Author Interviews, Hematology, JAMA, Stem Cells, Transplantation / 03.07.2014
Sickle Cell Disease Potentially Cured with Nonmyeloablative HLA-Matched Allogeneic Stem Cell Transplantation
MedicalResearch.com Interview with:
Dr. John Tisdale MD
Molecular and Clinical Hematology Branch
National Institute of Diabetes and Digestive and Kidney Diseases,
National Heart, Lung, and Blood Institute, Bethesda, Maryland
MedicalResearch: What are the main findings of the study?
Dr. Tisdale: Using a nonmyeloablative allogeneic HLA-match peripheral blood stem cell transplantation strategy aimed at tolerance induction, we were able to revert the phenotype in 26 of 30 adult patients with severe sickle cell disease ranging in age from 16 to 65 years. In contrast to standard transplantation strategies which rely on high doses of chemo and/or radiotherapy after which the entire bone marrow and blood system is replaced by that of the donor, our patients had a mixture of their own and that of their donor. This procedure was well tolerated, with no non-relapse mortality, and led to complete replacement of red blood cells by that of the donor in successfully engrafted patients. This replacement resulted in decreases in pain, pain medication usage, hospitalizations, and improvements in organ function.
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