Treatments and drugs

By Mayo Clinic Staff

Treatments for primary immunodeficiency involve preventing and treating infections, boosting the immune system, and treating the underlying cause of the immune problem. In some cases, primary immune disorders are linked to a serious illness, such as an autoimmune disorder or cancer, which also needs to be treated.

Managing infections

  • Antibiotics. Infections are typically treated with antibiotics. In cases where infections don't respond to standard medications, hospitalization and treatment with intravenous (IV) antibiotics may be necessary. Some people need to take antibiotics long term to prevent infections from occurring and to prevent permanent damage to the lungs and ears.
  • Treating symptoms. You may need medications to relieve symptoms caused by infections, such as ibuprofen for pain and fever, decongestants for sinus congestion, and expectorants to help clear your airways of mucus.

Treatment to boost the immune system

  • Immunoglobulin therapy. Also called gamma globulin therapy, this treatment can be a lifesaver for people who have an antibody deficiency. Immunoglobulin consists of antibody proteins needed for the immune system to fight infections. It can either be injected into a vein through an IV line or inserted underneath the skin (subcutaneous infusion). Treatment with intravenous gamma globulin is needed every few weeks to maintain sufficient levels of immunoglobulins. Subcutaneous infusion is needed once or twice a week.
  • Gamma interferon therapy. Interferons are naturally occurring substances that fight viruses and stimulate immune system cells. Gamma interferon is a man-made (synthetic) substance given as an injection in the thigh or arm three times a week. It's used to treat chronic granulomatous disease, one form of primary immunodeficiency.
  • Growth factors. When immune deficiency is caused by a lack of certain white blood cells, growth factor therapy — such as granulocyte-macrophage colony-stimulating factor (Leukine) and granulocyte colony-stimulating factor (Neupogen, Neulasta) — can help increase the levels of immune-strengthening white blood cells.

Treatment to cure primary immunodeficiency

  • Stem cell transplantation. Stem cell transplantation offers a permanent cure for several forms of life-threatening immunodeficiency. With this treatment, normal stem cells are transferred to the person with immunodeficiency, giving them a normally functioning immune system. Stem cells can be harvested through bone marrow, or they can be obtained from the placenta at birth (cord blood banking). For stem cell transplantation to work, the donor — usually a parent or other close relative — must have body tissues that are a close biological match to those of the person with primary immunodeficiency. Stem cells that aren't a good match may be rejected by the immune system. But even with a good match, stem cell transplants don't always work. Additionally, the treatment often requires that any functioning immune cells be destroyed using chemotherapy or radiation before the transplants, leaving the transplant recipient temporarily even more vulnerable to infection.

Future treatments

  • Gene therapy. Researchers hope this treatment will one day be a cure for primary immune disorders and many other conditions. Gene therapy actually replaces defective genes with genes that work correctly. A harmless virus is used to carry the genes into the body's cells. In turn, the newly introduced genes trigger the production of healthy immune system enzymes and proteins. Experts have identified many of the genes that cause primary immune deficiencies — but they still need to work out many problems. For example, some of the missing or defective genes are only activated during the early development of the immune system, so even if scientists can figure out how to get that gene where it needs to be, it would also have to trigger the development of the missing functions. Although the technique has shown promise in some initial trials, gene therapy is still experimental.
Jul. 30, 2013