How Is Alpha-1 Antitrypsin Deficiency Treated?

Alpha-1 antitrypsin (AAT) deficiency has no cure. However, the lung diseases linked to this inherited condition have many treatments. Most of these treatments are the same as the ones given to people who have lung diseases without AAT deficiency.

If you have emphysema or other lung diseases or symptoms related to AAT deficiency, your doctor may recommend:

• Medicines called inhaled bronchodilators (brong-ko-di-LA-tors) that help open your airways and make breathing easier. These medicines also are used to treat asthma and chronic obstructive pulmonary disease. 
• Flu and pneumococcus (noo-mo-KOK-us) vaccines to protect you from diseases that could make your condition worse.
• Pulmonary rehabilitation (rehab). This involves treatment by a team of experts at a special clinic. In rehab, you learn how to manage your condition and function at your best.
• Extra oxygen if needed.
• A lung transplant. You may need a transplant if your lung is so badly damaged that it severely affects your breathing. If you have a good chance of surviving the transplant surgery, you may be a candidate for it.

Augmentation therapy is a type of treatment given only to people who have AAT-related lung diseases. This therapy involves getting infusions of the AAT protein. This raises the level of the protein in your blood and lungs.

Not enough research has been done to show whether this type of therapy works. However, some suggest that this therapy may slow the development of AAT deficiency in people who don't have severe disease.

People who have AAT deficiency and develop related liver or skin diseases will be referred to doctors who treat those diseases.

Future Treatments

Researchers are working on possible treatments that will target the altered AAT genes and replace them with healthy genes. These treatments are in early stages of development.

If you're interested, talk to your doctor about whether any clinical trials of new AAT treatments might be right for you.