Abstract
Severe combined immune deficiency (SCID) associated with adenosine deaminase (ADA) deficiency presents an excellent model to study enzyme replacement therapy. To evaluate the effectiveness of enzyme replacement and other therapeutic regimens, we compared antibody responses to a T-dependent neoantigen, bacteriophage Ø× 174 in 4 patients with ADA deficiency: one was treated with repeated red blood cell transfusions, one with a single infusion of peripheral blood mononuclear cells, one with fetal liver cells, and one with bone marrow from an HLA matched sibling. Normal antibody responses to this antigen consist of a primary IgM response followed by a brisk secondary IgG response of high titer. The antibody responses of the patients varied depending on the mode of therapy. Enzyme replacement therapy by transfusion of red blood cells, or transplantation of peripheral blood mononuclear cells, although associated with clinical improvement and partial immune reconstitution if recall antigens or mitogens were used, did not restore the antibody responses to phage: neither amplification nor IgG production occurred. The infant transplanted with fetal liver cells responded with low antibody titers but a moderate IgG response (22%) was found. Only the child treated with matched bone marrow showed a quantitatively and qualitatively normal antibody response. These results demonstrate that extrinsic enzyme replacement alone is insufficient in this syndrome and that a full set of normal uncommitted lymphoid stem cells is required for complete immune reconstitution.
Article PDF
Author information
Authors and Affiliations
Rights and permissions
About this article
Cite this article
Ochs, H., Rich, K., Horowitz, S. et al. 952 IMMUNE RECONSTITUTION IN ADENOSINE DEAMINASE DEFICIENT SEVERE COMBINED IMMUNE DEFICIENCY. Pediatr Res 15 (Suppl 4), 601 (1981). https://doi.org/10.1203/00006450-198104001-00977
Issue Date:
DOI: https://doi.org/10.1203/00006450-198104001-00977