What is the drug treatment for alpha1-antitrypsin deficiency (AATD)?

Updated: Sep 11, 2020
  • Author: Dora E Izaguirre Anariba, MD, MPH; Chief Editor: John J Oppenheimer, MD  more...
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The most important health intervention for a person with AAT deficiency (AATD) is avoiding cigarette smoking. Smoking clearly advances the progression of emphysema in severely deficient individuals by as much as 15 years over their nonsmoking counterparts.

Airflow obstruction and symptoms resulting from AATD can be treated in a manner similar to emphysema. Bronchodilators may provide relief of some symptoms. Use antibiotics to treat bacterial complications, including pneumonia or purulent bronchitis. Neither bronchodilators nor antibiotics demonstrate any effect on disease progression. Likewise, corticosteroids may provide some short-term relief, but they have no proven long-term benefit in inhaled or oral preparations. Because of their long-term adverse effects, avoid oral steroids. For more information, see Emphysema.

Prescribe oxygen if patients are hypoxemic at rest, with activity, or during sleep.

Consider replacement (or augmentation) therapy to slow the progression of emphysema. At present, IV augmentation therapy is the only FDA-approved treatment specific for AATD. It is most clearly indicated for patients with moderate degrees of airflow obstruction (FEV1 35-65% of predicted). Three preparations are available. Although purifications and/or preparations differ, all are equivalent, and none have been a cause of hepatitis or HIV infection. Each is approved at the same dose and administration, that is, 60 mg/kg/wk given IV. See the Treatment section for more detail.

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