Thalassemia Program at The Children's Hospital of Philadelphia Awarded Federal Grant; Will Help Protect Nation's Blood Supply


PHILADELPHIA, March 5 /PRNewswire/ -- The Children's Hospital of Philadelphia has received a $2.7 million, five-year grant from the Centers for Disease Control and Prevention (CDC) for wide-ranging research regarding people suffering from the inherited blood disorder thalassemia.

Because patients with thalassemia require frequent blood transfusions, an important thrust of the grant will be to monitor the nation's blood supply for new infectious agents.

The principal investigator of the grant is Alan R. Cohen, M.D., director of Children's Hospital's Thalassemia Program. A pediatric hematologist, Dr. Cohen is Physician-in-Chief at The Children's Hospital of Philadelphia, and a world-renowned expert in thalassemia. He also chairs the Medical Advisory Board of the Cooley's Anemia Foundation, a national organization that advocates for patients with thalassemia, of which Cooley's anemia is the most severe form.

Entitled "Prevention of Complications of Thalassemia," the CDC grant recognizes the fact that the standard treatment for thalassemia carries its own set of problems. Thalassemia is a group of related disorders in which a genetic defect in hemoglobin, the iron-carrying component of blood, causes life-shortening tissue and organ damage. Although thalassemia is treatable with frequent blood transfusions, that treatment carries risks of transfusion- related infections and overburdening organs with excessive iron.

"Although the nation's blood supply is safer than it has ever been, it is essential to systematically detect new contaminants at an early stage," said Dr. Cohen. "Taking as our model the established program that monitors blood plasma products needed by patients with hemophilia, we will construct a corresponding system for patients who receive transfusions of red blood cells," he added. "This grant will allow us to enroll thalassemia patients in a surveillance program that uses systematic blood tests to regularly monitor patients for new infections."

Patients with thalassemia receive 12 to 50 units of blood per year, so they are particularly vulnerable to any undetected infectious agents in the blood supply. Dr. Cohen adds that developing a coordinated system for monitoring new infections will benefit all patients who receive a blood transfusion.

Patients receiving blood transfusions were inadvertently infected with hepatitis C before the hepatitis C virus was screened out of the blood supply. New concerns have arisen about potential blood-borne transmission of new variant Creutzfeld-Jakob disease, which is related to mad cow disease. "We cannot predict particular blood-borne infections, but a more rigorous surveillance system will allow us to quickly detect emerging pathogens," said Dr. Cohen.

The grant also supports efforts by the Thalassemia Program to identify new patients with the disease and to provide comprehensive services in managing thalassemia, such as full medical evaluations and access to the multidisciplinary services at Children's Hospital. It also provides for outreach to medically underserved populations having a high prevalence of thalassemia, such as immigrants from Southeast Asia.

"Patterns of thalassemia prevalence in the United States reflect our nation's changing demographics," said Dr. Cohen. "Many recent immigrants come from parts of the world where thalassemia is more prevalent, such as Southeast Asia. Our Thalassemia Program will expand our current efforts to develop community-based and culturally sensitive care to thalassemia patients in Southeast Asian communities."

The Thalassemia Program at Children's Hospital collaborates closely with other medical centers treating the disease. For instance, the Program recently formed a Mid-Atlantic Thalassemia Consortium with four other East Coast hospitals to coordinate research studies. That consortium is part of a larger organization, the Thalassemia Clinical Research Network, founded by the National Institutes of Health to pool the resources of leading centers to implement clinical studies. The Children's Hospital Thalassemia Program also has a longstanding clinical research collaboration with thalassemia programs in Europe.

Founded in 1855 as the nation's first pediatric hospital, The Children's Hospital of Philadelphia is ranked today as the best pediatric hospital in the nation by a comprehensive Child magazine survey. Its pediatric research program is among the largest in the country, ranking second in National Institutes of Health funding. Children's Hospital operates the largest pediatric healthcare system in the U.S. with more than 40 locations in Pennsylvania, New Jersey and Delaware. For more information, visit

CONTACT: John Ascenzi of The Children's Hospital of Philadelphia, +1-215-590-7332 or