Treating Alpha-1
Explore the treatment options for managing alpha-1 antitrypsin deficiency, including augmentation therapy, medications, and other supportive treatments to help manage lung and liver health.
Augmentation / Replacement Therapy for Alpha-1-related Lung Disease
Augmentation or replacement therapies, as they are called – have been developed specifically for treating Alpha-1 lung disease. Glassia®, Prolastin-C® and Zemaira® are approved for use in Canada. These therapies aim to replace or augment the missing Alpha-1 antitrypsin (AAT) protein and, in this way, hopefully slow or stop the progress of the disease. All are given by intravenous infusion once a week.
The primary goal of augmentation therapy is to increase or supplement the level of alpha-1 protein in the blood. Alpha-1 antitrypsin protects the lungs from the destructive effects of neutrophil elastase, an enzyme released by our body’s white blood cells as they respond to inflammation or infection.
The ultimate goal is to is to reduce the rate of decline of lung function towards normal by replacing the deficient protein. The therapy cannot restore lost lung function — nor is it considered a cure. There is also some evidence that augmentation therapy can reduce the frequency and severity of pulmonary exacerbations (flare-ups of lung disease), and it appears to be an effective treatment for the Alpha-1-related skin disease, Necrotizing Panniculitis.
Things to Consider before beginning augmentation therapy
Before beginning augmentation therapy, a patient should be tested for IgA deficiency, a hereditary condition that increases the likelihood of potentially severe allergic reactions to plasma products.
AlphaNet Canada recommends that all Alphas be considered for immunization against Hepatitis A and B to reduce the risk of liver injury. Vaccination entails a series of three injections, generally administered over six months. Augmentation therapy can be started before receiving the hepatitis vaccine or whether or not the vaccine is administered.
Are there any side effects?
Alphas receiving augmentation therapy have reported a variety of side effects, although the vast majority never have any significant problems. The most common side effect is feeling tired or having flu-like symptoms that typically last up to 24 hours after an infusion. These symptoms can often be reduced or eliminated by slowing the infusion rate. Some Alphas have symptoms that seem like mild allergic reactions: rash or hives, itching, tightness in the chest, dyspnea, and wheezing. Many of them can continue receiving augmentation therapy if they take an antihistamine, such as Benadryl, before their infusions. Rarely are side effects severe enough to cause an Alpha to stop augmentation therapy entirely.
NOTE: While augmentation therapy is considered the only specific therapy for Alpha-1 lung disease, the treatment plan for lung-affected people with Alpha-1 should also include the appropriate use of antibiotics, an immunization program including viral hepatitis and influenza strains, reduction or elimination of environmental risk factors, appropriate inhaled medications, an exercise program, and oxygen, if needed.
Bronchodilator
Bronchodilators are used to open up the narrowed breathing passages. The same airway opening inhalers used by people with chronic obstructive lung disease (COPD) are used by Alpha-1 emphysema patients. These medications generally fall into two categories:
Anticholinergics
These drugs work on the narrow passageways and neutralize the nerve pathways of the cholinergic system to stop spasms of the small muscles that encircle airways. Anticholinergic drugs include short-acting Atrovent (ipratropium bromide) and long-acting Spiriva (tiotropium).
BETA2-Agonists
These drugs have a different target than the anticholinergics. Beta2-agonists target the beta2 receptors in the muscles encircling the airways. This is a safe and selective version of the well-known drug adrenaline. Drugs in this category include short-acting bronchodilators such as Ventolin (salbutamol), Airomir (salbutamol), Apo-solvent (salbutamol), Bricanyl (terbutaline), Beretoc (fenoterol), and long-acting bronchodilators such as Oxeze (formoterol), Foradil (formoterol) and Serevent (salmeterol).
Anti-inflammatory medicines
These drugs reduce inflammation but are not ‘rescuers’ like bronchodilators. They are used mainly to prevent inflammation.
Corticosteroids
These can be taken by inhaler and go directly to the lungs and do not get into the blood system. Drugs in this category include Flovent (fluticasone), Qvar (beclomethasone), Alvesco (ciclesonide) and Pulmicort (budesonide). They are portable but heavy and must be pulled around on a cart. They are the type most often seen in hospitals for ambulatory patients.
Prednisone
Prednisone is a powerful anti-inflammatory drug taken orally. It is sometimes used when an exacerbation or flare-up of Alpha-1 COPD occurs.
Antibiotic
People with Alpha-1 may suffer from lung damage during chest infections and need to treat them early and aggressively so that damage is minimized.
Supplemental Oxygen
Supplemental or home oxygen may be required if a person’s oxygen levels are persistently low. There are currently three common types of delivery systems.
Liquid Oxygen
Oxygen is stored in liquid form in a reservoir that looks like a three-foot-tall robot. Oxygen tubing can be hooked up to it directly or used to fill portable canisters, which are becoming more user-friendly and compact. The canisters are usually of two types: continuous flow, where oxygen flows both as the person inhales and exhales, and on-demand or pulse systems that release oxygen only on inhalation. This newer technology allows the oxygen to last almost double the time of the continuous systems.
Concentrator
An oxygen concentrator is a machine about the size of an end table. It uses an electric motor and works by removing nitrogen from room air to make 94-98 percent oxygen. An oxygen concentrator requires an electrical source and operates by passing room air through a powder or a membrane to separate oxygen from nitrogen. The oxygen is concentrated and delivered while the nitrogen is periodically released back into the air. It is the least expensive oxygen delivery system but is restricted to in-home use. Its other disadvantage is that it is noisy and raise the electricity bill.
Portable oxygen concentrators (POC) are also available for those who desire a small, lightweight and affordable portable oxygen solution in a compact and mobile unit. These concentrators vary in weight, size, oxygen flow settings, liters per minute, battery life, and other specifications. POCs are not restricted to in-home use. They have adapters for charging in automobiles and are also outfitted with battery packs. Many of these units have been approved for use on airplanes, as well.
Cylinder
Cylinders contain oxygen in the form of compressed gas and can be fitted with a conserving device to prevent the escape of oxygen so that they can last a long while if not in use. Cylinders can be portable but are quite heavy and must be pulled around on a cart. They are the type most often seen in hospitals for ambulatory patients.
Lung Volume Reduction Surgery (LVRS)
Lung Volume Reduction Surgery (LVRS) involves cutting out those parts of the overinflated lung that are no longer performing their function. The theory behind this is that the remaining healthy tissue will have more room to expand and work more efficiently by removing the useless tissue. This surgery is thought not to be very appropriate for people with Alpha-1 as the damage to their lungs is mainly in the lower lobes, whereas LVRS is usually performed on the upper lobes. A study is currently underway to assess the value of LVRS. It involves a cross-section of people with emphysema, not just those with Alpha-1. For more information, contact your AlphaNet Canada Coordinator.
