Monoclonal Antibody T-Cell-Depleted HLA-Haploidentical Bone Marrow Transplantation for Wiskott-Aldrich Syndrome

• Published in: Blood Vol. 75; no. 4; pp. 1031 - 1035
• Main Authors: Rumelhart, Stephen L., Trigg, Michael E., Horowitz, Sheldon D., Hong, Richard
• Format: Journal Article
• Language: English
• Published: Washington, DC Elsevier Inc 15-02-1990; The Americain Society of Hematology
• Subjects: Antibodies, Monoclonal - therapeutic use; Biological and medical sciences; Bone Marrow Cells; Bone Marrow Transplantation - adverse effects; Bone Marrow Transplantation - immunology; Child; Child, Preschool; Complement System Proteins - therapeutic use; Graft vs Host Disease - etiology; Graft vs Host Disease - immunology; Haploidy; HLA Antigens - immunology; Humans; Immunodeficiencies; Immunodeficiencies. Immunoglobulinopathies; Immunopathology; Infant; Lymphocyte Depletion; Medical sciences; T-Lymphocytes - drug effects; Wiskott-Aldrich Syndrome - surgery; Human; Immunopathology; Homograft; Graft versus host reaction; Hemopathy; Monoclonal antibody; HLA»-System; Immune deficiency; Result; Platelet; Depletion; Treatment; Wiskott Aldrich syndrome; T-Lymphocyte; Bone marrow; Graft; Child; Haplotype; Coagulopathy
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood.V75.4.1031.1031

Four patients with Wiskott-Aldrich syndrome received bone marrow transplants (BMT) using monoclonal antibody T cell-depleted HLA-haploidentical marrow from a family member donor. The patients did not receive a significantly larger inoculum of mature T cells than other recipients of T cell-depleted marrow transplants. All four patients achieved quick engraftment, and three of the four patients are alive and well today. The three living patients have all had a complete return of normal T-cell and B-cell function. Infectious complications in the surviving patients were minimal; however, all three experienced some degree of graft-versus-host disease (GVHD). Two of these three patients received GVHD prophylaxis. The patient not receiving GVHD prophylaxis experienced severe GVHD and had a difficult posttransplant course. The patient who did not survive was chronically ill before BMT, whereas the other patients were in relatively good health at the time of BMT. Since the majority of individuals with this disease lack a matched bone marrow donor, our results using partially matched donors suggest that a greater number of patients can be successfully treated for Wiskott-Aldrich syndrome and that outcome is related to control of GVHD and state of health before BMT. Marrow transplantation should be offered earlier in the disease course before the onset of major infectious problems.