Alternative donor transplants for severe aplastic anemia: current experience
Andrea Bacigalupo1, Simona Sica1
1Department of Hematology, Universita' Cattolica del Sacro Cuore, Fondazione Policlinico Universitario Gemelli Roma, Largo Agostino Gemelli 1, Rome, Italy.
Seminars in Hematology
|March 23, 2016
Summary
For severe aplastic anemia (SAA) patients without a matched sibling, alternative donor transplants offer improved outcomes. Unrelated donor transplants show significant progress, while haploidentical grafts are promising but experimental.
Area of Science:
- Hematology
- Immunology
- Transplantation Medicine
Background:
- Acquired severe aplastic anemia (SAA) patients lacking an HLA-identical sibling donor face limited therapeutic choices.
- Current European Group for Blood and Marrow Transplantation (EBMT) guidelines recommend immunosuppressive therapy (IST) with anti-thymocyte globulin (ATG) and cyclosporine (CsA) first, followed by transplantation if IST fails.
Purpose of the Study:
- To review recent outcomes of alternative donor stem cell transplantation for SAA patients.
- To evaluate transplants from matched unrelated donors (UD), cord blood (CB), and human leukocyte antigen (HLA)-haploidentical (HAPLO) donors.
Main Methods:
- Review of recent clinical results and outcomes for SAA patients undergoing alternative donor stem cell transplantation.
- Analysis of data concerning matched UD, CB, and HAPLO grafts.
Main Results:
- Transplants from matched UD and sibling (SIB) donors demonstrate significantly improved outcomes, particularly UD transplants over the last decade.
- Cord blood (CB) transplants are discussed, highlighting challenges related to limited stem cell dose.
- Early results for haploidentical (HAPLO) grafts in patients without other donor options are encouraging, though this approach remains experimental.
Conclusions:
- Alternative donor transplantation, especially from matched unrelated donors, offers a viable and improving therapeutic option for SAA patients.
- Cord blood and haploidentical stem cell transplantation represent developing strategies with specific challenges and potential benefits.
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