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Wednesday, July 9, 2014

Sibling Blood Stem Cell Transplants Reverse Sickle Cell Disease

Half of patients in a trial have safely stopped taking immunosuppressant medication for their sickle cell disease following a modified blood stem cell transplant. The treatment (which consisted of alemtuzumab, total-body irradiation, sirolimus, and infusion of peripheral blood stem cells from human leukocyte antigen–matched siblings) reversed sickle cell disease in 26 of 30 patients and allowed them to achieve stable mixed donor chimerism, a condition in which a person has genetically distinct cell types in the blood. Fifteen patients discontinued immunosuppression medication with continued stable donor chimerism and no graft vs. host disease. The findings are published in JAMA

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