Treatments for Alpha-1 (Alpha-1 Antitrypsin Deficiency)

Medically Reviewed by David T. Derrer, MD on August 13, 2014
3 min read

If you have alpha-1 antitrypsin deficiency, treatment can help you can feel better, live longer, and breathe easier.

There's only one specific treatment to fight alpha 1: augmentation therapy. It's also called replacement therapy. It’s been around for 25 years, but it's attracting more attention.

"Augmentation therapy for alpha-1 seems to be very effective," says Robert A. Sandhaus, MD, PhD, clinical director of the Alpha-1 Foundation.

If you have alpha-1, you don't have enough of a protein that normally protects the lungs from damage. Augmentation therapy raises your levels of that protein.

You get it through an IV tube into a vein in your arm. The extra alpha-1 moves through your blood to your lungs and may slow down the damage.

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

The treatment 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.

There's debate about how well augmentation therapy works. Some studies show that it can reduce further lung damage and help people with alpha-1 live longer. Other studies have had less clear results.

"We don't have the most rigorous evidence yet, but it seems that augmentation therapy can slow down the disease," says Norman Edelman, MD, senior medical advisor 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, funded by CSL Behring.

These treatments help with the symptoms:

Drugs to help with breathing. For breathing problems, COPD treatments can help. These may include inhaled drugs called bronchodilators that open up the airways. Inhaled steroids can reduce the swelling in the lungs.

Antibiotics. Alpha-1 makes it more likely to get a lung infection. Your doctor may prescribe antibiotics to head off problems.

Vaccinations.  Protect yourself from dangerous infections that could make your symptoms worse. Get your flu, pneumococcal, and hepatitis shots.

Oxygen. Home and portable systems allow you to breathe extra oxygen.

There's a lot you can do on your own to improve your health. 

Make lifestyle changes. "If you have alpha-1 and you smoke, quitting is the single most important change you need to make," Edelman says. Smoking can speed up the damage to your lungs. If you live with a smoker, do whatever you can to get them to stop. Don't let him smoke in the house, and limit exposure to dust, chemical fumes, and pollution, too.

Work with an expert. Alpha-1 often doesn't get diagnosed. "You might be your doctor's first case," Sandhaus says. "That happens pretty often." If you want a second opinion or different treatment options, look for an alpha-1 expert.

Keep up-to-date. Treatment is changing. Sandhaus believes that within 4 years, you may be able to get augmentation therapy through an inhaler instead of by IV. Studies of different drugs, gene therapy, and other new techniques could change the course of the disease.

Be your own advocate. If you have concerns about your medical care, share them with your doctor. And get another opinion if you think you need it.

Sandhaus has received funding for clinical studies from CSL Behring, AstraZeneca, Grifols, and Kamada.