Transplant for non-malignant disorders: An International Society for Cell & Gene Therapy Stem Cell Engineering Committee report on the role of alternative donors, stem cell sources and graft engineering Guidelines


Authors: Klein, O. R.; Bonfim, C.; Abraham, A.; Ruggeri, A.; Purtill, D.; Cohen, S.; Wynn, R.; Russell, A.; Sharma, A.; Ciccocioppo, R.; Prockop, S.; Boelens, J. J.; Bertaina, A.
Title: Transplant for non-malignant disorders: An International Society for Cell & Gene Therapy Stem Cell Engineering Committee report on the role of alternative donors, stem cell sources and graft engineering
Abstract: Hematopoietic stem cell transplantation (HSCT) is curative for many non-malignant disorders. As HSCT and supportive care technologies improve, this life-saving treatment may be offered to more and more patients. With the development of new preparative regimens, expanded alternative donor availability, and graft manipulation techniques, there are many options when choosing the best regimen for patients. Herein the authors review transplant considerations, transplant goals, conditioning regimens, donor choice, and graft manipulation strategies for patients with non-malignant disorders undergoing HSCT. © 2023 International Society for Cell & Gene Therapy
Keywords: transplantation, homologous; busulfan; fludarabine; hydroxyurea; cyclophosphamide; dexamethasone; hematopoietic stem cell transplantation; cord blood stem cell transplantation; engraftment; myeloablative conditioning; nonmyeloablative conditioning; whole body radiation; donor; tissue donors; screening; graft versus host reaction; transplantation conditioning; azathioprine; immunosuppressive treatment; graft vs host disease; allotransplantation; thymocyte antibody; adverse event; hemoglobinopathies; metabolic disorders; treosulfan; humans; human; article; haploidentical donor; severe aplastic anemia; bone marrow failure disorders; primary immune deficiency disorders; primary immune regulatory disorders
Journal Title: Cytotherapy
Volume: 25
Issue: 5
ISSN: 1465-3249
Publisher: Elsevier Science Ltd.  
Date Published: 2023-05-01
Start Page: 463
End Page: 471
Language: English
DOI: 10.1016/j.jcyt.2022.12.005
PUBMED: 36710227
PROVIDER: scopus
DOI/URL:
Notes: Article -- Export Date: 1 May 2023 -- Source: Scopus
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  1. Jaap Jan Boelens
    211 Boelens