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Role of reduced intensity conditioning in T-cell and B-cell immune reconstitution after HLA-identical bone marrow transplantation in adenosine-deaminase severe combined immunodeficiency
Role of reduced intensity conditioning in T-cell and B-cell immune reconstitution after HLA-identical bone marrow transplantation in adenosine-deaminase severe combined immunodeficiency HAEMATOLOGICA-THE HEMATOLOGY JOURNAL Cancrini, C., Ferrua, F., Scarselli, A., Brigida, I., Romiti, M. L., Barera, G., Finocchi, A., Roncarolo, M. G., Caniglia, M., Aiuti, A. 2010; 95 (10): 1778-1782Abstract
The treatment of choice for severe combined immunodeficiency is bone marrow transplantation from an HLA-identical donor sibling without conditioning. However, this may result in low donor stem cell chimerism, leading to reduced long-term immune reconstitution. We compared engraftment, metabolic, and T-cell and B-cell immune reconstitution of HLA-identical sibling bone marrow transplantation performed in 2 severe combined immunodeficiency infants with adenosine deaminase deficiency from the same family treated with or without a reduced intensity conditioning regimen (busulfan/fludarabine). Only the patient who received conditioning showed a stable mixed chimerism in all lineages, including bone marrow myeloid and B cells. The use of conditioning resulted in higher thymus-derived naïve T cells and T-cell receptor excision circles, normalization of the T-cell repertoire, and faster and complete B-cell and metabolic reconstitution. These results suggest the utility of exploring the use of reduced intensity conditioning in bone marrow transplantation from HLA-identical donor in severe combined immunodeficiency to improve long-term immune reconstitution.
View details for DOI 10.3324/haematol.2010.025098
View details for Web of Science ID 000283275000023
View details for PubMedID 20460637