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Who should receive augmentation therapy?

Augmentation therapy should be given to individuals with documented emphysema and severe Alpha-1 (defined as individuals with two abnormal alpha-1 genes). There has been some controversy, however, about giving augmentation therapy to anyone whose lung disease is very mild or very severe.

In some research studies conducted since the introduction of augmentation therapy, the benefits identified were primarily seen in certain groups of people. In these studies, the most dramatic effects were seen in those people whose lung function fell in the mid-range of severity.

Some members of the healthcare community have used these results incorrectly, to suggest that only those with moderate lung disease should receive augmentation therapy. However, because augmentation therapy is considered preventive rather than curative – it puts the brakes on lung destruction rather than reversing it – it seems logical to begin therapy as soon as possible after Alpha-1 lung disease has been detected, especially if the lung disease is progressing in spite of eliminating risk factors such as smoking.

Augmentation Therapy – AlphaNet Canada

Intravenous augmentation therapy in those with AATD is recommended for:
• Individuals with an FEV1 less than or equal to 65% predicted.

For those with lung disease related to AATD and an FEV1 greater than 65%, we recommend discussion with each individual regarding the potential benefits of reducing lung function decline with consideration of the cost of therapy and lack of evidence for such benefit.

• Individuals with necrotizing panniculitis.

Intravenous augmentation therapy is not recommended for:
Individuals with the MZ genotype of AATD.
Individuals with lung disease due to AATD who continue to smoke.
Individuals with AATD and emphysema or bronchiectasis who do not have airflow obstruction.
The treatment of liver disease due to AATD.
Individuals who have undergone liver transplantation.

Additional recommendations regarding dosing of intravenous augmentation therapy:
Weekly doses higher than the current FDA-approved dose are not recommended.
Monitoring of trough AAT blood levels to evaluate the adequacy of AAT augmentation dosing is not recommended.

Clinical Practice GuidelinesCOPD Foundation

The use of IV augmentation therapy with plasma-derived alpha1-antitrypsin (AAT) has become the standard of care for the treatment of pulmonary disease associated with the severe genetic deficiency of AAT. The Medical and Scientific Advisory Committee of the Alpha-1 Foundation has become aware that physicians are prescribing this expensive blood product for the treatment of individuals with a single abnormal AAT gene, primarily the PI*MZ genotype. We are aware of no evidence that such therapy is effective in this patient population. The most important therapeutic interventions in such patients remain smoking cessation and elimination of other risk factors for lung disease. This commentary discusses the treatment of AAT deficiency and the concerns regarding treatment of PI*MZ individuals. We conclude that clinicians should avoid prescribing augmentation therapy for this heterozygote population.

alpha1-Antitrypsin augmentation therapy for PI*MZ heterozygotes: a cautionary noteNational Library of Medicine