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Alpha-1-antitrypsin deficiency

Treatment for alpha-1-antitrypsin deficiency

On this page we cover the different treatments available for conditions caused by alpha-1-antitrypsin deficiency.

At the moment, NICE does not recommend any specific treatment in the UK for alpha-1-antitrypsin deficiency (AATD). If you have a condition caused by AATD, such as COPD or liver disease, the focus is on usual treatment for those conditions.

On this page:

If you’re a smoker and you have AATD, the most important thing you can do to avoid damage to your lungs is stop smoking.

Treatment for COPD

Your treatment might include inhalers, pulmonary rehabilitation, or oxygen therapy. There’s also some evidence that people with AATD can benefit from lung volume reduction procedures, though the benefits for people with AATD may be less than for people with usual COPD. You should also have your regular flu vaccination.

It’s also very important to recognise and treat chest infections as quickly as possible to minimise damage to your lungs. Make sure you have an action plan that you’ve agreed with your health care professional so you know what to do if you have a flare-up of your symptoms. Take a look at our checklist.

Lung transplant

For the small number of people with AATD whose lungs become severely damaged, a lung transplant might be an option which may significantly improve your quality of life.

Because people with AATD often develop COPD at a younger age than other people with COPD, they may be more suitable for a lung transplant. This is because they are often fitter with fewer other health conditions compared to older people so they cope better with the surgery and treatment. Though there’s no absolute cut-off age for a lung transplant, it’s unusual for people to have this much over the age of 65.

You’ll only be considered for a lung transplant after very careful assessment and advice. Potential candidates need:

  • a healthy body mass index (BMI)
  • no other significant medical conditions or infections
  • an emotional and physical support network

To be considered suitable you’ll first have lots of tests to make sure your other major organs – such as the heart, kidneys and liver – will work well after the transplant. You’ll be assessed by a regional transplant centre. If you’re accepted, your name will be placed on a lung transplant waiting list for a suitable organ to become available.

Unfortunately, there aren’t enough organs available for transplant to meet the current demand in the UK. Not all people who are suitable will get one in time. In March 2019, 331 people were waiting for lung transplants. In 2018-19, 158 people had lung transplants.

Treatment for liver disease

The Children’s Liver Disease Foundation has more information on treating liver disease caused by AATD.

Possible future treatments

AAT augmentation therapy is a treatment where alpha-1-antitrypsin (AAT) is given into your veins to increase the levels in your body. The idea is to try to slow down the progression of COPD caused by AATD. It’s available in some other countries, but not currently approved in the UK.

Trials so far show that augmentation therapy can have a measurable effect on the appearance of emphysema on patients’ CT scans. It’s not clear how this finding translates into a meaningful clinical benefit for people with AATD. One possibility is that augmentation therapy will become available in the future for some people – for example those whose symptoms and lung damage get worse very quickly, once they’ve stopped smoking and are receiving the best possible treatment. The decision will depend on a review by experts in AATD management considering all the evidence about risks and benefits.

Research is also looking at drugs that have the same function as AAT, ways to correct the gene that causes AATD, to stop AAT collecting in the liver and to promote the release of AAT into the blood.

Next: Staying healthy with alpha-1-antitrypsin deficiency >

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Download our AATD information (PDF, 163KB)

Last medically reviewed: August 2020. Due for review: August 2023

This information uses the best available medical evidence and was produced with the support of people living with lung conditions. Find out how we produce our information. If you’d like to see our references get in touch.