Intravenous alpha-1 antitrypsin augmentation therapy for treating patients with alpha-1 antitrypsin deficiency and lung disease

Alpha-1 antitrypsin deficiency is an inherited disorder that can cause lung disease. It affects about 1 in 1600 to 5000 people. Those with lung disease suffer from shortness of breath, reduced ability to exercise and wheezing. People who smoke are more seriously affected and have a greater risk of dying from the disease. We reviewed the benefits and harms of treating patients who have the form of the disease that affects the lungs with alpha-1 antitrypsin extracted from human plasma. We found two randomised trials (total 140 patients) comparing this treatment with placebo for two to three years. All patients were ex-smokers or had never smoked but had the genetic problem that carried a high risk of developing lung problems. Neither trial reported on the number of lung infections, hospital admissions or death from the disease. The studies found no difference between the two groups in quality of life or in number of exacerbations of the disease. The lung function deteriorated slightly less measured by CT scan, but slightly more measured by forced expiratory volume in one second. Therapy with alpha-1 antitrypsin cannot be recommended, in view of the lack of evidence of clinical benefit and the high cost of treatment.