Characterizing the Complications Associated with Therapeutic Blood Transfusions for Hemoglobinopathies

and their health care providers are unaware of their diagnosis until overt clinical signs begin to manifest. The initiation of transfusion may be delayed until complications appear. If the diagnosis is never made, patients continue living without the benefits that transfusions could provide. Iron chelation therapy is indicated for all transfused patients when iron measures indicate concentrations that exceed threshold levels. Red blood cell alloimmunization occurs when the immune system is exposed to foreign antigens on red blood cells that are different from its own. Frequent blood transfusions may also expose individuals to infectious agents that may be transmitted through blood. By improving the identification of people who have these conditions and may benefit from appropriately administered transfusions, the understanding of treatment for them, and the standardization of information that is available about them, therapeutic transfusions may be administered in ways that maximize their benefits and minimize their adverse effects. This NOFO aims to improve access to, coordination of, and continuity of health care for individuals with thalassemia or sickle cell disease which will lead to a decreased occurrence of transfusion-related complications, improved quality and increased length of life. These health care improvements will result from a multi-faceted approach that includes (1) identification of patients, families, communities, and providers who will benefit from increased knowledge about therapeutic transfusions and their potential complications, (2) development and dissemination of materials that will increase understanding of the diagnosis and management of these blood disorders, and (3) collection of biological specimens from individuals with thalassemia or sickle cell disease. This NOFO builds upon findings from the work completed as part of CDC-RFA-DD14-1406. In particular, (1) the difficulty in identifying any thalassemia patient in the U.S. and their health care provider prior to medical complications manifesting, other than those born in states where the condition is a part of the mandatory newborn screening panel, (2) the lack of standardized practices across, and sometimes within, clinical care settings and blood banks for transfusions of patients with thalassemia or sickle cell disease, (3) the need for dissemination of evidence- or consensus-based guidelines about best practices for transfusions, and (4) the room for improvement in increasing blood donation from communities most affected by thalassemia or sickle cell disease.