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Treatments for AAT Deficiency

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WebMD Feature
Reviewed by James E. Gerace, MD

If you have alpha-1 antitrypsin deficiency (AAT deficiency), there's treatment that can help you can feel better, live longer, and breathe easier.

Augmentation Therapy

There's only one specific treatment that actually targets COPD caused by AAT deficiency -- augmentation therapy. It's also called replacement therapy. It’s been around for 25 years, but it's now attracting more attention. 

It's used alongside the regular COPD treatments.   " Augmentation therapy for [AAT deficiency] seems to be very effective," says Robert A. Sandhaus, MD, PhD, clinical director of the Alpha-1 Foundation and professor of medicine at National Jewish Health in Denver. "I think it could change the prognosis for the condition."

If you have AAT deficiency, you don't have enough of the AAT protein that normally protects the lungs from damage. Augmentation therapy raises your levels of that protein.

You get an infusion through an IV tube into a vein in your arm. Augmentation therapy won't improve your breathing on the spot. But the extra AAT moves through your blood to your lungs and may slow down COPD damage. 

Side effects are usually mild. You may have a headache, muscle aches, or flu-like symptoms that last about a day.

The infusion takes about an hour. You need to get it every week, probably for the rest of your life. Usually, you would get it at a hospital or medical center, or you may be able to learn how to do it at home on your own.

 

How Well Does Augmentation Therapy Work?

There's debate about how well augmentation therapy works. Some studies show it can reduce further lung damage and help people with AAT deficiency live longer. Other studies have had less clear results.

Even so, the bulk of the evidence seems to show it helps. "We don't have the most rigorous evidence yet, but it seems that augmentation therapy can slow down the disease," says Norman Edelman, MD, chief medical officer of the American Lung Association.

Sandhaus is more positive. "I'm very confident it works," he says. Sandhaus is one of the authors of a new study of augmentation therapy. The results of the study will be presented at the American Thoracic Society International Conference in 2013. It was funded by CSL Behring.

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