Alpha1-Antitrypsin Deficiency Medication

  • Author: Paul Fairman, MD; Chief Editor: Zab Mosenifar, MD   more...
 
Updated: Mar 16, 2011
 

Medication Summary

The most important health intervention for a person with alpha1-antitrypsin (AAT) deficiency 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 alpha1-antitrypsin deficiency 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.

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 alpha1-antitrypsin deficiency. 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, ie, 60 mg/kg/wk given IV.

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Class Summary

These drugs are used for long-term replacement in individuals with clinically demonstrable panacinar emphysema.

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Alpha1-protease inhibitor (Prolastin)

 

Sterile, stable, lyophilized preparation of purified human alpha1-antiprotease inhibitor prepared from pooled plasma. Each unit of plasma tested for HIV, hepatitis B, and hepatitis C before inclusion in product. Product heat-treated to reduce potential risk of infectious-agent transmission. No cases of viral infections have been attributed to the product. Indicated as replacement (or augmentation) for normal serum alpha1-antiprotease to prevent progression of emphysema.

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Alpha1-proteinase inhibitor (Aralast)

 

Sterile, stable, lyophilized preparation of purified human alpha1-antiprotease inhibitor prepared from pooled human plasma by using cold alcohol fractionation process followed by further purification steps. Each unit of plasma tested for HIV, hepatitis B, and hepatitis C before inclusion in product. Product treated with solvent detergent mixture to inactivate viral agents to reduce potential risk of infectious-agent transmission. No cases of viral infections have been attributed to the product. Indicated as replacement (or augmentation) for normal serum alpha1-antiprotease to prevent progression of emphysema in patients with congenital deficiency of AAT with clinically evident emphysema.

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Alpha1-proteinase inhibitor (Zemaira)

 

Sterile, stable, lyophilized preparation of purified human alpha1 antiprotease inhibitor prepared from pooled plasma by using cold alcohol fractionation process followed by further purification steps. Each unit of plasma tested for HIV, hepatitis B, and hepatitis C before inclusion in product. Product treated with solvent detergent mixture to inactivate viral agents to reduce potential risk of infectious-agent transmission. No cases of viral infections have been attributed to the product. Indicated as replacement (or augmentation) for normal serum alpha1-antiprotease to prevent progression of emphysema in patients with congenital deficiency of AAT with clinically evident emphysema.

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Contributor Information and Disclosures
Author

Paul Fairman, MD  Director, Pulmonary Hypertension Service, Professor, Department of Internal Medicine, Division of Pulmonary and Critical Care Medicine, Virginia Commonwealth University

Paul Fairman, MD is a member of the following medical societies: American College of Chest Physicians, American College of Physicians, and American Thoracic Society

Disclosure: Gilead Honoraria Speaking and teaching; United Therapeutics Corp Honoraria None

Coauthor(s)

Rajiv Malhotra, DO  Assistant Professor, Department of Internal Medicine, Division of Pulmonary and Critical Care Medicine, Virginia Commonwealth University Health System

Rajiv Malhotra, DO is a member of the following medical societies: American College of Chest Physicians, American College of Physicians, and American Osteopathic Association

Disclosure: Nothing to disclose.

Specialty Editor Board

Ryland P Byrd Jr, MD  Professor, Department of Internal Medicine, Division of Pulmonary Medicine and Critical Care Medicine, Program Director of Pulmonary Diseases and Critical Care Medicine Fellowship, James H Quillen College of Medicine, East Tennessee State University; Medical Director of Respiratory Therapy, James H Quillen Veterans Affairs Medical Center

Ryland P Byrd Jr, MD is a member of the following medical societies: American College of Chest Physicians and American Thoracic Society

Disclosure: Nothing to disclose.

Francisco Talavera, PharmD, PhD  Adjunct Assistant Professor, University of Nebraska Medical Center College of Pharmacy; Senior Pharmacy Editor, eMedicine

Disclosure: eMedicine Salary Employment

Om Prakash Sharma, MD, FRCP, FCCP, DTM&H  Professor, Department of Medicine, Division of Pulmonary and Critical Care Medicine, University of Southern California Keck School of Medicine

Om Prakash Sharma, MD, FRCP, FCCP, DTM&H is a member of the following medical societies: American Academy of Allergy Asthma and Immunology, American College of Chest Physicians, American College of Physicians, American Federation for Medical Research, American Osler Society, American Thoracic Society, New York Academy of Medicine, and Royal Society of Medicine

Disclosure: Nothing to disclose.

Timothy D Rice, MD  Associate Professor, Departments of Internal Medicine and Pediatrics and Adolescent Medicine, St Louis University School of Medicine

Timothy D Rice, MD is a member of the following medical societies: American Academy of Pediatrics and American College of Physicians

Disclosure: Nothing to disclose.

Chief Editor

Zab Mosenifar, MD  Director, Division of Pulmonary and Critical Care Medicine, Director, Women's Guild Pulmonary Disease Institute, Professor and Executive Vice Chair, Department of Medicine, Cedars Sinai Medical Center, University of California, Los Angeles, David Geffen School of Medicine

Zab Mosenifar, MD is a member of the following medical societies: American College of Chest Physicians, American College of Physicians, American Federation for Medical Research, and American Thoracic Society

Disclosure: Nothing to disclose.

References

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Close-up chest radiograph of the right lower zone of a 39-year-old woman with alpha1-antitrypsin (AAT) deficiency. Normal lung markings are absent in the costophrenic angle. Some lung markings are present in the pericardiac region, but even these are diminished.
CT scan of the right middle and right lower lobes in a 38-year-old patient with alpha1-antitrypsin (AAT) deficiency. Entire middle lobe and much of the lower lobe are emphysematous; normal lung structures have been replaced by abnormal airspaces. Only the posterior portions of the right lower lobe maintain a normal architecture.
Graph outlines alpha1-antitrypsin (AAT) levels and risk of lung disease for the 5 most common phenotypes of AAT deficiency. Dashed line at 11 mmol/L (80 mg/mL) represents the threshold level below which emphysema is common.
 
 
 
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