use data from studies focused on heart, lung, blood, and sleep disorders to better predict, prevent, diagnose, and treat blood disorders based on a patient’s unique genes, environment, and molecular signatures. Learn more about NHLBI precision medicine activities.

Funding Global Health Research Efforts

More than 75 percent of infants with sickle cell disease (SCD) are born in sub-Saharan Africa. By funding the SCD in Sub-Saharan Africa (SSA) Network, which includes a Clinical Coordinating Center, a Sickle Africa Data Coordinating Center, and established and new consortium sites, we are building the regional capabilities to research sickle cell disease and monitor patients in Africa. These efforts may ultimately improve treatment and care for Americans who have sickle cell disease.

Improving Blood Safety and Availability

The NHLBI started the Recipient Epidemiology and Donor Evaluation Study (REDS) program in 1989 to protect the nation’s blood supply from threats, improve the benefits of transfusions, and reduce the risks of transfusions. REDS is the largest multicenter research program of its kind in the United States, and after 30 years, it is entering a new, important phase. It now aims to evaluate and improve the safety and effectiveness of transfusion therapies with attention not only to adults but also to understudied populations, including newborns and children.

Addressing Issues in Blood and Marrow Transplantation

The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) was established to conduct large, multi-institutional clinical trials to understand the best possible treatment approaches in blood and marrow transplantation. In the United States, nearly 21,000 patients receive blood or marrow transplants annually, mainly for rare blood disorders.

Studying Underlying Factors in Severe Trauma Cases

The Trans-Agency Research Consortium for Trauma-Induced Coagulopathy (TACTIC) research program is no longer active but completed important work. It was established in collaboration with the U.S. Department of Defense to study the disruptions in the normal process of blood clotting that occur in individuals who experience severe physical trauma. Trauma, particularly severe trauma, can disturb the body’s mechanisms for stopping hemorrhage or lead to the overproduction of blood clots, both of which can cause major or even fatal complications.

Supporting scientists performing NHLBI-funded gene therapy research

Research on genetic therapies is part of our broader commitment to advancing scientific discovery aimed at developing safe and effective treatments for heart, lung, and blood disorders and diseases caused by faulty genes. Our National Gene Vector Biorepository and Gene Therapy Resource Program help investigators transform early-stage research into genetic therapies. These programs equip scientists with the tools, resources, safety testing services, and animal models they need to advance genetic therapy research from the laboratory into clinical trials.

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