Drug and Other Therapies

These are among the most most important types of medical therapy for the newly diagnosed individual with Alpha-1 Antitrypsin Deficiency. Although these are most relevant to lung-affected individuals, remember all Alphas have risks to their lungs. Some of the therapies listed are relevant for all Alpha-1 patients.

It’s important to have a yearly flu vaccine and a Pneumovax® shot every five to six years. Since your lungs are vulnerable to pollutants and infections, the use of these prophylactic vaccinations is of the utmost importance. Furthermore, your patient 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:

• Annual flu vaccine
• Administration or confirmation of Pneumovax vaccine (every 5 to 6 years)
• Hepatitis A vaccine
• Hepatitis B vaccine

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:

• fever
• increased shortness of breath
• increase coughing (may not be productive)
• chills with fever
• 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.

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:

• increased abdominal swelling or edema of the extremities
• coughing up or vomiting bright red blood
• blood in toilet or diaper
• blackish, purplish or dark-colored stools
• confusion, crankiness, unusual crying, disorientation, lethargy
• little or no urine
• dark (tea- or cola-colored) urine
• lack of energy, easily fatigued
• fever
• no appetite/refusal to eat or drink
• itching or increased itching
• peripheral edema
• 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 (such as Milk Thistle) or vitamin supplements are being taken.

Your doctor may prescribe bronchodilators to relive your Alpha-1 lung-related symptoms. Bronchodilators relax the large muscles around your airways so that more air is allowed in and out. There are two main kinds of bronchodilators: beta-agonists and anticholinergics. An example of a beta-agonist is the quite common asthma medication albuterol. 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. An example of this subclass is Atrovent.

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”).

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.

Supplemental oxygen is also recommended during exercise. For some Alphas, it is especially important when traveling by air, because cabin pressure changes with altitude.

Augmentation Therapy may be recommended for certain patients with lung disease. It is not used for patients with liver disease.