Nonmyeloablative Stem Cell Transplantation with Alemtuzumab/Low-Dose Irradiation to Cure and Improve the Quality of Life of Adults with Sickle Cell Disease

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Presentation transcript:

Nonmyeloablative Stem Cell Transplantation with Alemtuzumab/Low-Dose Irradiation to Cure and Improve the Quality of Life of Adults with Sickle Cell Disease Santosh L. Saraf, Annie L. Oh, Pritesh R. Patel, Yash Jalundhwala, Karen Sweiss, Matthew Koshy, Sally Campbell-Lee, Michel Gowhari, Johara Hassan, David Peace, John G. Quigley, Irum Khan, Robert E. Molokie, Lewis L. Hsu, Nadim Mahmud, Dennis J. Levinson, A. Simon Pickard, Joe G.N. Garcia, Victor R. Gordeuk, Damiano Rondelli Biology of Blood and Marrow Transplantation Volume 22, Issue 3, Pages 441-448 (March 2016) DOI: 10.1016/j.bbmt.2015.08.036 Copyright © 2016 American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 1 Neutropenia after alemtuzumab/TBI conditioning in adult SCD patients. Neutrophil counts after conditioning with alemtuzumab/total body irradiation and hematopoietic stem cell transplantation. Each value represents the mean neutrophil count ± standard error (n = 13). Biology of Blood and Marrow Transplantation 2016 22, 441-448DOI: (10.1016/j.bbmt.2015.08.036) Copyright © 2016 American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 2 Engraftment analysis after alemtuzumab/TBI conditioning in adult SCD patients. Whole blood mononuclear cell (MNC) and CD3+ lymphoid cell chimerism values at day +90, day +180, and day +365 after hematopoietic stem cell transplantation (HSCT) in the 12 patients with stable engraftment. Biology of Blood and Marrow Transplantation 2016 22, 441-448DOI: (10.1016/j.bbmt.2015.08.036) Copyright © 2016 American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 3 Normalization of hemoglobin after alemtuzumab/TBI conditioning in adult SCD patients. Hemoglobin fractionation studies and total hemoglobin concentrations after hematopoietic stem cell transplantation in 12 patients with stable engraftment. (A) The proportion of hemoglobin A in the recipients was stable and consistent with the proportion observed in the donor. (B) Mean hemoglobin concentrations ± standard errors by sex of recipient. Biology of Blood and Marrow Transplantation 2016 22, 441-448DOI: (10.1016/j.bbmt.2015.08.036) Copyright © 2016 American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 4 Cardiopulmonary improvement after alemtuzumab/TBI conditioning in adult SCD patients. Cardiopulmonary improvement at 1 year post-hematopoietic stem cell transplantation was observed in 12 patients with stable engraftment. (A) Brain-natriuretic peptide (BNP) concentrations, (B) Left atrial diameter, (C) Forced expiratory volume at 1 second (FEV1), and (D) Forced vital capacity (FVC) measurements are given as mean values ± standard error. Biology of Blood and Marrow Transplantation 2016 22, 441-448DOI: (10.1016/j.bbmt.2015.08.036) Copyright © 2016 American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 5 Rapid and sustained improvement in the HRQoL after HSCT in adult SCD patients. Improvements in normalized HRQoL scores and SF-6D scores between before and after HSCT in 9 patients with stable engraftment. (A) SF-36 mean scores ± standard error across the eight HRQoL domains. Differences between pre- and post-HRQoL scores were analyzed and P values are shown. (B) SF-6D mean scores ± standard error at each time point of assessment. Δ > .033 represents the commonly accepted minimally important difference. Biology of Blood and Marrow Transplantation 2016 22, 441-448DOI: (10.1016/j.bbmt.2015.08.036) Copyright © 2016 American Society for Blood and Marrow Transplantation Terms and Conditions