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There are effective treatment options for most patients with primary immunodeficiency (PI), although the types of treatments differ based on the type of PI.
Antibody replacement therapy
Antibody replacement therapy involves infusion of plasma-derived immunoglobulins, primarily IgG. Regular infusions of intravenous immunoglobulin (IVIG) are used to treat patients with antibody deficiencies, such as X-linked agammaglobulinemia, common variable immunodeficiency (CVID), hyper-IgM syndrome, Wiskott-Aldrich syndrome, and severe combined immunodeficiency (SCID). Because it replaces but does not stimulate production of antibodies, IVIG therapy must be used continuously.
Hematopoietic stem cell transplantation
Hematopoietic stem cell transplantation is the process in which stem cells from a normal donor are given to a PI patient. In the past, stem cells were taken from bone marrow, but now new techniques support separation from peripheral blood and cord blood. Stem cell transplantation is commonly used with PIs involving granulocytes, T-cell or combined T-cell and B-cell deficiencies, such as severe combined immunodeficiency disease (SCID), Wiskott-Aldrich syndrome, hyper-IgM syndromes and chronic granulomatous disease.
Learn more about hematopoietic stem cell transplantation:
- Buckley R.H. et al. Hematopoietic Stem-Cell Transplantation for the Treatment of Severe Combined Immunodeficiency.New England Journal of Medicine. 1999 Feb 18;340(7):508-16 (View Abstract)
- Haddad E et al. Long-Term Immune Reconstitution and Outcome After HLA-Nonidentical T-Cell-Depleted Bone Marrow Transplantation for Severe Combined Immunodeficiency: A European Retrospective Study of 116 Patients. Blood. 1998 May 15;91(10):3646-53 (View Abstract)
- Myers LA. Hematopoietic Stem Cell Transplantation for Severe Combined Immunodeficiency. Blood. 2002 Feb 1;99(3):872-8 (View Abstract)
Gamma-Interferon, PEG-ADA, and G-CSF
Gamma-Interferon, PEG-ADA, and G-CSF are therapies derived from enzymes and proteins which target specific processes in specific PIs. Gamma-Interferon may be used to help phagocytes kill bacteria more effectively in chronic granulomatous disease (CGD). PEG-ADA may be used in treating infants with a rare form of SCID involving deficiency of an enzyme, adenosine deaminase (ADA). G-CSF (Granulocyte-Colony Stimulating Factor) may be used with patients undergoing hematopoietic stem cell transplant or those with low numbers of granulocytes.
Gene therapy
Gene therapy involves replacement of the defective gene with a normal copy. Although there have been positive outcomes with young patients with several of the SCID diseases, this type of therapy is still regarded as experimental.
Learn more about gene therapy:
- Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. Gene therapy for immunodeficiency diseases. Seminars in Hematology. 2004 Oct;41(4):272-8. (View Abstract)
- Kohn DB. Gene Therapy for Genetic Haematological Disorders and Immunodeficiencies. Journal of Internal Medicine. 2001 Apr;249(4):379-90 (View Abstract)
These links provide a list of web sites about immune diseases that may be of interest to healthcare professionals. Baxter Healthcare Corporation does not possess editorial control over the content or the information on those other sites. As such, Baxter Healthcare Corporation and the compiler of the list make no representation as to the accuracy, completeness and any other aspect of the information contained in those sites.
Study abstracts have been provided to assist you in selecting literature that may be of interest in your practice. As a professional courtesy, we will be happy to provide full text articles upon request. Please feel free to contact our Medical Information-Professional Services department toll-free at 1-866-4BIOSCI (424-6724).
- Blaese RM, Winkelstein J. Patient & Family Handbook for Primary Immunodeficiency Diseases. Towson, MD: Immune Deficiency Foundation; 2007, p.93-103.
