About AAT Deficiency
Alpha1-proteinase inhibitor (A1-PI) deficiency*, also known as alpha-1 antitrypsin (AAT) deficiency, is an autosomal, co-dominant, hereditary disorder characterized by low serum and lung levels of A1-PI. Severe forms of the deficiency are frequently associated with slowly progressive, moderate to severe panacinar emphysema that most often manifests in the third to fourth decades of life, resulting in a significantly lower life expectancy.1
*Also known as Alpha-1, alpha-1 antitrypsin deficiency, AAT Deficiency, AATD, A1-PI deficiency, A1AD, or hereditary or genetic emphysema.
Individuals with A1-PI deficiency have little protection against neutrophil elastase (NE) released by a chronic, low-level of neutrophils in their lower respiratory tract, resulting in a protease:protease inhibitor imbalance in the lung. The emphysema associated with A1-PI deficiency is typically worse in the lower lung zones. It is believed to develop because there are insufficient amounts of A1-PI in the lower respiratory tract to inhibit NE. This imbalance allows unopposed destruction of the connective tissue framework of the lung parenchyma.1
The American Lung Association estimates there are approximately 100,000 people in the United States who suffer from alpha-1 antitrypsin (AAT) deficiency. 2 It is believed that up to 90% of those with AAT deficiency are undiagnosed.3 Not all individuals with AATD will develop symptomatic emphysema.
GLASSIA [Alpha1-Proteinase Inhibitor (Human)]
GLASSIA is indicated for chronic augmentation and maintenance therapy in individuals with emphysema due to congenital deficiency of alpha1-proteinase inhibitor (Alpha1-PI), also known as alpha1-antitrypsin (AAT) deficiency.
- The effect of augmentation therapy with GLASSIA or any Alpha1-PI product on pulmonary exacerbations and on the progression of emphysema in Alpha1-PI deficiency has not been demonstrated in randomized, controlled clinical trials.
- Clinical data demonstrating the long term effects of chronic augmentation and maintenance therapy of individuals with GLASSIA are not available.
- GLASSIA is not indicated as therapy for lung disease in patients in whom severe Alpha1-PI deficiency has not been established.
Detailed Important Risk Information for GLASSIA
- GLASSIA is contraindicated in IgA deficient patients with antibodies against IgA. GLASSIA is contraindicated in individuals with a history of severe immediate hypersensitivity reactions, including anaphylaxis, to Alpha1-PI products.
- GLASSIA is made from human plasma. It may carry a risk of transmitting infectious agents, such as viruses, and theoretically, the Creutzfeldt-Jakob disease (CJD) agent.
- Administer GLASSIA at room temperature at a rate not greater than 0.04 mL/kg body weight per minute. If anaphylactic or severe anaphylactoid reactions occur, discontinue the infusion immediately.
- Administer GLASSIA within 3 hours of entering the vials.
- Safety and effectiveness in patients over 65 years of age have not been established.
- Two serious adverse reactions observed on two separate occasions during clinical studies with GLASSIA were cholangitis and exacerbation of chronic obstructive pulmonary disease (COPD).
- The most common product-related adverse reactions in clinical studies were headache and dizziness.
Please see GLASSIA Full Prescribing Information for full prescribing details.
ARALAST NP [Alpha1-Proteinase Inhibitor (Human)]
ARALAST NP is indicated for chronic augmentation therapy in patients having congenital deficiency of A1-PI with clinically evident emphysema.
- The effect of augmentation therapy with ARALAST NP on pulmonary exacerbations and on the progression of emphysema in alpha1-antitrypsin deficiency has not been demonstrated in randomized, controlled clinical trials.
- Clinical data demonstrating the long-term effects of chronic augmentation or replacement therapy of individuals with ARALAST NP or ARALAST are not available.
- ARALAST NP is not indicated as therapy for lung disease patients in whom congenital A1-PI deficiency has not been established.
Detailed Important Risk Information for ARALAST NP
- ARALAST NP is contraindicated in IgA deficient patients with antibodies against IgA, due to the risk of severe hypersensitivity.
- ARALAST NP is derived from pooled human plasma. It may carry a risk of transmitting infectious agents, e.g., viruses and theoretically, the Creutzfeldt-Jakob disease (CJD) agent.
- The recommended rate of administration (≤0.08 mL/kg/min) should be closely followed and vital signs monitored continuously. If anaphylactic or severe anaphylactoid reactions occur, the infusion should be discontinued immediately.
- Safety and effectiveness in patients over age 65 years of age have not been established.
- ARALAST NP should be administered at room temperature within three (3) hours after reconstitution and should be administered alone, without mixing with other agents or diluting solutions.
- The safety of ARALAST NP was evaluated with ARALAST in a crossover clinical PK comparability study. The most common adverse events deemed related to ARALAST NP included headache and musculoskeletal discomfort. No serious adverse reactions or deaths were reported in the study. In the ARALAST pivotal study, the most common adverse events were headache and somnolence.
Please see ARALAST NP Full Prescribing Information for full prescribing details.
References
- ARALAST NP [Alpha1–Proteinase Inhibitor (Human)] Prescribing Information, Baxter International Inc., Westlake Village, CA; April 2010.
- American Lung Association. COPD Fact Sheet. http://www.lungusa.org/lung-disease/copd/resources/facts-figures/COPD-Fact-Sheet.html. Accessed June 16, 2011.
- Silverman EK, Sandhaus RA. Alpha1-Antitrypsin Deficiency. N Engl J Med. 2009;360:2749-57.
