Long-Term Outcome and Evaluation of Organ Function in Pediatric Patients Undergoing Haploidentical and Matched Related Hematopoietic Cell Transplantation.

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Long-Term Outcome and Evaluation of Organ Function in Pediatric Patients Undergoing Haploidentical and Matched Related Hematopoietic Cell Transplantation for Sickle Cell Disease Mari H. Dallas, Brandon Triplett, David R. Shook, Christine Hartford, Ashok Srinivasan, Joseph Laver, Russell Ware, Wing Leung Biology of Blood and Marrow Transplantation Volume 19, Issue 5, Pages 820-830 (May 2013) DOI: 10.1016/j.bbmt.2013.02.010 Copyright © 2013 American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 1 Kaplan-Meier estimates of OS and DFS after MRD HSCT and haploidentical HSCT. OS and DFS were determined with events defined as death, graft rejection, or recurrence of SCD after MRD HSCT (A) or haploidentical HSCT (B). Tick marks represent surviving patients and indicate the duration of follow-up after transplantation. The percentages above each curve indicate the estimates of OS, DFS, and cumulative incidence of graft rejection or recurrence of disease for the entire cohort. Biology of Blood and Marrow Transplantation 2013 19, 820-830DOI: (10.1016/j.bbmt.2013.02.010) Copyright © 2013 American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 2 Disease response after MRD HSCT (solid lines) and haploidentical HSCT (dashed lines) for SCD. Mean ± SEM values are plotted over time after MRD HSCT and haploidentical HSCT for hemoglobin and hematocrit (A), hemoglobin fractions (B), reticulocyte index and percent (C), and iron and ferritin levels (D). Biology of Blood and Marrow Transplantation 2013 19, 820-830DOI: (10.1016/j.bbmt.2013.02.010) Copyright © 2013 American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 3 IQ, renal function, and cardiac function before and after HSCT. IQ (A), renal function (B), and cardiac function (C) were monitored before (solid symbols) and yearly after (open symbols) MRD (●, ○) and haploidentical (HAPLO) (■, □) HSCT. Mean ± SEM values are plotted over time after HSCT for CrCl (Bi) and shortening fraction (Ci), and median and SEM values for CrCl (Bii) and shortening fraction (Cii) before HSCT are compared with the values measured at the most recent evaluation. Biology of Blood and Marrow Transplantation 2013 19, 820-830DOI: (10.1016/j.bbmt.2013.02.010) Copyright © 2013 American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 4 Pulmonary function before and after HSCT. PFTs, including FVC (A), DLCO (B), FEV1 (C), and FEV1/FVC ratio (D), were monitored before (solid symbols) and yearly after (open symbols) HSCT. Mean ± SEM values are plotted over time after MRD (●, ○) and haploidentical (HAPLO) (■, □) HSCT for FVC (Ai) and DLCO (Bi). The median ± SEM values before HSCT or first available values (closed symbols) are compared with the most recent value (open symbol) for FVC (Aii), DLCO (Bii), FEV1 (Ci), and FEV1/FVC ratio (Cii). Biology of Blood and Marrow Transplantation 2013 19, 820-830DOI: (10.1016/j.bbmt.2013.02.010) Copyright © 2013 American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 5 Bone density before and after HSCT. Whole-body (A) and vertebral body (B) BMD (i) and BMD z-scores (ii) were monitored before (solid symbols) and after (open symbols) HSCT. Median ± SEM values for the MRD (●, ○) and haploidentical (HAPLO) (■, □) recipients are compared with those measured at the most recent evaluation. Biology of Blood and Marrow Transplantation 2013 19, 820-830DOI: (10.1016/j.bbmt.2013.02.010) Copyright © 2013 American Society for Blood and Marrow Transplantation Terms and Conditions