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Children's national co-leads nationwide study of landmark sickle cell treatment
Date:8/18/2008

been routinely utilized to treat treatment-refractory leukemia and lymphoma for almost two decades," said Dr. Kamani, co-principal investigator of the study. "Preliminary results from the few unrelated donor transplants that have been performed for other non-malignant conditions show great promise for those who suffer a severely diminished quality of life due to sickle cell disease. Participants in this large-scale study will help us understand how to better treat, and possibly even cure, sickle cell disease for more people."

Starting in late August, the clinical trial will enroll 45 patients ages 3 to 16 with severe symptoms of sickle cell disease, including stroke, recurring acute chest syndrome, or frequent pain crises. For all marrow and cord blood transplants, patients must undergo intense chemotherapy to prevent their immune systems from rejecting donor cells. This study will use a reduced-intensity chemotherapy to minimize side effects and increase the likelihood that the donor cells will be accepted by the body. After the transplant, participants will be followed for two years to gauge the effectiveness of the transplant at minimizing the ravages of sickle cell disease.


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Contact: Emily Dammeyer
edammeye@cnmc.org
202-476-4500
Children's National Medical Center
Source:Eurekalert

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