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Outcomes of Unrelated Donor Stem Cell Transplantion with Post-Transplant Cyclophosphamide for Graft-versus-Host Disease Prophylaxis in Patients with Severe Sickle Cell Disease.
- Source :
-
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation [Biol Blood Marrow Transplant] 2018 Feb; Vol. 24 (2), pp. 413-417. Date of Electronic Publication: 2017 Oct 20. - Publication Year :
- 2018
-
Abstract
- Unrelated donor (URD) hematopoietic cell transplantation (HCT) in children with sickle cell disease (SCD) is associated with a high incidence of rejection and graft-versus-host disease (GVHD). We report on the first 4 patients with severe SCD who underwent URD HCT using a novel myeloablative and immunosuppressive regimen composed of busulfan, fludarabine, and antithymocyte globulin with a single dose of post-transplant cyclophosphamide along with tacrolimus and mycophenolate mofetil for GVHD prophylaxis. Three patients engrafted and remain disease-free after a median follow-up period of 2.5 years. One patient had primary graft failure attributed to low stem cell content of the graft. Of interest, none of the engrafted patients developed acute or chronic GVHD. This preparative regimen along with the use of post-transplant cyclophosphamide offers a promising approach for unrelated donor transplants in patients with SCD and needs further corroboration in larger number of patients.<br /> (Copyright © 2017 The American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.)
- Subjects :
- Adolescent
Antilymphocyte Serum
Antineoplastic Combined Chemotherapy Protocols therapeutic use
Busulfan
Child
Child, Preschool
Female
Humans
Male
Mycophenolic Acid
Premedication methods
Tacrolimus
Tissue Donors
Transplantation, Homologous
Treatment Outcome
Vidarabine analogs & derivatives
Young Adult
Anemia, Sickle Cell therapy
Cyclophosphamide therapeutic use
Graft vs Host Disease prevention & control
Hematopoietic Stem Cell Transplantation methods
Subjects
Details
- Language :
- English
- ISSN :
- 1523-6536
- Volume :
- 24
- Issue :
- 2
- Database :
- MEDLINE
- Journal :
- Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
- Publication Type :
- Academic Journal
- Accession number :
- 29061531
- Full Text :
- https://doi.org/10.1016/j.bbmt.2017.10.030