Therapeutic blood transfusions are essential for the management of individuals with thalassemia and sickle cell disease. However, complications arising from transfusions are a major cause of morbidity. The overall aim of this project is to enhance the appropriate use and safety of transfusions in hemoglobinopathies through education, surveillance, and promotion of consensus- or evidence-based guidelines.
Information on the prevalence of thalassemia in the United States remains inadequate, which constrains efforts to improve access to expert management and continuity of care. Certain individuals who are not getting transfused may develop complications later in life that will require transfusions. This project will develop and validate a method for identifying previously undiagnosed individuals with thalassemia. This will achieve the outcome of improved understanding of the prevalence and natural history of thalassemia, which will assist in the rational use of transfusion therapy and improved quality of life of individuals with thalassemia.
Hemoglobinopathies disproportionately affect individuals from minority populations. Donor-recipient mismatch of red cell antigens because of different ethnicities contributes to the high rate of alloimmunization observed in thalassemia and sickle cell disease. Alloimmunization is a serious complication that can jeopardize further transfusion therapy, but its prevalence may be lowered if donor-recipient red cell antigens are better matched. This project will develop processes to disseminate information pertaining to thalassemia and sickle cell disease that raises awareness of the need for blood donations from population groups most impacted by hemoglobinopathies. Increased diversity of the blood supply will be achieved through the outcome of higher proportion of donors from the minority communities.
The extensive genotypic diversity of hemoglobinopathies necessitates complex laboratory testing protocols. The appropriate management of thalassemia and sickle cell disease and the rational use of transfusions will be facilitated by improved understanding of the laboratory results. In order to achieve this outcome, a toolkit of educational materials will be developed to interpret laboratory results that indicate a potential or confirmed diagnosis of thalassemia or sickle cell disease. The target populations are individuals affected by thalassemia and sickle cell disease, their families and caretakers, and health care providers in primary and specialty-care setting.
The adoption of standardized guidelines to manage transfusion therapy of individuals with thalassemia and sickle cell disease will reduce the prevalence of complications. This outcome will be achieved through an increased understanding of the guidelines for management of transfusions in hemoglobinopathies. This project will develop a toolkit of educational materials on evidence- or consensus-based guidelines for the management of transfusions. The target populations are blood bank administrators and transfusion medicine providers, individuals with thalassemia and sickle cell disease, their families and caretakers, and their health care providers.
Since the exposure to blood transfusions in individuals with hemoglobinopathies is recurring and indefinite, the risk of transfusion-associated infections is significant. This project will achieve the outcome of understanding how infectious agents play a role in transfusion complications in thalassemia and sickle cell disease through the collection of biological specimens over the project period for testing.
The multi-faceted activities and outcomes in this project will address critical areas in transfusion management that will lead to a decreased occurrence of complications and an improvement in the quality of life for individuals with thalassemia and sickle cell disease.