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Treatment May Reverse Sickle Cell Disease in Adults

Regimen includes stem cell transplantation, irradiation, and immunosuppression

WEDNESDAY, Dec. 9 (HealthDay News) -- A treatment regimen that includes stem cell transplantation can lead to stable grafts and reversal of disease in adults with sickle cell disease, according to a study in the Dec. 10 issue of the New England Journal of Medicine.

Matthew M. Hsieh, M.D., from the National Institutes of Health in Bethesda, Md., and colleagues performed non-myeloablative transplantation with CD34+ peripheral-blood stem cells in 10 adults with sickle cell disease. All patients received total body irradiation and alemtuzumab before transplantation, and sirolimus after transplantation.

After a median of 30 months, the researchers found that nine patients had long-term, stable grafts that were able to reverse their sickle cell disease. The mean donor-recipient chimerism was 53.3 percent for T cells and 83.3 percent for myeloid cells. Mean hemoglobin values improved from 9.0 before transplantation to 12.6 after transplantation. No patient developed graft-versus-host disease.

"If the results seen in these first 10 patients withstand the test of time, the regimen that Hsieh et al used could be ideal for patients with severe sickle cell disease," Miguel R. Abboud, M.D., from the American University of Beirut Medical Center in Lebanon writes in an accompanying editorial. However, he adds that "its applicability is still limited by the small number of available HLA-matched siblings."

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