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There are many components to treating patients with alpha1-antitrypsin (AAT) deficiency. The goal is to maintain better lung function. This can be done through smoking cessation, asthma medications (if necessary), infection control, good nutrition, environment modifications, exercise, and stress management. Also, replacement therapy helps restore the natural balance of enzymes in the lungs and protects from the damage caused by neutrophil elastase. An available treatment is Prolastin®. Since 1988, over 2 million doses of Prolastin® have been administered.
Drug Therapy for Alpha-1
Drug therapy is among the most important type of medical therapy for the newly diagnosed individual with AAT deficiency.
1. Vaccinations (influenza/pneumonia)
It's important to have a yearly flu vaccine and a Pneumovax® shot every 5 to 6 years. Since your lungs are vulnerable to pollutants and infections, the use of these vaccinations is of the utmost importance. Furthermore, you may find this is the easiest and most convenient type of therapy available. Effective vaccines are available for hepatitis A and B. These are especially important in patients with established liver disease.
Recommendations:
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Annual flu vaccine
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Administration or confirmation of Pneumovax vaccine (every 5 to 6 years)
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Hepatitis A vaccine
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Hepatitis B vaccine |
2. Aggressive treatment of lung infections
Prompt and aggressive treatment of infections is recommended due to the increased neutrophil elastase burden during periods of infection. It's important to notify your doctor immediately when you suspect a lung infection. Here is a list of symptoms you should watch for carefully:
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fever
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increased shortness of breath
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increase coughing (may not be productive)
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chills with fever
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changes in color of phlegm |
Because the lungs attract more leukocytes when an infection is present, and the leukocytes release neutrophil elastase, it is important to control lung inflammation. Antibiotics may help to speed recovery.
3. Aggressive evaluation of liver complications
It is important for parents, caregivers, or significant others to be aware and advised of any indication of complications related to liver disease.
Here is a list of common symptoms that may require therapy:
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increased abdominal swelling or edema of the extremities
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coughing up or vomiting bright red blood
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blood in toilet or diaper
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blackish, purplish or dark-colored stools
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confusion, crankiness, unusual crying, disorientation, lethargy
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little or no urine
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dark (tea- or cola-colored) urine
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lack of energy, easily fatigued
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fever
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no appetite/refusal to eat or drink
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itching or increased itching
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peripheral edema
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change in or the appearance of jaundice |
It's very important to carefully read the labels on over-the-counter medications. Be sure to inform your doctor or other healthcare provider if any alternative medicines or vitamin supplements are being taken.
4. Bronchodilators
Your doctor may prescribe bronchodilators to relieve your AAT deficiency lung-related symptoms. Bronchodilators relax the large muscles around your airways so that more air is allowed in and out. There are 2 main kinds of bronchodilators: beta-agonists and anticholinergics. Beta-agonists are typically quick-acting "rescue" medications. They work in 5 to 15 minutes and last from 4 to 6 hours. Anticholinergics are longer-lasting than beta-agonists, but still fall in the quick-acting category.
5. Corticosteroids
Inhaled corticosteroids can be useful as a preventive treatment for Alpha-1 lung disease and oral corticosteroids may be prescribed by your doctor, particularly during flare-ups (which are referred to by healthcare as "exacerbations").
6. Supplemental oxygen
Supplemental oxygen may be needed for some alpha-1 related lung disease. Oxygen is important for individuals with low blood oxygen levels, during active infections and/or with progressive destruction of the lung tissue. Supplemental oxygen may be needed during exercise or sleep.
For some people with AAT deficiency, supplemental oxygen is especially important when traveling by air, because cabin pressure changes with altitude.
7. Augmentation therapy
Augmentation Therapy may be recommended for certain patients with lung disease. It is not used for patients with liver disease.
Important Safety Information
Prolastin®, Alpha1-Proteinase Inhibitor (Human) is indicated for chronic replacement therapy of individuals having congenital deficiency of alpha-1 PI (alpha1-antitrypsin deficiency) with clinically demonstrable panacinar emphysema. Weekly Prolastin® therapy has demonstrated a low occurrence of side effects. In clinical studies with Prolastin®, reactions were observed in 1.16% of infusions, the most common events being fever (0.77%), light-headedness (0.19%), and dizziness (0.19%). As with all plasma-derived therapeutics, the potential to transmit infectious agents cannot be totally eliminated. Individuals with selective IgA deficiencies who have known antibody against IgA (anti-IgA antibody) should not receive Prolastin®, since these patients may experience severe reactions, including anaphylaxis, to IgA which may be present.
You are encouraged to report negative side effects of prescription drugs
to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Please click here for Prolastin® full Prescribing Information.
Remember, your doctor or healthcare provider is the single best source of information regarding you and your health. Please consult your doctor or healthcare provider if you have any questions about your health or any of your medications.
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