Treatment of the immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome (IPEX) by allogeneic bone marrow transplantation

Article Abstract:

A four-month-old child with IPEX syndrome was treated with a bone marrow transplant, but died at the age of three. IPEX syndrome is an X-linked autoimmune disease that is usually fatal.

Care and treatment, Autoimmune diseases

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Anti-B-cell monoclonal antibodies in the treatment of severe B-cell lymphoproliferative syndrome following bone marrow and organ transplantation

Article Abstract:

The immune system contains many checks and balances. Often, the immune response to some infection requires the rapid proliferation (multiplication) of some white blood cells, often B lymphocytes, but the checks and balances prevent this proliferation from going too far once the necessary immune response is complete. In some conditions, however, the regulatory system breaks down. Normal individuals routinely suppress infection with Epstein-Barr virus without any awareness. However, if a person is tired or run down, the proliferation of cells generated to fight the virus may not be halted by the checks and balances, and mononucleosis results. If a patient is severely immunocompromised the same sort of proliferative response of the B lymphocytes rages unchecked in a potentially deadly B-lymphoproliferative syndrome. Patients receiving transplants of bone marrow or other organs are often treated to suppress the immune response (to prevent rejection of the donated tissue). While the risk of infection in these patients is far greater than the risk of lymphoproliferative syndrome, if lymphoproliferative disease occurs it can be deadly. A study was undertaken to determine the feasibility of using antibodies against human B cells to bring B lymphoproliferative syndrome into check. Two different antibodies that react specifically with B cells were used to treat the disorder in 14 patients following bone marrow transplantation and in 12 patients following transplantation of organs. The prognosis for B-lymphoproliferative syndrome is particularly poor in patients with donor bone marrow; 29 of 32 reported patients have died. In the present series of patients, 16 patients were able to achieve complete remission. Five of 14 bone marrow patients and 6 of 12 organ transplant recipients remain alive and free of disease an average of 35 months after treatment. This study was not controlled, and the grave nature of the disorder required that the antibody treatment was not the only treatment administered to these patients. However, although the recovery of the patients can not be attributed to the antibody treatment with certainty, the results suggest that antibodies against B cells may be effective in controlling some forms of B-lymphoproliferative disorder. (Consumer Summary produced by Reliance Medical Information, Inc.)

Health aspects, Complications and side effects, Drug therapy, Lymphoproliferative disorders, Immunoproliferative disorders, Monoclonal antibodies, Transplantation of organs, tissues, etc., Organ transplantation, Tissue transplantation, Transplantation

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Brief report: correction of X-linked hyper-IgM syndrome by allogeneic bone marrow transplantation

Article Abstract:

A five-month-old boy with X-linked hyper-immunoglobulin (Ig) M syndrome received a successful bone marrow transplant and regained normal immune function within 6 months. X-linked hyper-IgM syndrome is a genetic immunodeficiency disease in which patients fail to convert IgM to other immunoglobulins (IgG, IgA, IgE) and may develop potentially fatal opportunistic infections. The patient had a family history of the syndrome, including two fatal cases, and had developed a Pneumocystis carinii infection. As a result, he received an allogeneic bone marrow transplant from his sister. Incorporation of the transplant was shown by a change in blood type and altered expression of the gene which had caused the syndrome. Six months after the transplant the patient had normal levels of IgG, IgA, and IgE and normal T- and B-cell counts. Seventeen months after the transplant his condition remained good. In general, the relationship of genetic make-up to the severity of this syndrome is not understood.

Pediatric diseases, Genetic aspects, Immunologic diseases, Immunologic diseases in children

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