Characterizing the Complications Associated with Therapeutic Blood Transfusions for Hemoglobinopathies

Red blood cell (RBC) transfusions are an important part of medical care for many patients with thalassemia or sickle cell disease.

Some patients never need a transfusion, others may have infrequent or intermittent needs, and still others may need chronic transfusions every few weeks.

Determining

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which patients need to receive transfusions, when and how often they need to receive them, and what steps minimize complications from the transfusions may lead to improved health outcomes.

For individuals with thalassemia, specifically, there are no universal screening programs in the U. S. and so many patients 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-DD14-140 6. 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.
Related Programs

Blood Disorder Program: Prevention, Surveillance, and Research

Department of Health and Human Services


Agency: Department of Health and Human Services

Office: Centers for Disease Control - NCBDDD

Estimated Funding: $3,750,000


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Date Posted:
2019-02-26

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Archive Date:
2019-06-02


Ganesh Natarajan is the Founder and Chairman of 5FWorld, a new platform for funding and developing start-ups, social enterprises and the skills eco-system in India. In the past two decades, he has built two of India’s high-growth software services companies – Aptech and Zensar – almost from scratch to global success.






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