Synopsis

Source of validity: Scientific
Rating (out of 5): 4

Globulins, specifically alpha-1 antitrypsin (AAT), are scientifically validated for use in treating certain forms of emphysema. Emphysema, a component of chronic obstructive pulmonary disease (COPD), may be caused by an inherited deficiency of alpha-1 antitrypsin, a type of globulin produced in the liver. This deficiency leads to unregulated activity of neutrophil elastase, which damages lung tissue and results in early-onset emphysema. AAT augmentation therapy involves intravenous infusion of purified human AAT (a globulin) to raise blood and lung levels of the protein in deficient individuals. Multiple studies have shown that this therapy can slow the decline in lung function, reduce the frequency of exacerbations, and decrease lung tissue destruction, as measured by CT density and pulmonary function tests in patients with severe AAT deficiency. Major guidelines (such as those from the American Thoracic Society and European Respiratory Society) recommend AAT augmentation therapy for individuals with documented severe deficiency and evidence of emphysema. However, this treatment is not indicated for most other forms of emphysema unrelated to AAT deficiency, nor does it reverse existing lung damage. The evidence base is strongest for its use in genetically confirmed AAT-deficient patients, with ongoing research into optimal dosing and long-term outcomes. In summary, the use of globulins (specifically AAT) for emphysema is scientifically validated, but applies only to a subset of patients with a specific genetic cause.

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