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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.
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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
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Collect 25-50 Billion White
Blood Cells Using Apheresis
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Isolate Precursor Cells
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Transfer Normal Gene
Into Precursor Cells
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Intravenously Administer Precursor
Cells Containing Normal Gene
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December 22, 1998
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