1. Nonmyeloablative Stem Cell Transplantation with Alemtuzumab/Low-Dose Irradiation to Cure and Improve the Quality of Life of Adults with Sickle Cell Disease.
- Author
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Saraf, Santosh L., Oh, Annie L., Patel, Pritesh R., Jalundhwala, Yash, Sweiss, Karen, Koshy, Matthew, Campbell-Lee, Sally, Gowhari, Michel, Hassan, Johara, Peace, David, Quigley, John G., Khan, Irum, Molokie, Robert E., Hsu, Lewis L., Mahmud, Nadim, Levinson, Dennis J., Pickard, A. Simon, Garcia, Joe G.N., Gordeuk, Victor R., and Rondelli, Damiano
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MYELOSUPPRESSION , *STEM cell transplantation , *ALEMTUZUMAB , *DRUG dosage , *QUALITY of life , *SICKLE cell anemia in adolescence - Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is rarely performed in adult patients with sickle cell disease (SCD). We utilized the chemotherapy-free, alemtuzumab/total body irradiation 300 cGy regimen with sirolimus as post-transplantation immunosuppression in 13 high-risk SCD adult patients between November 2011 and June 2014. Patients received matched related donor (MRD) granulocyte colony–stimulating factor–mobilized peripheral blood stem cells, including 2 cases that were ABO incompatible. Quality-of-life (QoL) measurements were performed at different time points after HSCT. All 13 patients initially engrafted. A stable mixed donor/recipient chimerism was maintained in 12 patients (92%), whereas 1 patient not compliant with sirolimus experienced secondary graft failure. With a median follow-up of 22 months (range, 12 to 44 months) there was no mortality, no acute or chronic graft-versus-host disease (GVHD), and no grades 3 or 4 extramedullary toxicities. At 1 year after transplantation, patients with stable donor chimerism have normalized hemoglobin concentrations and improved cardiopulmonary and QoL parameters including bodily pain, general health, and vitality. In 4 patients, sirolimus was stopped without rejection or SCD-related complications. These results underscore the successful use of a chemotherapy-free regimen in MRD HSCT for high-risk adult SCD patients and demonstrates a high cure rate, absence of GVHD or mortality, and improvement in QoL including the applicability of this regimen in ABO mismatched cases (NCT number 01499888). [ABSTRACT FROM AUTHOR]
- Published
- 2016
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