Primary Immunodeficiency (PI) : Treatments

by National Institute of Health

(Page 5 of 13)

Treating PI involves not only curing infections but also correcting the underlying immunodeficiency. In addition, any associated conditions, such as autoimmune disorders or cancer, need special attention.

Treating Infections

The first goal of treatment is to clear up any current infection. Doctors can prescribe a wide range of infection-fighting antimicrobials. Some are broad-spectrum antibiotics that combat a range of germs. Others zero in on specific germs.

When an infection fails to respond to standard medications, the patient may need to be hospitalized to be treated with antibiotics and other drugs intravenously.

For chronic infections, a variety of medicines can help relieve symptoms and prevent complications. These may include drugs like aspirin or ibuprofen to ease fever and general body aches, decongestants to shrink swollen membranes in the nose, sinuses, or throat, and expectorants to thin mucus secretions in the airways.

People who have chronic respiratory infections may be made more comfortable with a technique known as postural drainage (or bronchial drainage). Developed for persons with cystic fibrosis, postural drainage uses gravity, along with light blows to the chest wall, to help clear secretions from the lungs.

Preventing Infections

When the immune defenses are weak, it is essential to avoid germs. Precautions range from common sense practices like good hygiene (using mild soaps to keep the skin clean and brushing teeth twice a day) and good nutrition to elaborate measures to prevent all contact with infectious agents.

Anyone with an immunodeficiency needs to avoid unnecessary exposure to infectious agents. This means staying away from people with colds or other infections, and avoiding large crowds. (On the other hand, it is important not to become overly cautious. Children are encouraged to attend school, to play in small groups, and to participate in sports.)

Antibiotics are important for preventing or controlling infections. If infections threaten to become chronic, the doctor may prescribe continuous long-term low-dose antibiotics. Such preventive, or "prophylactic," therapy may help prevent hearing loss or permanent breathing problems.

When Pneumocystis pneumonia is a danger - for instance, in children with a profound T cell deficiency - an appropriate prophylactic treatment may consist of a combination of two drugs, trimethoprim and sulfamethoxazole.

Correcting Immunodeficiencies

Not long ago, little could be done to actually cure an immunodeficiency. Today, researchers have developed several possibilities for replenishing the immune defenses. No single approach works for all immunodeficiencies or in all cases but, taken together, these new treatments have transformed a dismal prognosis into one of hope and promise.

For several life-threatening immunodeficiencies, bone marrow transplantation (BMT) offers the chance of a dramatic, complete, and permanent cure. Since the first BMT was performed in 1968, nearly 1,000 children with PI, including SCID, Wiskott-Aldrich Syndrome, Leukocyte Adhesion Defect, and other disorders, have shown a remarkable recovery. They recover from infections, gain weight, and move on to essentially normal lives.

Unfortunately, bone marrow transplants don't work for everyone. To be successful, the transplant needs to come from a donor whose body tissues are a close biological "match." That is, the donor's tissues and the recipient's tissues should have identical, or nearly identical, sets of marker molecules (known as HLA antigens) that serve as unique tissue ID tags.

Without a good match, a reaction known as graft-versus-host disease (GVHD) may occur, in which cells in the donor marrow see the recipient's tissues as foreign and react against them.

Because tissue marker molecules come in many varieties, finding a good match is not easy. With new techniques and the availability of large donor banks, however, finding a suitable match is easier. The best matches are likely to be with close relatives, especially brothers or sisters.

Another option is marrow from a close relative - typically a parent - who shares half of the patient's major HLA antigens (and many of the minor antigens as well.) Cleansed of mature T cells that could trigger a GVHD, such half-matched transplants have saved the lives of many children.

BMT works especially well for SCID, because children with SCID lack T cells that could attack the bone marrow graft and cause rejection. Anyone with T cells may need to be treated, prior to transplantation, with radiation or drugs. Although this eliminates the recipient's T cells, it also temporarily wipes out other immune defenses, further increasing the patient's risk of infection.

Even with a good match, BMT does not always succeed. Results are best when the child is young, in fairly good health, and free of serious infection at the time of the transplantation.

About the Author

NIH is the nation's medical research agency - making important medical discoveries that improve health and save lives. The National Institutes of Health (NIH), a part of the U.S. Department of Health and Human Services, is the primary Federal agency for conducting and supporting medical research.