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Bone marrow transplantation in the treatment of severe immunodeficiencies: possibilities and problems
1Department of Pediatrics, University Hospital Leiden, The Netherlands.
Infants and children suffering from severe primary immunodeficiencies may be cured by bone marrow transplantation from a healthy donor. Data obtained in 14 European centers show that about 60% of the patients are surviving disease-free, if they were grafted with bone marrow cells from an HLA-identical related donor. Results of transplantation of T-cell depleted bone marrow from an HLA-haploidentical related donor were also excellent in infants with severe combined immunodeficiency, with 60% recovery. This therapy is superior to transplantation of fetal tissues. HLA-haploidentical T-cell depleted marrow transplantation for other severe immunodeficiencies was less successful. This was mainly due to failure of engraftment, despite intensive conditioning of the recipient, and to infectious complications e.g. by reactivation of latently present viruses.
Infants and children suffering from severe primary immunodeficiencies may be cured by bone marrow transplantation from a healthy donor. Data obtained in 14 European centers show that about 60% of the patients are surviving disease-free, if they were grafted with bone marrow cells from an HLA-identical related donor. Results of transplantation of T-cell depleted bone marrow from an HLA-haploidentical related donor were also excellent in infants with severe combined immunodeficiency, with 60% recovery. This therapy is superior to transplantation of fetal tissues. HLA-haploidentical T-cell depleted marrow transplantation for other severe immunodeficiencies was less successful. This was mainly due to failure of engraftment, despite intensive conditioning of the recipient, and to infectious complications e.g. by reactivation of latently present viruses.