Study Targets Stroke Prevention in Children With Sickle Cell Anemia

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Wed Oct 28, 2009 11:08am EDT

St. Jude Children's Research Hospital scientists awarded federal grant to lead
comparison of treatment options

MEMPHIS, Tenn., Oct. 28 /PRNewswire-USNewswire/ -- St. Jude Children's
Research Hospital investigators were recently awarded a $23 million federal
grant to launch a national study of the drug hydroxyurea to prevent first
strokes in children and adolescents with sickle cell anemia (SCA).

The effort will be the fifth at St. Jude involving hydroxyurea to treat
children with SCA. The focus will be on SCA patients who have not suffered
strokes but have been identified as high risk for the complication by
transcranial Doppler ultrasound (TCD) screening. The five-year study is
expected to include a total of 26 medical centers and about 140 patients.

"Our goal with this study is to prevent brain complications in children with
sickle cell disease," explained Russell Ware, M.D., Ph.D., principal
investigator for the study, which is funded by the National Heart, Lung, and
Blood Institute. Ware chairs the St. Jude Department of Hematology and holds
the Lemuel Diggs Endowed Chair in Sickle Cell Disease.

SCA is an inherited blood disorder. In the U.S., it is diagnosed most
frequently in African American individuals. The sickled red blood cells they
produce can disrupt circulation and cause pain, strokes and other debilitating
and sometimes deadly complications. 

The new study is called TWiTCH, short for TCD With Transfusions Changing to
Hydroxyurea. Patients will be randomly assigned to either continue a standard
therapy of monthly blood transfusions and chelation to remove the resulting
iron buildup or to receive a daily dose of hydroxyurea and monthly phlebotomy
to remove excess iron. The goal is to compare the two treatments, including
their ability to prevent a first stroke. 

St. Jude Children's Research Hospital
St. Jude Children's Research Hospital is internationally recognized for its
pioneering work in finding cures and saving children with cancer and other
catastrophic diseases. Founded by late entertainer Danny Thomas and based in
Memphis, Tenn., St. Jude freely shares its discoveries with scientific and
medical communities around the world. No family ever pays for treatments not
covered by insurance, and families without insurance are never asked to pay.
St. Jude is financially supported by ALSAC, its fundraising organization. For
more information, please visit www.stjude.org.



SOURCE  St. Jude Children's Research Hospital

Carrie Strehlau, +1-901-595-2295, carrie.strehlau@stjude.org, or Summer
Freeman, +1-901-595-3061, summer.freeman@stjude.org, both of St. Jude
Children's Research Hospital
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