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Saturday, November 22, 2008
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Primary Immunodefiency Diseases

 

Improved Therapy Means a Better and Longer Life

Research has led to improved therapy for individuals with primary immunodeficiency diseases.

Bone Marrow Transplantation. Transplantation of bone marrow cells from a family member with identical human leukocyte antigens (HLA) has led to normal immune function in patients with combined immunodeficiencies. HLA proteins mark the donor tissue as self, which minimizes the potential for rejection by the recipient's immune system. Investigators are working to improve success using bone marrow from an unrelated HLA-matched donor for the two-thirds of patients who lack an HLA-identical family member and to use stem cell transplantation (stem cells can generate all the cells of the immune system).

Replacement of Antibodies. Research has demonstrated the safety and efficacy of replacement therapy with intravenous immunoglobulin (i.e., antibodies) in patients with defects in antibody production. This treatment is life saving, prevents many types of infection, and should be given to all primary immunodeficiency disease patients with defects in antibody production.

Complement Proteins
Two brothers with inherited antibody
deficiency receiving intravenous
immunoglobulin to provide antibodies
Antimicrobials. The use of antibiotics to treat and prevent infections is a key element in the treatment of primary immunodeficiency disease patients. NIAID-supported investigators helped identify new antimicrobial agents and new treatment regimens. For example, NIAID researchers showed that chronic granulomatous disease patients receiving daily doses of the antibiotic cotrimoxazole had 65 percent fewer infections.

Immunomodulation. Immune system molecules can be injected into patients to improve immune function. NIAID researchers showed that the immune system molecule interferon gamma improves the function of the white blood cells called neutrophils in patients with chronic granulomatous disease, reducing infections by 70 percent.



Stimulate Immune Cell
Precursors with Growth Factor

Collect 25-50 Billion White
Blood Cells Using Apheresis

Isolate Precursor Cells
Transfer Normal Gene
Into Precursor Cells

Intravenously Administer Precursor
Cells Containing Normal Gene


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December 22, 1998