Hematopoietic cell transplantation for aplastic anemia in adults
- Robert S Negrin, MD
Robert S Negrin, MD
- Section Editor — Bone Marrow Transplantation
- Professor of Medicine
- Stanford University School of Medicine
Aplastic anemia (AA) is a potentially fatal condition, especially if the disease does not respond to immunotherapy and/or progresses to severe pancytopenia. The role of hematopoietic cell transplantation (HCT) in these settings is evolving. As outcomes from HCT improve, more patients may be able to benefit from this approach.
This topic review discusses the use of HCT for adults with severe aplastic anemia (SAA). Separate topic reviews discuss the use of HCT for AA in children and adolescents and other therapies for AA in adults.
●HCT for children with AA – (See "Hematopoietic cell transplantation for idiopathic severe aplastic anemia and Fanconi anemia in children and adolescents".)
●Other therapies for adults with AA – (See "Treatment of aplastic anemia in adults".)
Discussions of HCT for other bone marrow failure syndromes are also presented separately. (See "Hematopoietic cell transplantation for idiopathic severe aplastic anemia and Fanconi anemia in children and adolescents" and "Hematopoietic cell transplantation for Diamond-Blackfan anemia and the myelodysplastic syndromes in children and adolescents".)
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- OVERVIEW OF APPROACH
- Decision to use HCT
- Patient/disease/donor factors
- Evidence for efficacy
- PRETRANSPLANT TESTING AND INTERVENTIONS
- Transfusion issues
- OVERVIEW OF HCT CONSIDERATIONS
- Preferred donor and preparative regimen
- - HLA-matched sibling donor
- - Matched unrelated donor
- - Haploidentical related donor
- - Identical twin (syngeneic) donor
- Preferred stem cell source
- GVHD and infection prophylaxis
- Graft failure/graft rejection
- Graft-versus-host disease
- Late malignancy
- Other late complications
- SURVIVAL AND QUALITY OF LIFE
- INVESTIGATIONAL STRATEGIES
- SUMMARY AND RECOMMENDATIONS