Alpha-1-antitrypsin deficiency

Treatment for 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.

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Treatment for COPD

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.

It’s also very important to recognise and treat chest infections as quickly as possible to minimise damage to your lungs. Infections which turn your phlegm green will be treated with antibiotics.

If you’ve developed COPD, your treatment might include inhalers, pulmonary rehabilitation, flu vaccination or oxygen therapy. There’s also some evidence that people with AATD can benefit from lung volume reduction surgery, though the benefits for people with AATD may be less than for people with usual COPD.

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 people with standard 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 to undergo rigorous testing of your other major organs – such as the heart, kidneys and liver – to make sure they 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 2016, 321 people were waiting for lung transplants. In 2015-16, 179 people had lung transplants.


Treatment for liver disease

The Children’s Liver Disease Foundation website 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 and slow down the progression of COPD caused by AATD. It’s available in 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 and 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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Last medically reviewed: September 2017. Due for review: September 2020

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.