Patients with SCID have a poor prognosis and in most cases do not survive more than a few months without appropriate treatment. Supportive treatment with intravenous immunoglobulin infusions at regular intervals, and prophylaxis for Pneumocystis car-inii is indicated. Bacterial or fungal infections are treated with intravenous infusions of antibiotics or antifungal agents. Epstein-Barr virus, herpes virus and cytomegalovirus infection may result in systemic and lethal disorders and should be treated with antiviral reagents. Prophylactic infusions of immunoglobulin with high titer for cytomegalovirus has been advocated. For ADA deficiency, enzyme replacement with polyethylene glycol-modified ADA (PEG-ADA) resulted in limited but significant improvement of the immunodeficiency.
To reconstitute the impaired immune system in SCID, bone marrow transplantation is the treatment of choice. If successfully engrafted, BMT is a curative treatment, although incomplete reconstitution of the immune system and post-transplant lymphoprolifer-ative disorders caused by Epstein-Barr virus are major problems that need to be solved. To improve engraftment, various conditioning regimens including chemotherapy, total body irradiation, and anti-lymphocyte globulin (ALG) have been tried. HLA-haploidentical marrow from a parent can be used for BMT, although T cell depletion is necessary to prevent GVHD. Recently, a successful in utero transplantation of parental CD34+ hematopoietic progenitor cells into an X-SCID fetus has been reported.
Gene therapy has been attempted in patients with ADA deficiency. Two patients repeatedly received infusions of autologous T cells transduced with the ADA gene inserted in a retroviral vector. Circulating T cells producing ADA were detected, and a possible improvement of the immune responses were observed, including increased numbers of T cells, appearance of T cell cytotoxicity, positive delayed type hypersensitivity skin tests, and proliferative responses to antigen by specific T cells. The fact that these patients also received PEG-ADA makes the interpretation of these findings difficult. Although many technological breakthroughs are needed, gene therapy has the potential to become a curative therapy for some primary immunodeficiencies whose genetic bases have been identified.
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