Towards graft-<i>versus</i>-host disease-free alternative donor transplant platforms for patients with acquired aplastic anemia
Hematopoietic stem cell transplantation is a well-established treatment option for acquired aplastic anemia. Historically, upfront hematopoietic stem cell transplantation (HCT) with HLA-matched sibling donors is used in young patients and immunosuppressive therapy (IST) is used for all others. Over...
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| Format: | Article |
| Language: | English |
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Ferrata Storti Foundation
2025-05-01
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| Series: | Haematologica |
| Online Access: | https://haematologica.org/article/view/12079 |
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| author | Joseph H. Oved Andrea Bacigalupo Amy E. DeZern |
| author_facet | Joseph H. Oved Andrea Bacigalupo Amy E. DeZern |
| author_sort | Joseph H. Oved |
| collection | DOAJ |
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Hematopoietic stem cell transplantation is a well-established treatment option for acquired aplastic anemia. Historically, upfront hematopoietic stem cell transplantation (HCT) with HLA-matched sibling donors is used in young patients and immunosuppressive therapy (IST) is used for all others. Over time, innovations in the transplant platform have decreased unwanted complications such as graft versus host disease (GVHD), failure to engraft, and infections thereby expanding the use of alternative donors. This has led to an evolution towards HCT and away from IST despite increased intensity of treatment due to improved event free survival. The ideal conditioning regimen for HCT in aplastic anemia results in sustained engraftment, minimal toxicity, lack of GVHD and is not limited by donor availability. Two transplant platforms have been refined to meet these needs. TCR αβ+ T cell depletion has excellent outcomes with minimal graft-rejection and GVHD in the matched and mismatched unrelated donor setting. Haploidentical grafts however still require further optimization. Bone marrow grafts given with ATG and post-transplant cyclophosphamide have similarly excellent results with extremely low rates of GVHD. Peripheral mobilized grafts need further optimization in this setting. This review provides an overview of the current perspective on regimens that minimize GVHD in aplastic anemia and how they can be further refined so that all patients with aplastic anemia have curative therapy available to them.
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| format | Article |
| id | doaj-art-f72a46cbe7c84b1b86f20ca811b6e5d8 |
| institution | OA Journals |
| issn | 0390-6078 1592-8721 |
| language | English |
| publishDate | 2025-05-01 |
| publisher | Ferrata Storti Foundation |
| record_format | Article |
| series | Haematologica |
| spelling | doaj-art-f72a46cbe7c84b1b86f20ca811b6e5d82025-08-20T02:34:33ZengFerrata Storti FoundationHaematologica0390-60781592-87212025-05-01999110.3324/haematol.2024.286544Towards graft-<i>versus</i>-host disease-free alternative donor transplant platforms for patients with acquired aplastic anemiaJoseph H. Oved0Andrea Bacigalupo1Amy E. DeZern2Division of Transplantation and Cellular Therapies, Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, NY 10065Dipartimento di Diagnostica per Immagini, Radioterapia Oncologica ed Ematologia, Università Cattolica del Sacro Cuore, Roma, Italy; Dipartimento di Scienze di Laboratorio ed Ematologiche, Fondazione Policlinico Universitario A. Gemelli IRCCS, RomaDivision of Hematologic Malignancies, The Johns Hopkins University School of Medicine, Baltimore, MD, 21287-0013 Hematopoietic stem cell transplantation is a well-established treatment option for acquired aplastic anemia. Historically, upfront hematopoietic stem cell transplantation (HCT) with HLA-matched sibling donors is used in young patients and immunosuppressive therapy (IST) is used for all others. Over time, innovations in the transplant platform have decreased unwanted complications such as graft versus host disease (GVHD), failure to engraft, and infections thereby expanding the use of alternative donors. This has led to an evolution towards HCT and away from IST despite increased intensity of treatment due to improved event free survival. The ideal conditioning regimen for HCT in aplastic anemia results in sustained engraftment, minimal toxicity, lack of GVHD and is not limited by donor availability. Two transplant platforms have been refined to meet these needs. TCR αβ+ T cell depletion has excellent outcomes with minimal graft-rejection and GVHD in the matched and mismatched unrelated donor setting. Haploidentical grafts however still require further optimization. Bone marrow grafts given with ATG and post-transplant cyclophosphamide have similarly excellent results with extremely low rates of GVHD. Peripheral mobilized grafts need further optimization in this setting. This review provides an overview of the current perspective on regimens that minimize GVHD in aplastic anemia and how they can be further refined so that all patients with aplastic anemia have curative therapy available to them. https://haematologica.org/article/view/12079 |
| spellingShingle | Joseph H. Oved Andrea Bacigalupo Amy E. DeZern Towards graft-<i>versus</i>-host disease-free alternative donor transplant platforms for patients with acquired aplastic anemia Haematologica |
| title | Towards graft-<i>versus</i>-host disease-free alternative donor transplant platforms for patients with acquired aplastic anemia |
| title_full | Towards graft-<i>versus</i>-host disease-free alternative donor transplant platforms for patients with acquired aplastic anemia |
| title_fullStr | Towards graft-<i>versus</i>-host disease-free alternative donor transplant platforms for patients with acquired aplastic anemia |
| title_full_unstemmed | Towards graft-<i>versus</i>-host disease-free alternative donor transplant platforms for patients with acquired aplastic anemia |
| title_short | Towards graft-<i>versus</i>-host disease-free alternative donor transplant platforms for patients with acquired aplastic anemia |
| title_sort | towards graft i versus i host disease free alternative donor transplant platforms for patients with acquired aplastic anemia |
| url | https://haematologica.org/article/view/12079 |
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