Abstract
Six children with severe combined immunodeficiency disease have received bone marrow transplants from a haplotype mismatched parent. The marrow was treated by monoclonal antibody and complement prior to infusion. Complicating factors involved the presence of prior maternal graft versus host in 1, prior thymus transplantation in 1 and presence of sufficient native immunity to require ablation in one. Two children died without engraftment. The survivors have B and T cell engraftment as demonstrated by normal in vitro T cell proliferative responses, normal immunoglobul in and/or functional antibody levels except one of the patients appears to have IgA deficiency. The longest period of follow up is 20 months and the shortest is three months. In 1 patient, thymus biopsy after transplant confirmed a normal distribution of cells and the presence of a marker for dendritic cells. This marker was not present in a patient who was not successfully engrafted.
These data show: 1. Successful engraftment can be accomplished with haplotype mismatched marrow even in the face of maternal graft versus host disease. 2. Haplotype mismatched marrow can home to the host thymus and be appropriately differentiated. 3. Criteria for prior ablative therapy need to be established; thymus immunohistochemistry may be helpful in this regard. 4. Mechanisms of the immune response can be inferred from the nature of the reconstitution.
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Moen, R., Hong, R., Stiehm, E. et al. 1008 THEORETICAL AND BIOLOGICAL CONSIDERATIONS IN SUCCESSFUL MISMATCHED BONE MARROW TRANSPLANTATION. Pediatr Res 19, 278 (1985). https://doi.org/10.1203/00006450-198504000-01038
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DOI: https://doi.org/10.1203/00006450-198504000-01038