831 wiskott-aldrich syndrome: immune status after bone marrow transplantation

831 wiskott-aldrich syndrome: immune status after bone marrow transplantation

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ABSTRACT An 18 month old boy with Wiskott-Aldrich Syndrome (WAS) received a marrow transplant from an HLA-matched sister after being conditioned with cyclophosphamide and dimethyl-myleran; post transplant methotrexate was given for 6 weeks. Complete hematologic reconstitution with donor cells occurred. In vitro immunoglobulin (Ig) synthesis was determined with a direct and an indirect hemolytic plaque assay using pokeweed mitogen activation and co-culture techniques. Before transplantation, Ig secretion of B cells was impaired; T-cells provided adequate help and suppressor T-cells were not detected. Following transplantation, a transient impairment of IgG secretion by patient's B-cells in the presence of autologous or normal allogeneic T-cells was observed. During three months of post-transplant study, T-helper activity was persistently defective and Ig secretion by normal B and T cells was suppressed (> 95%) by the patient's T cells. Recovery from the transplant procedure was rapid and 3 months after the transplant the patient was infection-free and without signs of graft versus host disease; serum immunoglobulin concentrations were normal and isohemagglutinins became demonstrable; he responded to bacteriophage ØX 174 with adequate antibody titers and in vitro lymphocyte transformation to mitogens was 16-51% of normal. Presently, eight months after the transplant, he is asymptomatic and thriving. These observations indicate that hematologic and immunologic reconstitution can be achieved in WAS without total body irradiation prior to marrow infusion, and that the appearance of donor derived suppressor T cells may play a role in the homeostasis between the host and graft. ARTICLE PDF AUTHOR INFORMATION AUTHORS AND AFFILIATIONS * Departments of Pediatrics and Medicine, University of Washington, Seattle Lawrence G Lum, Hans D Ochs, F Leonard Johnson & Rainer F Storb Authors * Lawrence G Lum View author publications You can also search for this author inPubMed Google Scholar * Hans D Ochs View author publications You can also search for this author inPubMed Google Scholar * F Leonard Johnson View author publications You can also search for this author inPubMed Google Scholar * Rainer F Storb View author publications You can also search for this author inPubMed Google Scholar RIGHTS AND PERMISSIONS Reprints and permissions ABOUT THIS ARTICLE CITE THIS ARTICLE Lum, L., Ochs, H., Johnson, F. _et al._ 831 WISKOTT-ALDRICH SYNDROME: IMMUNE STATUS AFTER BONE MARROW TRANSPLANTATION. _Pediatr Res_ 15 (Suppl 4), 581 (1981). https://doi.org/10.1203/00006450-198104001-00856 Download citation * Issue Date: 01 April 1981 * DOI: https://doi.org/10.1203/00006450-198104001-00856 SHARE THIS ARTICLE Anyone you share the following link with will be able to read this content: Get shareable link Sorry, a shareable link is not currently available for this article. Copy to clipboard Provided by the Springer Nature SharedIt content-sharing initiative

ABSTRACT An 18 month old boy with Wiskott-Aldrich Syndrome (WAS) received a marrow transplant from an HLA-matched sister after being conditioned with cyclophosphamide and dimethyl-myleran;


post transplant methotrexate was given for 6 weeks. Complete hematologic reconstitution with donor cells occurred. In vitro immunoglobulin (Ig) synthesis was determined with a direct and an


indirect hemolytic plaque assay using pokeweed mitogen activation and co-culture techniques. Before transplantation, Ig secretion of B cells was impaired; T-cells provided adequate help and


suppressor T-cells were not detected. Following transplantation, a transient impairment of IgG secretion by patient's B-cells in the presence of autologous or normal allogeneic T-cells


was observed. During three months of post-transplant study, T-helper activity was persistently defective and Ig secretion by normal B and T cells was suppressed (> 95%) by the


patient's T cells. Recovery from the transplant procedure was rapid and 3 months after the transplant the patient was infection-free and without signs of graft versus host disease;


serum immunoglobulin concentrations were normal and isohemagglutinins became demonstrable; he responded to bacteriophage ØX 174 with adequate antibody titers and in vitro lymphocyte


transformation to mitogens was 16-51% of normal. Presently, eight months after the transplant, he is asymptomatic and thriving. These observations indicate that hematologic and immunologic


reconstitution can be achieved in WAS without total body irradiation prior to marrow infusion, and that the appearance of donor derived suppressor T cells may play a role in the homeostasis


between the host and graft. ARTICLE PDF AUTHOR INFORMATION AUTHORS AND AFFILIATIONS * Departments of Pediatrics and Medicine, University of Washington, Seattle Lawrence G Lum, Hans D Ochs, F


Leonard Johnson & Rainer F Storb Authors * Lawrence G Lum View author publications You can also search for this author inPubMed Google Scholar * Hans D Ochs View author publications You


can also search for this author inPubMed Google Scholar * F Leonard Johnson View author publications You can also search for this author inPubMed Google Scholar * Rainer F Storb View author


publications You can also search for this author inPubMed Google Scholar RIGHTS AND PERMISSIONS Reprints and permissions ABOUT THIS ARTICLE CITE THIS ARTICLE Lum, L., Ochs, H., Johnson, F.


_et al._ 831 WISKOTT-ALDRICH SYNDROME: IMMUNE STATUS AFTER BONE MARROW TRANSPLANTATION. _Pediatr Res_ 15 (Suppl 4), 581 (1981). https://doi.org/10.1203/00006450-198104001-00856 Download


citation * Issue Date: 01 April 1981 * DOI: https://doi.org/10.1203/00006450-198104001-00856 SHARE THIS ARTICLE Anyone you share the following link with will be able to read this content:


Get shareable link Sorry, a shareable link is not currently available for this article. Copy to clipboard Provided by the Springer Nature SharedIt content-sharing initiative