Explore Alpha-1 Antitrypsin Deficiency
Alpha-1 antitrypsin (AAT) deficiency has no cure, but its related lung diseases have many treatments. Most of these treatments are the same as the ones used for a lung disease called COPD (chronic obstructive pulmonary disease).
If you have symptoms related to AAT deficiency, your doctor may recommend:
Augmentation (og-men-TA-shun) therapy is a treatment used only for people who have AAT-related lung diseases. This therapy involves getting infusions of the AAT protein. The infusions raise the level of the protein in your blood and lungs.
Not enough research has been done to show how well this therapy works. However, some research suggests 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.
Researchers are working on possible treatments that will target the faulty AAT genes and replace them with healthy genes. These treatments are in the early stages of development.
Researchers also are studying therapies that will help misshapen AAT proteins move from the liver into the bloodstream. They're also studying a type of augmentation therapy in which the AAT protein is inhaled instead of injected into a vein.
If you're interested in new treatments, ask your doctor about ongoing clinical trials for AAT deficiency.
Clinical trials are research studies that explore whether a medical strategy, treatment, or device is safe and effective for humans. To find clinical trials that are currently underway for Alpha-1 Antitrypsin Deficiency, visit www.clinicaltrials.gov.
September 2, 2014
Gary H. Gibbons
Researcher Brings Medicine One Step Closer to Widely Available Cure for Sickle Cell Disease
The NHLBI updates Health Topics articles on a biennial cycle based on a thorough review of research findings and new literature. The articles also are updated as needed if important new research is published. The date on each Health Topics article reflects when the content was originally posted or last revised.