Treosulfan and Fludarabine Conditioning for Hematopoietic Stem Cell Transplantation in Children with Primary Immunodeficiency: UK Experience Mary A.

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Treosulfan-based conditioning regimens for hematopoietic stem cell transplantation in children with primary immunodeficiency: United Kingdom experience.

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Presentation transcript:

Treosulfan and Fludarabine Conditioning for Hematopoietic Stem Cell Transplantation in Children with Primary Immunodeficiency: UK Experience Mary A. Slatter, Kanchan Rao, Intan Juliana Abd Hamid, Zohreh Nademi, Robert Chiesa, Reem Elfeky, Mark S. Pearce, Persis Amrolia, Austen Worth, Terence Flood, Mario Abinun, Sophie Hambleton, Waseem Qasim, Hubert B. Gaspar, Andrew J. Cant, Andrew R. Gennery, Paul Veys Biology of Blood and Marrow Transplantation Volume 24, Issue 3, Pages 529-536 (March 2018) DOI: 10.1016/j.bbmt.2017.11.009 Copyright © 2017 The American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 1 OS. Biology of Blood and Marrow Transplantation 2018 24, 529-536DOI: (10.1016/j.bbmt.2017.11.009) Copyright © 2017 The American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 2 Event-free survival. An event was death or additional procedure. Biology of Blood and Marrow Transplantation 2018 24, 529-536DOI: (10.1016/j.bbmt.2017.11.009) Copyright © 2017 The American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 3 T and myeloid cell chimerism according to stem cell source. All patients who survived longer than 1 year post-HSCT were included. Four patients without split cell lineage chimerism data were excluded. Biology of Blood and Marrow Transplantation 2018 24, 529-536DOI: (10.1016/j.bbmt.2017.11.009) Copyright © 2017 The American Society for Blood and Marrow Transplantation Terms and Conditions

Figure 4 OS by diagnosis. Survival at 2 years post-HSCT: SCID, 94.6% (95% CI, 80.2% to 98.6%); WAS, 100%; CGD, 93.7% (95% CI, 63.2% to 99.1%); HLH, 62.5% (95% CI, 34.8% to 81.0%). P = .0001, log-rank test. Biology of Blood and Marrow Transplantation 2018 24, 529-536DOI: (10.1016/j.bbmt.2017.11.009) Copyright © 2017 The American Society for Blood and Marrow Transplantation Terms and Conditions