Transplantation for bone marrow failure: current issues
- 2 December 2016
- journal article
- research article
- Published by American Society of Hematology in Hematology
- Vol. 2016 (1), 90-98
- https://doi.org/10.1182/asheducation-2016.1.90
Abstract
The preferred treatment of idiopathic aplastic anemia (AA) is allogeneic hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA)–identical sibling donor. Transplantation from a well-matched unrelated donor (MUD) may be considered for patients without a sibling donor after failure of immunosuppressive therapy, as may alternative transplantation (mismatched, cord blood or haplo-identical HSCT) for patients without a MUD. HSCT may also be contemplated for congenital disorders in cases of pancytopenia or severe isolated cytopenia. Currently, HSCT aims are not only to cure patients but also to avoid long-term complications, notably chronic graft-versus-host disease (GVHD), essential for a good quality of life long term. This paper summarizes recent advances in HSCT for idiopathic and inherited AA disorders. The effect of age on current transplantation outcomes, the role of transplantation in paroxysmal nocturnal hemoglobinuria, and the prevention of GVHD are also discussed. Emerging strategies regarding the role of up-front unrelated donor and alternative donor HSCT in idiopathic AA, along with advances in the treatment of clonal evolution in Fanconi anemia, are also examined.This publication has 57 references indexed in Scilit:
- Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuriaHaematologica, 2012
- Excellent outcome of matched unrelated donor transplantation in paediatric aplastic anaemia following failure with immunosuppressive therapy: a United Kingdom multicentre retrospective experienceBritish Journal of Haematology, 2012
- Long-term follow up after allogeneic stem cell transplantation in patients with severe aplastic anemia after cyclophosphamide plus antithymocyte globulin conditioningHaematologica, 2011
- Late effects among pediatric patients followed for nearly 4 decades after transplantation for severe aplastic anemiaBlood, 2011
- Decreased Infection-Related Mortality and Improved Survival in Severe Aplastic Anemia in the Past Two DecadesClinical Infectious Diseases, 2011
- Influence of Nucleated Cell Dose on Overall Survival of Unrelated Cord Blood Transplantation for Patients with Severe Acquired Aplastic Anemia: A Study by Eurocord and the Aplastic Anemia Working Party of the European Group for Blood and Marrow TransplantationTransplantation and Cellular Therapy, 2011
- Impact of age on outcomes after bone marrow transplantation for acquired aplastic anemia using HLA-matched sibling donorsHaematologica, 2010
- Fludarabine, cyclophosphamide, antithymocyte globulin, with or without low dose total body irradiation, for alternative donor transplants, in acquired severe aplastic anemia: a retrospective study from the EBMT-SAA working partyHaematologica, 2010
- Improved outcome of patients older than 30 years receiving HLA-identical sibling hematopoietic stem cell transplantation for severe acquired aplastic anemia using fludarabine-based conditioning: a comparison with conventional conditioning regimenHaematologica, 2009
- HLA-Matched Related Donor Hematopoietic Cell Transplantation in 43 Patients with Fanconi Anemia Conditioned with 60 mg/kg of CyclophosphamideTransplantation and Cellular Therapy, 2007