Beta Thallasemia

Beta Thalassemia is a genetic disorder characterized by abnormal hemoglobin production or a shortage of healthy red blood cells. It occurs in 280 million people with at least 439,000 severe cases. This disease is most prevalent among individuals of Mediterranean, South Asia and Middle Eastern descent. Severe Beta thalassemia affects about 1,300 people in the United States.

Stem cell transplantation may offer the possibility of cure in people who have an HLA-matched donor, and more recently this appears to be true also for patients who only have mismatched family donors available.

Currently, many patients require continuous red blood cell replacement through a program of red blood cell transfusions. Without these transfusions, these patients develop severe deformities, pain and other serious symptoms which often lead to patients dying while in their early twenties. For patients that require blood transfusions, it is a 10-hour procedure every three to four weeks in the hospital. Busulfan combinations have been documented to eradicate thalassemia and facilitate sustained allogeneic engraftment, also in patients who have poorly matched donors. Additional clinical trials could justify regulatory approval for this very large disease indication.

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