Augmentation therapy consists of weekly IV infusions of alpha-1 antitrypsin derived from human plasma. It is used to increase the concentration of the protein in the blood and lungs. Augmentation therapy is the only FDA-approved treatment for alpha-1 antitrypsin deficiency. Long-term controlled clinical trials have not been done to show that augmentation therapy alters the course of lung disease; however, it is currently the standard of care for lung-affected patients under certain circumstances. You should discuss any questions you have about augmentation therapy with your doctor.

Clinical Criteria for Use

Currently, augmentation therapy can only be prescribed for patients with Alpha-1 related emphysema and certain other rare manifestations of Alpha-1. This is not a treatment option for Alpha-1 liver disease. Augmentation therapy cannot be recommended for individuals with normal lung function. It should be reserved for those patients with phenotypes Pi Z, Pi Z/null, Pi null null, and/or patients who have Alpha-1 serum levels of less than 11 micromoles. It is not generally given to people who have mildly deficient phenotypes.

• Augmentation can be administered in a doctor's office, hospital clinic or in a facility where intravenous infusions are routinely given. Additionally, many companies offer home infusion services. You must check with your individual insurance carrier as criteria for services and benefits vary greatly.
   
• Patients who are motivated and physically able may be taught to administer augmentation therapy. A willing family member or significant other may also be trained and be involved in the process and care. It is imperative for individuals interested in self infusion discuss their intention with their physician and develop of plan of care. They must identify a nurse willing to work with them on a consistent basis to complete training on all aspects of therapy. Patient safety with home self-infusion is always a primary goal.

Safety of Augmentation Therapy

Augmentation therapy is prepared from pooled human plasma that has been screened for hepatitis A, B, and C and tested for HIV. Additional antiviral procedures are utilized as a precaution against transmission of infectious agents.

Known Side Effects

There are relatively few side effects that have been reported: headaches, muscle and joint pain, and lower-back pain are the most frequent complaints. For patients with severe COPD or heart failure, worsening of shortness of breath may occur.
 

Your doctor will probably test you for IgA deficiency prior to starting augmentation therapy because if you have both Alpha-1 Antitrypsin Deficiency and IgA, you could develop a severe allergic reaction (anaphylaxis) to the Alpha-1 augmentation therapy.

Augmentation Therapy Resources