Drug may eliminate transfusions in patients with blood disorder

(WASHINGTON, DC, August 12, 2003) The use of hydroxyurea may eliminate the need for future blood transfusions in children with beta-thalassemia major, an inherited blood disorder, according to a study published in the August 15th issue of Blood.

Beta-thalassemia is an inherited blood disorder that occurs when a person is unable to produce adequate levels of hemoglobin, the oxygen-carrying component of red blood cells. This genetic disorder probably arose about 6,000 years ago as a partial defense against malaria.

The most common treatment for severe forms of beta-thalassemia is red blood cell transfusions, which provide the patient with a temporary supply of healthy cells that function normally and supply the body with the needed oxygen. However, patients may experience severe complications from these transfusions, and in the developing world there are limited supplies of suitably tested red blood cells. Such complications lead researchers to look for alternative treatments to boost hemoglobin levels. Researchers from the Service d'Hematologie, Hopital Franz Fanon, in Blida, Algeria, hoped that administration of the drug hydroxyurea to patients with severe forms of beta-thalassemia would result in production of a useful form of hemoglobin called fetal hemoglobin. Hydroxyurea has previously been proven useful in treating sickle cell anemia.

In the study, seven transfusion-dependent beta-thalassemic children in Algeria were treated daily with hydroxyurea at the maximum levels. Of the seven patients, six had experienced transfusion-related complications, such as anaphylactic reactions, severe fever and chills, post-transfusional iron-metabolism problems and lack of venous access. Median follow-up was 19 months. Researchers targeted post-transfusional hemoglobin levels of 7 9 g/dl (grams per deciliter), since Algeria's blood supplies are limited. This range is under the normally recommended post-transfusional hemoglobin levels in co

Contact: Aimee Frank
American Society of Hematology

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