Fludarabine-Based Nonmyeloablative Stem Cell Transplantation for Sickle Cell Disease with and without Renal Failure: Clinical Outcome and Pharmacokinetics

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Fludarabine-Based Nonmyeloablative Stem Cell Transplantation for Sickle Cell Disease with and without Renal Failure: Clinical Outcome and Pharmacokinetics Mitchell E. Horwitz, Ivan Spasojevic, Ashley Morris, Marilyn Telen, James Essell, Cristina Gasparetto, Keith Sullivan, Gwynn Long, John Chute, Nelson Chao, David Rizzieri Biology of Blood and Marrow Transplantation Volume 13, Issue 12, Pages 1422-1426 (December 2007) DOI: 10.1016/j.bbmt.2007.08.050 Copyright © 2007 American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 1 Hematopoietic recovery and donor/recipient chimerism following transplantation. A, Sickle cell disease patient #1 white blood cell count and platelet count during the peritransplant period. B, Sickle cell disease patient #1 donor/recipient myeloid and erythroid (as measured by percent hemoglobin A) chimerism and hematocrit following stem cell transplantation. C, Sickle cell disease patient #2 white blood cell count and platelet count during the peritransplant period. D, Sickle cell disease patient #2 donor/recipient myeloid and erythroid (as measured by percent hemoglobin A) chimerism and hematocrit following stem cell transplantation. Of note, the donor for patient #2 has sickle cell trait. Elevated pretransplantation hemoglobin A reflects protocol-mandated exchange transfusions. Biology of Blood and Marrow Transplantation 2007 13, 1422-1426DOI: (10.1016/j.bbmt.2007.08.050) Copyright © 2007 American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 2 T cell and NK cell recovery following stem cell transplantation. Pretransplant and 6-month posttransplant data points are not available for patient #2. Biology of Blood and Marrow Transplantation 2007 13, 1422-1426DOI: (10.1016/j.bbmt.2007.08.050) Copyright © 2007 American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 3 F-ara-A pharmacokinetics in a patient with dialysis-dependent renal failure and sickle cell disease and two patients with normal renal function. Biology of Blood and Marrow Transplantation 2007 13, 1422-1426DOI: (10.1016/j.bbmt.2007.08.050) Copyright © 2007 American Society for Blood and Marrow Transplantation Terms and Conditions